[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"duchenne-muscular-dystrophy\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:duchenne-muscular-dystrophy":26},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,38,0,25,[9,43,68,99,129,153,184,207,234,261,289,320,347,372,393,420,440,464,486,504,524,545,562,580,604],{"id":10,"slug":11,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":15,"acronym":4,"eligibilityCriteria":16,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":24,"conditions":25,"keywords":27,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100491764","affinity-beyond-anti-aav8-antibody-assessment-study-of-males-with-dmd-100491764",false,"NCT05683379","AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD","Anti-AAV8 Antibody Assessment Study of Males With Duchenne Muscular Dystrophy Aged 0 to \u003C25 Years","Inclusion Criteria:\n\n* Males at least 0 to \\\u003C25 years of age\n* Diagnosis of DMD\n* Provision of signed and dated informed consent form (ICF) and assent as required per local regulations or requirements\n\nExclusion Criteria:\n\n* Prior participation in a gene therapy trial OR recipient of a gene therapy drug\n* Other inclusion\u002Fexclusion criteria apply","MALE","0 Years","25 Years",{"count":21,"type":22},200,"ESTIMATED","OBSERVATIONAL","This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD).",[26],"Duchenne Muscular Dystrophy",[28,26,29],"DMD","Duchenne","RECRUITING","2026-06-25",{"date":33,"type":34},"2026-06-30","ACTUAL",{"date":36,"type":34},"2022-12-20",{"date":38,"type":22},"2026-09",{"name":40,"class":41},"REGENXBIO Inc.","INDUSTRY",1,{"id":44,"slug":45,"hasResults":12,"nctId":46,"briefTitle":47,"officialTitle":48,"acronym":4,"eligibilityCriteria":49,"healthyVolunteers":12,"sex":17,"minAge":50,"maxAge":4,"enrollmentInfo":51,"targetDuration":4,"studyType":53,"phases":54,"briefSummary":57,"conditions":58,"keywords":59,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":61,"startDateStruct":62,"completionDateStruct":64,"leadSponsor":66,"locationsCount":67},"100492515","phase-2-affinity-duchenne-rgx-202-gene-therapy-in-participants-with-duchenne-muscular-dystrophy-dmd-100492515","NCT05693142","AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)","A Phase 1\u002F2\u002F3 Open-label Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacodynamics, and Pharmacokinetics of Intravenous RGX-202 Gene Therapy in Males With Duchenne Muscular Dystrophy (DMD)","Part 1 - Key Inclusion Criteria:\n\n* The participant's legal guardian(s) is (are) willing and able to provide written, signed informed consent prior to any study-related procedures; and, where applicable, the minor participant has provided written or verbal assent according to local requirements.\n* Is a male at least 4 years of age and less than 12 years of age at consent or 1 to \\\u003C4 years of age at the time of dosing and ≥ 10 kg at the time of screening.\n* Must meet any of the following criteria:\n\n  * DMD gene mutation in exons 18 and above, and a clinical picture consistent with typical DMD with the exception of a participant (Cohort 1b) with DMD gene mutation in exons 12-17.\n  * Participant is able to walk 100 meters independently without assistive devices. Cohort 2c participant must be able to walk 10 meters independently without assistive devices. Cohort 1b participant must be able to walk with or without assistive devices.\n  * Participant is able to complete the TTSTAND per protocol-specific criteria.\n  * Participant has been on a stable dose of systemic glucocorticoids according to the standard of care for at least 12 weeks. Cohort 2c participants must be consistently on or off a stable dose of systemic glucocorticoids according to the standard of care for at least 12 weeks.\n  * Clinical laboratory test results, including hepatic and renal function, are within the normal range during screening, or if abnormal, are not clinically significant, in the opinion of the investigator.\n  * Documentation is provided at screening visit for participant's adherence to the local country's vaccination schedule. The parent(s) or legal guardian(s) must be willing to have their child receive a meningococcal vaccine, if not already vaccinated.\n  * Participant and parent(s)\u002Flegal guardian(s) are willing and able to comply with scheduled visits, study intervention administration plan, and study procedures.\n\nPart 2 and 3 Inclusion Criteria:\n\n* The participant's legal guardian(s) is (are) willing and able to provide written, signed informed consent prior to any study-related procedures; and, where applicable, the minor participant has provided written or verbal assent according to local requirements.\n* DMD gene mutation with any mutation except for those with deletions or point mutations in exons 8, 9 and\u002For 10.\n* Participant is able to complete the TTSTAND per protocol-specific criteria.\n* Clinical laboratory test results, including hepatic and renal function, are within the normal range during screening, or if abnormal, are not clinically significant, in the opinion of the investigator.\n* Documentation is provided at screening visit for participant's adherence to the local country's vaccination schedule. The parent(s) or legal guardian(s) must be willing to have their child receive a meningococcal vaccine, if not already vaccinated.\n* Participant and parent(s)\u002Flegal guardian(s) are willing and able to comply with scheduled visits, study intervention administration plan, and study procedures.\n* Is a male at least 1 year of age and ≥ 10 kg at the time of screening.\n* Participants 1 to \\\u003C4 years of age must meet the following criteria:\n\n  * is able to walk 10 meters independently without assistive devices.\n  * must be consistently on or off a stable dose of systemic glucocorticoids according to the standard of care for at least 12 weeks.\n* Participants 4 years and older must meet the following criteria:\n\n  * are able to walk 100 meters independently without assistive devices.\n  * have been on a stable dose of systemic glucocorticoids according to the standard of care for at least 12 weeks.\n  * have a NSAA total score ≥16.\n\nPart 1 Exclusion Criteria:\n\n* Participant has any condition that would contraindicate treatment with immunosuppression.\n* Participant has received ataluren (a protein restoration therapy) or an exon-skipping therapy for the treatment of DMD within 6 months of study entry or is unable to refrain from taking ataluren or exon-skipping therapy for a duration of 5 years from the time of RGX-202 administration.\n* Participant has received any investigational or commercial gene therapy product over his lifetime.\n* Participant is currently taking any other investigational intervention (other than corticosteroids) or has taken any other investigational intervention (other than corticosteroids) within 3 months prior to the scheduled Day 1 intervention. If your corticosteroid is vamorolone, the participant will be asked to temporarily convert his daily dosing to prednisolone\u002Fprednisone during a short period of time around RGX-202 administration. He will be allowed to revert back to his baseline vamorolone regimen at the original per kilogram dose at which he entered the study and should remain on this for 24 months unless the investigator determines that this is not clinically indicated or possible.\n* Participant has impaired cardiac function defined as a left ventricular ejection fraction of \\\u003C 55% on screening cardiac assessments (echocardiogram or MRI).\n* Participant is not a good candidate for the study, in the opinion of the investigator.\n\nPart 2 and 3 Exclusion Criteria:\n\n* Participant has any condition that would contraindicate treatment with immunosuppression.\n* Participant has received givinostat within 3 months of study entry or has received ataluren (a protein restoration therapy) or an exon-skipping therapy for the treatment of DMD within 6 months of study entry or is unable to refrain from taking ataluren or exon-skipping therapy for a duration of 5 years from the time of RGX-202 administration.\n* Participant has received any investigational or commercial gene therapy product over his lifetime.\n* Participant is currently taking any other investigational intervention (other than corticosteroids) or has taken any other investigational intervention (other than corticosteroids) within 3 months prior to the scheduled Day 1 intervention. If your corticosteroid is vamorolone, the participant will be asked to temporarily convert his daily dosing to prednisolone\u002Fprednisone during a short period of time around RGX-202 administration. He will be allowed to revert back to his baseline vamorolone regimen at the original per kilogram dose at which he entered the study and should remain on this for 24 months unless the investigator determines that this is not clinically indicated or possible.\n* Participant has detectable AAV8 total binding antibodies in serum.\n* Participant has impaired cardiac function defined as a left ventricular ejection fraction of \\\u003C 55% on screening cardiac assessments echocardiogram or MRI).\n* Participant is not a good candidate for the study, in the opinion of the investigator.","1 Year",{"count":52,"type":22},65,"INTERVENTIONAL",[55,56],"PHASE2","PHASE3","RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain.\n\nThis is a multicenter, open-label dose evaluation clinical study to assess the safety, tolerability, and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in participants with Duchenne.\n\nFor additional information on how to participate (or be considered for the study), please follow this link: https:\u002F\u002Fmytomorro.ws\u002Faffinity-ct-gov",[26],[60,28,26,29],"Gene therapy",{"date":33,"type":34},{"date":63,"type":34},"2023-01-04",{"date":65,"type":22},"2028-08",{"name":40,"class":41},17,{"id":69,"slug":70,"hasResults":12,"nctId":71,"briefTitle":72,"officialTitle":73,"acronym":4,"eligibilityCriteria":74,"healthyVolunteers":12,"sex":17,"minAge":75,"maxAge":76,"enrollmentInfo":77,"targetDuration":4,"studyType":53,"phases":79,"briefSummary":81,"conditions":82,"keywords":83,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":89,"lastUpdatePostDateStruct":90,"startDateStruct":92,"completionDateStruct":94,"leadSponsor":96,"locationsCount":98},"100644768","phase-1-a-study-to-evaluate-the-tolerability-safety-and-efficacy-of-gnr-097-gene-therapy-in-pediatric-patients-with-duchenne-muscular-dystrophy-100644768","NCT07673809","A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy","Multicenter, Single-blind, Randomized, Placebo-controlled Study of a Single Intravenous Infusion of a Gene Therapy Product GNR-097 in Pediatric Patients With Duchenne Muscular Dystrophy","Inclusion Criteria:\n\n1. Written informed consent for participation in the trial.\n2. Ambulatory boys aged 4-9 years with a documented diagnosis of DMD and clinical manifestations of the disease.\n3. A frameshift mutation or nonsense mutation in the DMD gene.\n4. Сreatine phosphokinase level \\>5000 U\u002FL.\n5. Binding antibody titer to AAV9 ≤1:50 \\[method: ELISA\\].\n6. The patient is able to interact with the study physician and perform tests to assess functional activity.\n7. Results of functional activity assessment tests at screening (at least in one of the two attempts performed on different days):\n\n   * NSAA ≥22;\n   * time to rise from a supine position without using surrounding objects or furniture \\\u003C5 sec;\n   * 6MWT distance ≥350 m.\n8. The patient received oral glucocorticosteroids at a stable dose for ≥12 weeks prior to signing the Informed Consent Form, and it is planned that glucocorticosteroids will be continued during the screening stage and after the patient's inclusion in the study.\n9. For patients receiving deflazacort at study entry: switching the patient from deflazacort to prednisolone, in the opinion of the investigator, will not result in a significant deterioration in the patient's health.\n10. The patient has been immunized with a vaccine against meningococcal serotypes A, C, Y, W135 (and B, if available) no later than 4 weeks prior to administration of GNR-097\u002Fplacebo, and the immunization period expires no more than three months after the expected date of administration of GNR-097\u002Fplacebo.\n\nExclusion Criteria:\n\n1. Hypersensitivity to any component of GNR-097 or placebo.\n2. Patient with cognitive impairment or a sedentary lifestyle that, in the opinion of the investigator, may interfere with the development or manifestation of motor activity.\n3. Mutations in exons 8 and\u002For 9 of the DMD gene; for patients planned for inclusion in Cohort A, additionally: mutations in exons 1-17 and\u002For 59-71 of the DMD gene.\n4. Clinical signs of cardiomyopathy, including left ventricular ejection fraction (Simpson) \\\u003C40% based on echocardiography performed during screening.\n5. Contraindications to magnetic resonance imaging.\n6. History of any autoimmune disease, with the exception of drug-compensated autoimmune thyroiditis.\n7. History of tuberculosis; positive or indeterminate result of Diaskintest® TigraTest® or T-SPOT.TB screening.\n8. Positive results of tests for hepatitis B, hepatitis C, or HIV screening.\n9. Acute infectious diseases that resolved less than 4 weeks before administration of GNR-097\u002Fplacebo.\n10. Immunization with a live attenuated vaccine less than 3 months before administration of GNR-097\u002Fplacebo OR immunization with any inactivated vaccine less than 4 weeks before administration of GNR-097\u002Fplacebo.\n11. Abnormal laboratory parameters:\n\n    * GGT level is more than three upper limits of normal;\n    * total bilirubin \\>50.0 μmol\u002FL (except for patients with a confirmed diagnosis of Gilbert's syndrome);\n    * creatinine \\>160.0 μmol\u002FL;\n    * hemoglobin \\\u003C80 or \\>180 g\u002FL;\n    * white blood cell count \\>18,500\u002FμL;\n    * platelet count below the lower limit of normal.\n12. History of taking antisense oligonucleotides, ataluren, gene therapy using vector constructs, or cell therapy.\n13. Use of immunosuppressive drugs other than glucocorticosteroids less than 12 weeks prior to signing the Informed Consent Form.\n14. Participation in clinical trials less than 6 months prior to signing the Informed Consent Form.\n15. Unwillingness or inability of the patient and\u002For their parent\u002Flegal guardian to comply with the protocol requirements and\u002For the trial procedures.\n16. Other diseases or conditions not listed above that, in the opinion of the physician investigator and\u002For the Sponsor, prevent the patient from participating in the trial, including for safety reasons.","4 Years","9 Years",{"count":78,"type":22},32,[80,55],"PHASE1","The study will evaluate the tolerability, safety and efficacy of gene therapy product in boys with Duchenne muscular dystrophy (DMD). In Phase I the participants will be included in two sequential dose cohorts with increasing doses of the investigational product. Based on the results of Phase I, the dose of the investigational product for use in Phase II will be determined. Phase II is a randomized, single-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.",[26],[26,28,84,85,86,87,88],"Ambulatory","Gene Therapy","Micro-dystrophin","AAV","AAV9","2026-06-23",{"date":91,"type":34},"2026-06-29",{"date":93,"type":34},"2025-09-30",{"date":95,"type":22},"2029-08-02",{"name":97,"class":41},"AO GENERIUM",6,{"id":100,"slug":101,"hasResults":12,"nctId":102,"briefTitle":103,"officialTitle":104,"acronym":105,"eligibilityCriteria":106,"healthyVolunteers":12,"sex":17,"minAge":107,"maxAge":4,"enrollmentInfo":108,"targetDuration":4,"studyType":53,"phases":110,"briefSummary":111,"conditions":112,"keywords":113,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":119,"lastUpdatePostDateStruct":120,"startDateStruct":122,"completionDateStruct":124,"leadSponsor":126,"locationsCount":128},"100410604","phase-1-a-gene-transfer-therapy-study-to-evaluate-the-safety-of-and-expression-from-delandistrogene-moxeparvovec-srp-9001-in-participants-with-duchenne-muscular-dystrophy-dmd---non-ambulatory-cohort-100410604","NCT04626674","A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort","An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)","ENDEAVOR","Inclusion Criteria:\n\n* For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.\n* Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥3 at the Screening visit and has a total PUL score of ≥20 and ≤40 at the time of Screening.\n* Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study.\n* Cohort 1: Is ambulatory, and ≥4 to \\\u003C8 years of age at the time of Screening.\n* Cohort 2: Is ambulatory, and ≥8 to \\\u003C18 years of age at the time of Screening.\n* Cohort 3: Non-ambulatory per protocol specified criteria at the time of Screening.\n* Cohort 4: Is ambulatory and ≥3 to \\\u003C4 years of age at the time of Screening.\n* Cohort 5a: Is ambulatory and ≥4 to \\\u003C9 years of age with time to rise from the floor ≤7 seconds at the screening visit.\n* Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening.\n* Cohort 6: Is ambulatory, and ≥2 to \\\u003C3 years of age at the time of Screening.\n* Cohort 7: Non-ambulatory per protocol-specified criteria at the time of Screening.\n* Cohorts 4 and 6: Do not yet require use of chronic steroids for treatment of their DMD, in the opinion of the Investigator, and are not receiving steroids at the time of Screening.\n* Genetic mutation inclusion criteria vary by cohort.\n\nAll Cohorts:\n\n* Ability to cooperate with motor assessment testing.\n* rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.\n\nExclusion Criteria:\n\n* Cohort 8: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients.\n* Has a concomitant illness, autoimmune disease, chronic drug treatment, and\u002For cognitive delay\u002Fimpairment that in the opinion of the Investigator creates unnecessary risks for gene transfer.\n* Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol-specified time limits.\n* Abnormality in protocol-specified diagnostic evaluations or laboratory tests.\n\nNote: Other inclusion\u002Fexclusion criteria apply.","2 Years",{"count":109,"type":22},83,[80],"Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.\n\nThis is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.",[26],[26,114,85,28,115,116,117,118],"Gene-Delivery","Ambulatory Non-ambulatory","Pediatric","Adult","Dystrophin","2026-06-19",{"date":121,"type":34},"2026-06-24",{"date":123,"type":34},"2020-11-23",{"date":125,"type":22},"2028-02-29",{"name":127,"class":41},"Sarepta Therapeutics, Inc.",7,{"id":130,"slug":131,"hasResults":12,"nctId":132,"briefTitle":133,"officialTitle":134,"acronym":4,"eligibilityCriteria":135,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":136,"enrollmentInfo":137,"targetDuration":4,"studyType":53,"phases":139,"briefSummary":140,"conditions":141,"keywords":142,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":143,"lastUpdatePostDateStruct":144,"startDateStruct":146,"completionDateStruct":148,"leadSponsor":150,"locationsCount":152},"100526737","phase-1-a-study-of-sgt-003-gene-therapy-in-duchenne-muscular-dystrophy-inspire-duchenne-100526737","NCT06138639","A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)","A Phase 1\u002F2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)","Inclusion Criteria:\n\n* Cohort 1: 4 to \\\u003C7 years of age\n* Cohort 2: 7 to \\\u003C12 years of age\n* Cohort 3: 0 to \\\u003C 4 years of age\n* Cohort 4: 12 to \\\u003C 18 years of age\n* Cohort 5: 10 to \\\u003C 18 years of age\n* Participant ambulatory status at the time of Screening Part A or Rescreening, as defined by the ability to complete a 10-meter walk\u002Frun test in \\\u003C 30 seconds:\n\n  * Cohorts 1, 2, and 4: Ambulatory\n  * Cohort 3: Either ambulatory or non-ambulatory\n  * Cohort 5: Non-ambulatory, but having been previously ambulatory by history\n* Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype confirmed by Sponsor genetic testing. In cases where a genotype may be predictive of residual dystrophin production and\u002For a clear clinical diagnosis of DMD cannot be made (e.g., due to age), evaluation of dystrophin levels in baseline muscle biopsies may be required to determine eligibility under this criterion.\n* Negative for AAV antibodies.\n* Steroid regimen:\n\n  * Cohorts 1, 2, 4, and 5: A stable daily oral steroid regimen of at least 0.5 mg\u002Fkg\u002Fday of prednisone or 0.75 mg\u002Fkg\u002Fday of deflazacort for ≥12 weeks prior to Screening Part A or Rescreening, allowing for weight-based modifications consistent with clinical practice.\n  * Cohort 3: N\u002FA\n* Meet 10-meter walk\u002Frun time criteria\n* Meet time to rise from supine criteria\n* Cohort 5: Meet Performance of Upper Limb (PUL) 2.0 criteria\n* Participant has body weight: ≤ 90 kg\n\nExclusion Criteria:\n\n* Treatment with dystrophin modifying drugs within 3 months prior to screening.\n* Current or prior treatment with an approved or investigational gene transfer drug.\n* Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last administration, whichever is longer.\n* Established clinical diagnosis of DMD that is associated with any deletion mutation invariant or variant predicted to not express exons 1 to 11 or, exons 42 to 45, or exons 57 to 69, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing.\n\nOther inclusion or exclusion criteria apply.","17 Years",{"count":138,"type":22},60,[80,55],"This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this study. Cohort 1 will include participants 4 to \\\u003C 7 years of age. Cohort 2 will include participants 7 to \\\u003C 12 years of age. Cohort 3 will include participants 0 to \\\u003C 4 years of age. Cohort 4 will include participants 12 to \\\u003C 18 years of age. Cohort 5 will include participants 10 to \\\u003C 18 years of age. Initiation of participant enrollment in Cohorts 4 and 5 will be subject to the accrual of safety and efficacy data from Cohorts 1-3. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.",[26],[28,85],"2026-06-17",{"date":145,"type":34},"2026-06-22",{"date":147,"type":34},"2024-05-06",{"date":149,"type":22},"2031-05-06",{"name":151,"class":41},"Solid Biosciences Inc.",15,{"id":154,"slug":155,"hasResults":12,"nctId":156,"briefTitle":157,"officialTitle":158,"acronym":159,"eligibilityCriteria":160,"healthyVolunteers":12,"sex":17,"minAge":161,"maxAge":76,"enrollmentInfo":162,"targetDuration":4,"studyType":53,"phases":164,"briefSummary":165,"conditions":166,"keywords":169,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":174,"lastUpdatePostDateStruct":175,"startDateStruct":177,"completionDateStruct":179,"leadSponsor":181,"locationsCount":183},"100615022","phase-2-phase-2-study-of-sat-3247-in-pediatric-ambulatory-patients-100615022","NCT07287189","Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients","A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Dose Comparison and Exploratory Efficacy Study of Orally Administered SAT-3247 in Ambulatory DMD Patients","BASECAMP","Key Inclusion Criteria:\n\n* Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing with a confirmed mutation in the DMD gene.\n* Male DMD patients who are ambulatory and aged ≥ 7 to \\\u003C 10 years at the time of screening.\n* Stable dose of systemic glucocorticoids (i.e., prednisolone, deflazacort, or vamorolone) according to the standard of care for ≥ 3 months prior to the Screening Visit and for the duration of the trial. Patients who are not receiving glucocorticosteroids are also eligible if stopped ≥ 3 months prior to the Screening Visit.\n* Stable doses of prescription medicines including ACE inhibitors, β-blockers, and diuretics (excluding glucocorticosteroids) and over-the-counter medicines and\u002For herbal supplements for supportive care ≥ 1 month prior to the Screening Visit and for the duration of the trial.\n* Participants that have previously received delandistrogene moxeparvovec (brand name Elevidys) either in a prior clinical trial or in the commercial setting \\> 18 months prior to screening whose muscle function tests have stabilized or demonstrated decline ≥ 3 months prior to Screening, as determined by investigator and documented in chart notes, will be eligible.\n* Participants that have previously received an exon skipper \\> 6 months prior to Screening whose muscle function tests have stabilized or demonstrated decline ≥ 3 months prior to Screening, as determined by investigator and documented in chart notes, will be eligible.\n* Participants receiving a stable dose of givinostat (brand name Duvyzat) for at least 18 months or longer prior to the Screening Visit will be eligible. Participants unable to tolerate givinostat who discontinued treatment before 18 months are eligible to enroll if date of last dose is ≥ 30 days from the Screening date. Givinostat should not be discontinued, if tolerated, to meet study entry criteria.\n* Participants that have received prior treatment with an investigational gene therapy product (other than delandistrogene moxeparvovec) ≥ 24 months prior to the Screening Visit.\n* If participating in a physical therapy\u002Fstrength training regimen, must be stable for ≥ 2 months prior to the Screening Visit and for the duration of the trial.\n\nKey Exclusion Criteria:\n\n* Ambulatory patients expected to experience loss of ambulation within ≤ 12 months.\n* Participants for whom MRI or open muscle biopsy are contraindicated.\n* Evidence of significant hepatic dysfunction, defined as GLDH \\> 2X upper limit of normal (ULN) at the Screening Visit.\n* Impaired cardiac function defined as a left ventricular ejection fraction of \\\u003C 50% on screening cardiac assessments (echocardiogram or MRI) or evidence of symptomatic cardiomyopathy.\n* A forced vital capacity \\\u003C 60% predicted at the Screening Visit.\n* Ongoing participation in any other therapeutic clinical trial or follow-up study for a therapeutic intervention\n* Consumption of grapefruit juice or grapefruit containing products\n* Severe behavioural or cognitive problems that preclude participation in the study, in the opinion of the investigator.\n\nAdditional entry criteria will be reviewed with the clinical site investigator.","7 Years",{"count":163,"type":22},51,[55],"Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and \\\u003C 10 years. The trial will study two doses of SAT-3247 in a randomized, double-blind, placebo-controlled weekday regimen for 12 weeks to determine the optimal dose, safety, tolerability, and preliminary efficacy.",[26,29,28,167,168],"Neuromuscular Diseases","Muscular Dystrophies",[170,171,172,173],"muscle regeneration","satellite cell","asymmetric division","dystrophin","2026-06-12",{"date":176,"type":34},"2026-06-16",{"date":178,"type":34},"2025-12-08",{"date":180,"type":22},"2027-06-30",{"name":182,"class":41},"Satellos Bioscience, Inc.",21,{"id":185,"slug":186,"hasResults":12,"nctId":187,"briefTitle":188,"officialTitle":189,"acronym":190,"eligibilityCriteria":191,"healthyVolunteers":12,"sex":17,"minAge":107,"maxAge":75,"enrollmentInfo":192,"targetDuration":4,"studyType":53,"phases":194,"briefSummary":195,"conditions":196,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":197,"lastUpdatePostDateStruct":198,"startDateStruct":200,"completionDateStruct":202,"leadSponsor":204,"locationsCount":206},"100578906","phase-1-a-study-to-investigate-the-safety-and-biodistribution-of-a-single-intrathecal-it-injection-of-ins1201-in-ambulatory-males-with-duchenne-muscular-dystrophy-dmd-100578906","NCT06817382","A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)","A Phase 1, Multicenter, Open-label Study to Investigate the Safety and Biodistribution of a Single Intrathecal Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (The ASCEND Study)","ASCEND","Inclusion Criteria\n\n* Participant must be male at birth, 3 to \\\u003C5 years of age, inclusive (Part 1) and 2 to \\\u003C3 years of age (Part 2), at the time of legally authorized representative (LAR) signing and dating the informed consent form.\n* Ambulatory -as defined as the ability to walk at least 10 meters unassisted (ie, without personal assistance or use of any assistive devices) Note: children who have not yet developed the ability to walk by the time of screening (for whatever reason) will not be eligible for the study.\n* Has a definitive diagnosis of DMD prior to Screening or as part of Screening based on genetic testing. Note that participants who rescreen do not have to repeat genetic testing for the diagnosis of DMD if one is already on file. Genetic reports must describe a frameshift deletion, frameshift duplication, premature stop (\"nonsense\"), canonical splice site mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 58 (inclusive) that is expected to lead to absence of a functional dystrophin protein (mutations in exons 1-17 or 59-71 are therefore not permitted).\n* Able to cooperate with motor assessment testing.\n* Has received vaccinations recommended for the participant's age and DMD disease according to Centers for Disease Control and Prevention (CDC) Child and Adolescent Immunization Schedule by Age, World Health Organization, or local recommendation incorporating the Advisory Committee on Immunization Practices (ACIP) Vaccine Recommendations and Guidelines for Patients with Altered Immunocompetence.\n\nException is made for seasonal influenza and coronavirus disease 2019 (COVID-19) vaccines, for which shared decision-making with the participant's physician is encouraged.\n\nExclusion Criteria\n\n* Prior treatment with gene or cell-based therapy at any time.\n* Oligonucleotide-based exon skipping or small molecule stop codon readthrough-promoting therapies for at least 6 months prior to enrolment.\n* Has left ventricular ejection fraction \\\u003C 50% on the screening echocardiogram (ECHO) or clinical signs and\u002For symptoms of cardiomyopathy.\n* Has cardiac arrhythmia or significant electrocardiogram (ECG) interval abnormalities.\n* Major surgery within 3 months prior to Day 1 or planned surgery or procedures that would interfere with the conduct of the study at any time during this study.\n* The presence of any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic\u002Fallergic, behavioural disease, infection, unhealed injury, malignancy, concomitant illness, extenuating circumstance, or requirement for chronic drug treatment that, in the opinion of the Investigator:\n\n  1. Creates unnecessary risks for undergoing gene transfer;\n  2. Might compromise the participant's ability to comply with the protocol-required testing or procedures; or\n  3. Might compromise the participant's well-being, safety, or clinical interpretability.\n* Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection.\n* Has signs of clinically significant symptomatic infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.\n* Has contraindications for IT administration of the product or for lumbar puncture, such as anatomical abnormalities, bleeding disorders or other medical conditions (eg, spina bifida, meningitis, or significant clotting abnormalities).\n* Demonstrates cognitive or developmental delay or impairment that could confound assessment of motor development in the opinion of the Investigator.\n* Total serum anti-AAV9 antibody titers of \\> 1:50 as determined by ELISA within 14 days of Day 1.\n\nNote: Other inclusion\u002Fexclusion criteria may apply.",{"count":193,"type":22},12,[80],"The primary objective of this study is to evaluate the safety and tolerability of a single dose of INS1201 via IT administration in ambulatory male participants with DMD.",[26],"2026-06-10",{"date":199,"type":34},"2026-06-11",{"date":201,"type":34},"2025-07-22",{"date":203,"type":22},"2028-03-31",{"name":205,"class":41},"Insmed Gene Therapy LLC",10,{"id":208,"slug":209,"hasResults":12,"nctId":210,"briefTitle":211,"officialTitle":212,"acronym":4,"eligibilityCriteria":213,"healthyVolunteers":12,"sex":17,"minAge":161,"maxAge":214,"enrollmentInfo":215,"targetDuration":4,"studyType":53,"phases":217,"briefSummary":218,"conditions":219,"keywords":220,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":225,"lastUpdatePostDateStruct":226,"startDateStruct":228,"completionDateStruct":230,"leadSponsor":232,"locationsCount":233},"100605293","phase-3-a-study-of-sgt-003-gene-therapy-in-ambulant-males-with-duchenne-muscular-dystrophy-impact-duchenne-100605293","NCT07160634","A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)","A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy","Inclusion Criteria:\n\n* Participant is ambulatory.\n* Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype.\n* Negative for antibodies against adeno-associated virus.\n* On a stable daily oral regimen of at least 0.5 mg\u002Fkg\u002Fday prednisone or 0.75 milligrams per kilogram per day (mg\u002Fkg\u002Fday) deflazacort for at least 6 months prior to entering the study, allowing for weight-based dose modifications in accordance with clinical practice.\n* Meet 10-meter walk\u002Frun time criteria.\n* Meet time to rise from supine criteria.\n* Participant has bodyweight ≤50 kg.\n\nExclusion Criteria:\n\n* Current or prior treatment with an approved or investigational gene transfer drug or gene editing therapy.\n* Exposure to vamorolone, givinostat, approved or investigational dystrophin- or disease-modifying drugs (such as eteplirsen, golodirsen, casimersen, viltolarsen, and ataluren), or another investigational drug for any indication within 6 months or 5 half-lives, whichever is longer, prior to enrollment.\n* Established clinical diagnosis of DMD that is associated with any deletion variant or variant predicted not to express exons 1 to 11, exons 42 to 45, or exons 57 to 69, inclusive of the DMD gene as documented by a genetic report.\n\nOther Inclusion\u002FExclusion criteria to be applied as per protocol.","11 Years",{"count":216,"type":22},80,[56],"This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety outcomes. The study will be divided into 2 parts. Participants will be randomized 1:1 to either SGT-003 in Part 1 followed by placebo in Part 2 or to placebo in Part 1 followed by SGT-003 in Part 2. Participants will continue to be monitored in long term follow up (LTFU) for at least 5 years from their SGT-003 dosing date.",[26],[221,222,223,224],"SGT-003","Duchenne Muscular Dystrophy (DMD)","adeno-associated virus (AAV)","IMPACT DUCHENNE","2026-06-01",{"date":227,"type":34},"2026-06-02",{"date":229,"type":34},"2025-10-22",{"date":231,"type":22},"2034-01",{"name":151,"class":41},5,{"id":235,"slug":236,"hasResults":12,"nctId":237,"briefTitle":238,"officialTitle":239,"acronym":240,"eligibilityCriteria":241,"healthyVolunteers":12,"sex":17,"minAge":75,"maxAge":4,"enrollmentInfo":242,"targetDuration":4,"studyType":53,"phases":244,"briefSummary":246,"conditions":247,"keywords":248,"overallStatus":252,"whyStopped":4,"lastUpdateSubmitDate":253,"lastUpdatePostDateStruct":254,"startDateStruct":256,"completionDateStruct":258,"leadSponsor":260,"locationsCount":4},"100634640","phase-4-study-to-evaluate-the-safety-and-effectiveness-of-elevidys-in-participants-with-duchenne-muscular-dystrophy-treated-in-a-post-marketing-setting-100634640","NCT07542314","Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting","Phase 4 Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Patients With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting (ENHANCE)","ENHANCE","Key Inclusion Criteria:\n\n1. Cohort 1 only: Is male at birth, ambulatory, and ≥ 4 years of age at the time of dosing.\n2. Cohort 1 only: Is eligible for commercial ELEVIDYS.\n3. Cohort 2 only: Is male at birth and has previously received ELEVIDYS in a commercial setting after pre-treatment with sirolimus and corticosteroids.\n4. Cohort 1 only: Participants who are sexually active must agree to use, for the entire duration of the study, a condom and the female sexual partner must also use a medically acceptable form of birth control (eg, oral contraceptive).\n5. Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with the study visit schedule and all other protocol requirements, or is ≥ 18 years of age and personally able to understand and comply with the protocol requirements.\n6. Either has a parent or legal guardian who is willing to provide informed consent, or is ≥ 18 years of age and able to provide informed consent independently.\n\nKey Exclusion Criteria:\n\n1. Cohort 1 only: Contraindicated to receive ELEVIDYS per the United States Package Insert (USPI).\n2. Cohort 1 only: Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection.\n3. Has a medical condition or confounding circumstances (eg, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of the Investigator, might compromise:\n\n   1. The participant's ability to comply with the protocol-required procedures, and\u002For\n   2. The participant's well-being or safety, and\u002For\n   3. The clinical interpretability of the data collected from the participant\n4. Cohort 1 only: Has a symptomatic infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.\n5. Cohort 1 only: Has received a live virus vaccine within 4 weeks or inactive vaccine within 2 weeks of the Day 1 visit or expects to receive a vaccination during the first 3 months after Day 1.\n6. Cohort 1 only: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients.\n7. Cohort 1 only: Any wounds or recent injuries that, in the opinion of the Investigator, would be at risk of dehiscence or impaired healing in the setting of sirolimus administration.\n\nOther inclusion\u002Fexclusion criteria may apply, per protocol.",{"count":243,"type":22},20,[245],"PHASE4","The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.",[26],[26,28,249,250,251],"ELEVIDYS","Delandistrogene moxeparvovec-rokl","SRP-9001","NOT_YET_RECRUITING","2026-05-22",{"date":255,"type":34},"2026-05-26",{"date":257,"type":22},"2026-07-31",{"date":259,"type":22},"2027-03-31",{"name":127,"class":41},{"id":262,"slug":263,"hasResults":12,"nctId":264,"briefTitle":265,"officialTitle":266,"acronym":4,"eligibilityCriteria":267,"healthyVolunteers":268,"sex":17,"minAge":269,"maxAge":4,"enrollmentInfo":270,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":271,"conditions":272,"keywords":273,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":280,"lastUpdatePostDateStruct":281,"startDateStruct":282,"completionDateStruct":284,"leadSponsor":286,"locationsCount":42},"100525627","wearable-technology-to-evaluate-hyperglycemia-and-hrv-in-dmd-100525627","NCT06124196","Wearable Technology to Evaluate Hyperglycemia and HRV in DMD","Wearable Technology to Evaluate Hyperglycemia and Heart Rate Variability in Duchenne Muscular Dystrophy","CASE, DMD inclusion criteria:\n\n* Male\n* Age ≥10years\n* Clinical phenotype of DMD confirmed with muscle biopsy or genotype.\n* Informed consent for individuals ≥18 years\n* Parent\u002Fguardian informed consent and child assent for individuals \\\u003C 18 years\n\nCASE, DMD exclusion criteria:\n\n* Refusal to participate.\n* Diagnosis of diabetes prior to the study and\u002For taking insulin or other anti-diabetic drug therapy in \\\u003C 4 weeks prior to treatment\n* Use of a pacemaker, Implantable cardioverter-defibrillator (ICD), or other implanted device\n* Unable to comply with study procedures, in the opinion of the investigator.\n\nCONTROL inclusion criteria:\n\n* Male\n* Age ≥10years\n* Informed consent for individuals ≥18 years\n* Parent\u002Fguardian informed consent and child assent for individuals \\\u003C 18 years\n* BMI matched by Centers for Disease Control and Prevention (CDC) category (underweight, normal, overweight, obese) to cases.\n* Self-reported race\u002Fethnicity matched to cases.\n* No known evidence of diabetes, impaired fasting glucose, or impaired glucose tolerance:\n* For individuals (all ≥10 years) of age with obesity, we anticipate that they will have hemoglobin A1c (HbA1c) screening based on American Academy of Pediatrics (AAP) recommendations.\n* Participants will be included if they have a normal HbA1c (\\\u003C 5.7%) or if they have an elevated HbA1c (5.7-6.4%) with no evidence of impaired fasting glucose or impaired glucose tolerance on clinically obtained oral glucose tolerance tests (OGTT) (e.g., fasting glucose \\\u003C100mg\u002FdL and 2-hour glucose \\\u003C140mg\u002FdL).\n\nCONTROL, exclusion criteria:\n\n* Refusal to participate.\n* Diagnosis of diabetes prior to the study and\u002For taking insulin or other anti-diabetic drug therapy in \\\u003C 4 weeks prior to treatment\n* Use of a pacemaker, Implantable cardioverter-defibrillator (ICD), or other implanted device\n* Unable to comply with study procedures, in the opinion of the investigator.\n* Diagnosis of DMD or Becker muscular dystrophy",true,"10 Years",{"count":216,"type":22},"Duchenne muscular dystrophy (DMD) is an X-linked disorder that causes muscle wasting, cardiopulmonary failure, and premature death. Heart failure is a leading cause of death in DMD, but substantial knowledge gaps exist regarding predisposing risk factors. In the general population, hyperglycemia, insulin resistance, and decreased heart rate variability (HRV; reflecting autonomic dysfunction) are associated with cardiomyopathy (CM). It is unclear whether these factors are associated with DMD-CM. Closing this knowledge gap may lead to novel screening and therapeutic strategies to delay progression of DMD-CM, now the leading cause of death in patients with DMD. Despite risk factors for hyperglycemia, including the use of glucocorticoids (GCs), sarcopenia, obesity, and reduced ambulation, little is known regarding glucose abnormalities in DMD. Some of these same risk factors, along with the distance needed to travel for specialty care, present significant barriers to research participation and clinical care for individuals with DMD. Remote wearable technology may improve research participation in this vulnerable population. Therefore, this study will leverage remote wearable technologies to overcome these barriers and define the relationship between dysglycemia and DMD-CM.\n\nThe goal of this remote study is to evaluate rates of hyperglycemia in individuals with DMD compared to control participants using continuous glucose monitors, and to determine the relationship between hyperglycemia and heart rate variability. Participants will utilize continuous glucose monitors, cardiac monitors, and activity monitors to evaluate glucose levels, heart rate, activity, and sleep.",[26],[26,274,275,276,277,278,279],"heart failure","cardiomyopathy","hyperglycemia","heart rate variability","remote","controls","2026-05-19",{"date":253,"type":34},{"date":283,"type":34},"2024-03-20",{"date":285,"type":22},"2031-02",{"name":287,"class":288},"Vanderbilt University Medical Center","OTHER",{"id":290,"slug":291,"hasResults":12,"nctId":292,"briefTitle":293,"officialTitle":294,"acronym":4,"eligibilityCriteria":295,"healthyVolunteers":12,"sex":17,"minAge":296,"maxAge":4,"enrollmentInfo":297,"targetDuration":4,"studyType":53,"phases":299,"briefSummary":300,"conditions":301,"keywords":307,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":311,"lastUpdatePostDateStruct":312,"startDateStruct":314,"completionDateStruct":316,"leadSponsor":318,"locationsCount":42},"100538429","phase-2-vasodilator-and-exercise-study-for-dmd-vaso-rex-100538429","NCT06290713","Vasodilator and Exercise Study for DMD (VASO-REx)","Vasodilators and Exercise as Adjuvant Therapy for Duchenne Muscular Dystrophy (VASO-REx Study)","Inclusion Criteria:\n\n* Diagnosis of DMD confirmed by genetic report\n* Minimum entry age of 6.0 years old\n* Ambulatory\n* On stable glucocorticoid regimen (for \\> 3 months)\n\nExclusion Criteria:\n\n* Contraindication to a Magnetic resonance Imaging examination (e.g. severe claustrophobia, magnetic implants, unable\u002Funwilling to perform test)\n* Presence of unstable medical problems, including severe cardiomyopathy, left ventricular ejection fraction \\\u003C45%, cardiac conduction abnormalities as evidenced on ECG, uncontrolled seizure disorder, uncontrolled hypo or hypertension\n* Presence of a secondary condition that impacts muscle function or muscle metabolism (e.g., myasthenia gravis, endocrine disorder, mitochondrial disease)\n* Presence of a secondary condition leading to developmental delay or impaired motor control (e.g., cerebral palsy) or previous history of unprovoked rhabdomyolysis\n* Contraindications to phosphodiesterase 5 inhibitors (use of nitrates, alpha-adrenergic blockers, other phosphodiesterase 5 inhibitors) or other medications known to modulate blood flow or muscle metabolism\n* Participation in currently approved FDA trials or other investigational clinical trials during the period of the study","6 Years",{"count":298,"type":22},50,[55],"Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal muscle and improve cardiovascular health) and tadalafil, an FDA-approved vasodilator (to optimize blood flow and muscle perfusion which is impaired and often overlooked in DMD). Target: improved muscle function, vascular health, and DMD treatment.",[26,302,303,304,305,306,28],"Duchenne Disease","Muscular Dystrophy","Muscular Dystrophy in Children","Vasodilation","Exercise",[28,308,309,310],"Tadalafil","Drug and Exercise Intervention","Treatment Strategy","2026-05-12",{"date":313,"type":34},"2026-05-15",{"date":315,"type":34},"2024-06-05",{"date":317,"type":22},"2026-11",{"name":319,"class":288},"University of Florida",{"id":321,"slug":322,"hasResults":12,"nctId":323,"briefTitle":324,"officialTitle":325,"acronym":4,"eligibilityCriteria":326,"healthyVolunteers":12,"sex":327,"minAge":4,"maxAge":4,"enrollmentInfo":328,"targetDuration":330,"studyType":23,"phases":4,"briefSummary":331,"conditions":332,"keywords":337,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":339,"lastUpdatePostDateStruct":340,"startDateStruct":342,"completionDateStruct":344,"leadSponsor":346,"locationsCount":42},"100214523","the-duchenne-registry-100214523","NCT02069756","The Duchenne Registry","The Duchenne Registry: An International, Patient-Report Registry for Individuals With Duchenne and Becker Muscular Dystrophy (Member of TREAT-NMD Neuromuscular Network)","Inclusion Criteria:\n\n* Diagnosis of Duchenne or Becker muscular dystrophy; Manifesting female carriers and asymptomatic female carriers also included in registry.\n\nExclusion Criteria:\n\n* Diagnosis of any other type of muscular dystrophy (including limb-girdle muscular dystrophy).","ALL",{"count":329,"type":22},10000,"40 Years","The Duchenne Registry is an online, patient-report registry for individuals with Duchenne and Becker muscular dystrophy and carrier females. The purpose of the Registry is to connect Duchenne and Becker patients with actively recruiting clinical trials and research studies, and to educate patients and families about Duchenne and Becker care and research. At the same time, The Duchenne Registry is a valuable resource for clinicians and researchers in academia and industry, allowing access to de-identified datasets provided by patients and their families-information that is vital to advances in the care and treatment of Duchenne. The Duchenne Registry is a member of the TREAT-NMD Neuromuscular Network.",[26,333,334,335,336],"Becker Muscular Dystrophy","Dystrophinopathy","Dystrophinopathy Symptomatic Female Carrier","Dystrophinopathy Female Carrier",[29,338,303],"Becker","2026-05-05",{"date":341,"type":34},"2026-05-08",{"date":343,"type":34},"2007-10",{"date":345,"type":22},"2047-10",{"name":324,"class":288},{"id":348,"slug":349,"hasResults":12,"nctId":350,"briefTitle":351,"officialTitle":352,"acronym":4,"eligibilityCriteria":353,"healthyVolunteers":12,"sex":17,"minAge":75,"maxAge":354,"enrollmentInfo":355,"targetDuration":4,"studyType":53,"phases":356,"briefSummary":357,"conditions":358,"keywords":360,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":363,"lastUpdatePostDateStruct":364,"startDateStruct":366,"completionDateStruct":368,"leadSponsor":370,"locationsCount":7},"100515784","phase-2-ns-089ncnp-02-201-in-boys-with-duchenne-muscular-dystrophy-dmd-100515784","NCT05996003","NS-089\u002FNCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)","A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089\u002FNCNP-02 in Boys With Duchenne Muscular Dystrophy (DMD)","Inclusion Criteria:\n\n* Male ≥ 4 years and \\\u003C15 years of age\n* Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 44 to restore the dystrophin mRNA reading frame\n* Able to walk independently without assistive devices\n* Ability to complete the TTSTAND without assistance in \\\u003C20 seconds\n* Stable dose of glucocorticoid for at least 3 months and the dose is expected to remain on a stable dose for the duration of the study.\n* Other inclusion criteria may apply.\n\nExclusion Criteria:\n\n* Has a body weight of \\\u003C20 kg at the time of informed consent (applies to participants screening for Part 1 only)\n* Evidence of symptomatic cardiomyopathy\n* Current or previous treatment with anabolic steroids (e.g., oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug\n* Current or previous treatment with any other investigational drug within 3 months prior to the first dose of study drug or within 5 times the half-life of a medication, whichever is longer\n* Surgery within the 3 months prior to the first dose of study drug or planned during the study duration\n* Previously treated in an interventional study of NS-089\u002FNCNP-02\n* Having received exon skipping oligonucleotide within 1 year prior to the first dose of IP\n* Other exclusion criteria may apply.","14 Years",{"count":243,"type":22},[55],"This is a Phase 2, open-label, multi-center, 2-part study of NS-089\u002FNCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to \\\u003C15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089\u002FNCNP-02 administered once weekly.\n\nThe study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.",[26,359,28],"Exon 44",[361,28,362],"Exon 44 Skipping","Brogidirsen","2026-03-04",{"date":365,"type":34},"2026-03-06",{"date":367,"type":34},"2024-02-22",{"date":369,"type":22},"2026-09-11",{"name":371,"class":41},"NS Pharma, Inc.",{"id":373,"slug":374,"hasResults":12,"nctId":375,"briefTitle":376,"officialTitle":377,"acronym":4,"eligibilityCriteria":378,"healthyVolunteers":12,"sex":327,"minAge":379,"maxAge":4,"enrollmentInfo":380,"targetDuration":4,"studyType":53,"phases":382,"briefSummary":383,"conditions":384,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":385,"lastUpdatePostDateStruct":386,"startDateStruct":387,"completionDateStruct":389,"leadSponsor":391,"locationsCount":42},"100569298","phase-1-trial-of-cell-based-therapy-for-dmd-100569298","NCT06692426","Trial of Cell Based Therapy for DMD","Phase I Clinical Trial of Cell Based Therapy for Duchenne Muscular Dystrophy","Inclusion Criteria:\n\n* Duchenne muscular dystrophy, diagnosed by mutations in the DMD (dystrophin) gene and\u002For absence of immunohistochemical staining for dystrophin on muscle biopsy\n* Non-ambulatory\n* Intact extensor digitorum brevis (EDB) muscles bilaterally\n* Off investigational therapies for \\> 30 days\n* Age 18 years of age or older at the time of consent\n* Have adequate organ function confirmed by the following laboratory values obtained within 14 days prior to enrollment (28 days for cardiac and pulmonary function):\n* Participants with partners of childbearing potential must be willing to use at least two forms of effective birth control (one form must be a barrier method) while receiving the study product and for 3 months after stopping tacrolimus therapy.\n* Ability to follow commands sufficiently to perform voluntary aspects of outcome measures throughout the study period\n* Willing to consent to monitoring for 15 years, including an extension period, as required for all interventional studies involving the transplantation of cells that have been genetically modified\n* Voluntary written consent from the subject or parent(s)\u002Fguardian(s) and assent from participant prior to the performance of any research related activity.\n\nExclusion Criteria:\n\n* Presence of HLA antibodies directed toward HLA antigens on MyoPAXon\n* Active treatment with another investigational therapy\n* Known allergy to MyoPAXon components","18 Years",{"count":381,"type":22},8,[80],"This is a single-center, single-arm, interventional phase 1 trial to evaluate the safety and tolerability of local injection of induced pluripotent stem cell (iPSC)- derived CD54+ allogeneic muscle progenitor cells in individuals with Duchenne muscular dystrophy (DMD)",[26],"2026-03-02",{"date":363,"type":34},{"date":388,"type":34},"2025-03-20",{"date":390,"type":22},"2027-03-03",{"name":392,"class":288},"Masonic Cancer Center, University of Minnesota",{"id":394,"slug":395,"hasResults":12,"nctId":396,"briefTitle":397,"officialTitle":398,"acronym":399,"eligibilityCriteria":400,"healthyVolunteers":12,"sex":17,"minAge":107,"maxAge":4,"enrollmentInfo":401,"targetDuration":403,"studyType":23,"phases":4,"briefSummary":404,"conditions":405,"keywords":406,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":410,"lastUpdatePostDateStruct":411,"startDateStruct":413,"completionDateStruct":415,"leadSponsor":417,"locationsCount":419},"100559503","registry-study-to-observe-long-term-safety-of-vamorolone-agamree-in-patients-with-duchenne-muscular-dystrophy-summit-100559503","NCT06564974","Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT","Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy [SUpplemental Patient dMd assessMents Investigating ouTcomes (SUMMIT)]","DMD-001 SUMMIT","Inclusion Criteria:\n\n1. Patient or parent\u002Flegal guardian is willing and able to provide written informed consent once the nature of the registry has been explained and prior to the start of any registry-related procedures.\n2. Patient and\u002For parent\u002Fguardian are willing and able to complete QoL questionnaires.\n3. Male patients at least 2 years old.\n4. Confirmed diagnosis of DMD (via genetic testing or muscle biopsy with absent dystrophin staining to anti- dystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).\n5. Patient has a current, active prescription for, or is on, AGAMREE®.\n\nExclusion Criteria:\n\n1\\. Any contraindication to AGAMREE® or medical condition, which, in the opinion of the Investigator, would affect registry participation, performance, or interpretation of registry assessments.",{"count":402,"type":22},250,"5 Years","The goal of this study is to collect additional information on the safety of long-term treatment with AGAMREE® and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires) in male patients aged 2 years and older with Duchenne muscular dystrophy (DMD).",[26],[26,407,408,409,28,303],"Neuromuscular","AGAMREE®","Vamorolone","2026-02-23",{"date":412,"type":34},"2026-02-27",{"date":414,"type":34},"2024-09-25",{"date":416,"type":22},"2032-02",{"name":418,"class":41},"Catalyst Pharmaceuticals, Inc.",27,{"id":421,"slug":422,"hasResults":12,"nctId":423,"briefTitle":424,"officialTitle":425,"acronym":4,"eligibilityCriteria":426,"healthyVolunteers":12,"sex":17,"minAge":75,"maxAge":76,"enrollmentInfo":427,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":429,"conditions":430,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":431,"lastUpdatePostDateStruct":432,"startDateStruct":434,"completionDateStruct":436,"leadSponsor":438,"locationsCount":152},"100353510","natural-history-of-duchenne-muscular-dystrophy-100353510","NCT03882827","Natural History of Duchenne Muscular Dystrophy","A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 9 Years","Inclusion Criteria:\n\n1. Male\n2. 4 to 9 years old inclusive\n3. Body-weight ≤ 95th percentile or the BMI scale ≤ 95th percentile (according to validated scale in force in country site).\n\n   Related to the DMD disease:\n4. Diagnosis of DMD based upon documented gene testing with detailed genotyping\n5. Able to achieve at inclusion and screening visits:\n\n   1. NSAA (North Star Ambulatory Assessment) scale \\> 18 or ≥ 16 if participant is between 4 and \\\u003C 5 years old at screening and:\n   2. Gowers test \\\u003C or = 7 sec and\u002For\n   3. 6-Minute Walk Test (6MWT): a distance ≥ 350 meters at inclusion visit (M0)\n6. Ongoing corticosteroid therapy or initiation of corticosteroid therapy according to standard of care prior to Screening visit\n\n   Related to the study protocol and ICH\u002FGCP (Good Clinical Practice) requirements:\n7. Signed informed consent by at least one parent or both parents or legal guardian representative(s), when applicable and according to the country regulation\n8. Affiliated to or a beneficiary of a Health Care scheme (according to country regulation)\n\n   Exclusion Criteria:\n\n   Subject will be excluded from enrolment into the study for any of the following reasons:\n\n   Related to the DMD disease severity:\n9. Cardiomyopathy based on physical\u002Fcardiological examination and echocardiography with Left Ventricular Simpson biplane Ejection Fraction (LVEF) below 55%\n10. Respiratory Assistance: need for either a diurnal and\u002For a nocturnal ventilation\n11. Any co-morbidity (ies) and or previous or planned surgical event(s) which may interfere with DMD natural evolution and or evaluation of outcomes designed to assess DMD Natural History\n\n    Related to specific assessments:\n12. Muscle testing: inability to cooperate with\n13. MRI: metal implants in regions of interest for the study\n\n    Related to the study protocol and ICH\u002FGCP requirements:\n14. Unwilling and\u002For unable to comply with all the study protocol requirements and\u002For procedures\n15. Previous inclusion to another clinical trial with an Investigational Medicinal Product (IMP), within the 3 months or IMP washout period (whichever is longer) prior to the screening visit of the study\n16. Previously treated with a gene therapy drug for DMD, such as:\n\n    * any AAV mediated gene transfer products or any gene editing products in a clinical trial or in a clinical setting,\n    * if exons skipping drug was used, the last dose of exon skipping drug within 5 half-lives prior to the screening visit\n17. Concomitant participation to any other interventional clinical trial",{"count":428,"type":22},220,"Baseline Study on Duchenne Muscular Dystrophy (DMD) in view to collect data on the natural disease course in a cohort in young male subjects aged from 4 to 9 Years over a period of 6 to 36 months using disease appropriate evaluations.",[26],"2026-02-10",{"date":433,"type":34},"2026-02-12",{"date":435,"type":34},"2019-12-19",{"date":437,"type":22},"2029-09-30",{"name":439,"class":288},"Genethon",{"id":441,"slug":442,"hasResults":12,"nctId":443,"briefTitle":444,"officialTitle":444,"acronym":28,"eligibilityCriteria":445,"healthyVolunteers":12,"sex":17,"minAge":446,"maxAge":379,"enrollmentInfo":447,"targetDuration":4,"studyType":53,"phases":448,"briefSummary":450,"conditions":451,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":454,"lastUpdatePostDateStruct":455,"startDateStruct":457,"completionDateStruct":459,"leadSponsor":461,"locationsCount":42},"100547051","gamified-occupational-therapy-for-adolescents-with-duchenne-muscular-dystrophy-100547051","NCT06402942","Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy","Inclusion Criteria:\n\n* Being diagnosed with Duchenne muscular dystrophy\n* To be literate in Turkish\n* To have scored 27 points or more on the Modified Mini Mental Test\n* Having a computer, tablet and internet connection\n* Volunteering to participate in the study by their parents and reading and signing the informed consent form\n\nExclusion Criteria:\n\n* Having a neurological disease other than Duchenne muscular dystrophy and\u002For another diagnosed neurological disease accompanying Duchenne muscular dystrophy\n* Having a cooperation problem that prevents completing the assessments for any reason\n* Difficulty understanding and speaking the Turkish language","13 Years",{"count":243,"type":22},[449],"NA","This research aims to improve the quality of life, occupational performance, occupational satisfaction and emotional health of young people with Duchenne muscular dystrophy compared to the classical occupational therapy program. The findings are planned to shed light on the development of new and effective strategies in the rehabilitation of adolescents with Duchenne muscular dystrophy.",[26,452,453],"Gamification","Occupational Therapy","2026-01-24",{"date":456,"type":34},"2026-01-27",{"date":458,"type":34},"2025-05-01",{"date":460,"type":22},"2026-12-08",{"name":462,"class":463},"Başak Çağla Arslan","OTHER_GOV",{"id":465,"slug":466,"hasResults":12,"nctId":467,"briefTitle":468,"officialTitle":469,"acronym":4,"eligibilityCriteria":470,"healthyVolunteers":12,"sex":17,"minAge":161,"maxAge":4,"enrollmentInfo":471,"targetDuration":4,"studyType":53,"phases":473,"briefSummary":474,"conditions":475,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":476,"lastUpdatePostDateStruct":477,"startDateStruct":479,"completionDateStruct":481,"leadSponsor":483,"locationsCount":485},"100314473","phase-2-givinostat-in-duchennes-muscular-dystrophy-long-term-safety-and-tolerability-study-100314473","NCT03373968","Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study","Open Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT Studies","Inclusion Criteria:\n\n1. Must have participated in one of the previous studies with GIVINOSTAT in DMD and have attended the End of Study Visit or must have been screened in study DSC\u002F14\u002F2357\u002F48 and met:\n\n   * all the inclusion criteria and none of the exclusion criteria,\n   * had a baseline vastus lateralis muscle fat fraction (VL MFF) assessed by MRS in the range ≤5% or \\>30%, i.e. included in\"off-target\" group,\n   * never been randomized because, the enrollment in the off target group was completed.\n2. Aged ≥6 years old;\n3. Are able to give informed assent and\u002For consent in writing signed by the subject and\u002For parent\u002Flegal guardian (according to localregulations);\n4. Subjects must be willing to use adequate contraception:\n\n   * Contraceptive methods must since the previous GIVINOSTAT study through 3 months after the last dose of study drug, and include the following:\n\n     * True abstinence (absence of any sexual intercourse), when in line with the preferred and usual lifestyle of the subject.\n     * Periodic abstinence (e.g. calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception.\n     * Condom with spermicide and the female partner must use an acceptable method of contraception, such as an oral,\n     * transdermal, injectable or implanted steroid-basedcontraceptive, or a diaphragm or a barrier method of contraception in conjunction with spermicidal jelly such asfor example cervical cap with spermicide jelly.\n\nExclusion Criteria:\n\n1. Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to be enrolled in this study (e.g., growth hormone); Vitamin D, calcium, and any other supplements will be allowed;\n2. Use of any current investigational drug other than Givinostat;\n3. Have presence of other clinically significant disease, which, in the Investigator's opinion, could adversely affect the safety of the subject, making it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results;\n4. Have a diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD;\n5. Have platelets count, White Blood Cell and Hemoglobin at screening \\\u003C Lower Limit of Normal (LLN)\\* (for abnormal screening laboratory test results (\\\u003CLLN), the platelets count, White Blood Cell and Hemoglobin will be repeated once; if the repeat test result is still \\\u003CLLN, then exclusionary);\n6. Have Triglycerides \\> 300 mg\u002FdL (3.42 mmol\u002FL) in fasting condition at screening visit\\* (for abnormal screening laboratory test results (\\>300 mg\u002FdL), the triglycerides will be repeated once; if the repeat test result is still \\>300 mg\u002FdL, then exclusionary);\n7. Have inadequate renal function, as defined by serum Cystatin C \\>2 x the upper limit of normal (ULN) at screening visit\\*. If the value is \\>2 x ULN, the serum Cystatin C will be repeated once; if the repeated test result is still \\>2 x ULN, the subject should be excluded);\n8. Have heart failure (New York Heart Association Class III or IV)\n9. Have a current liver disease or impairment, including but not limited to an elevated total bilirubin\\* (i.e. \\> 1.5 x ULN), unless secondary to Gilbert disease or pattern consistent with Gilbert's;\n10. Have a baseline QTcF \\>450 msec, (as the mean of 3 consecutive readings 5 minutes apart) or history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, or family history of long QT syndrome);\n11. Have a psychiatric illness\u002Fsocial situation rendering the potential subject unable to understand and comply with the muscle function tests and\u002For with the study protocol procedures.\n12. Have any hypersensitivity to the components of study medication;\n13. Have a sorbitol intolerance or sorbitol malabsorption or have the hereditary form of fructose intolerance.\n\n    * the Investigators to evaluate these exclusion criteria can use the laboratory results obtained within 5 months from V1, to allow the continuity of the treatment. It is worth noting, as soon as the site will receive the laboratory results done in screening\u002Fbaseline (Visit 1) visit they will check the GIVINOSTAT dose and modify it as per protocol safety rules and\u002For dosage modifications rules.",{"count":472,"type":22},206,[55,56],"This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.",[26],"2026-01-19",{"date":478,"type":34},"2026-01-21",{"date":480,"type":34},"2017-10-24",{"date":482,"type":22},"2029-12",{"name":484,"class":41},"Italfarmaco",39,{"id":487,"slug":488,"hasResults":12,"nctId":489,"briefTitle":490,"officialTitle":491,"acronym":4,"eligibilityCriteria":492,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":493,"phases":4,"briefSummary":494,"conditions":495,"keywords":496,"overallStatus":498,"whyStopped":4,"lastUpdateSubmitDate":499,"lastUpdatePostDateStruct":500,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":502,"locationsCount":193},"100351996","expanded-access-protocol-for-boys-with-duchenne-muscular-dystrophy-100351996","NCT03863119","Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy","An Open-Label, Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy Who Have Completed the Long-Term Extension (VBP15-LTE) or VBP15-004 or VBP15-006 Studies","Inclusion Criteria:\n\n* Subject's parent or legal guardian has provided written informed consent\u002FHIPAA authorization\n* Subject has previously completed at a participating US or Canada study site VBP15-LTE up to and including the Month 24 assessments, OR VBP15-004 up to and including the Week 48 assessments, VBP15-006 up to and including the Week 12 assessment\n* Subject and parent\u002Fguardian are willing and able to comply with recommended study drug administration plan, and standard of care follow-up and monitoring as recommended by their Treating Physician\n\nExclusion Criteria:\n\n* Subject had a serious or severe adverse event in study VBP15-LTE or VBP15-004 or VBP15-006 that, in the opinion of the Treating Physician and Sponsor, was probably or definitely related to vamorolone use and precludes safe use of vamorolone for the subject in this expanded access program\n* Subject and\u002For parent\u002Fguardian are unable and\u002For unwilling to comply with regular medical care and follow-up as recommended by their Treating Physician throughout participation in the VBP15-EAP","EXPANDED_ACCESS","The intent of this protocol is to provide continued access to vamorolone for subjects in the United States and Canada who have completed the VBP15-LTE, VBP15- 004, or VBP15-006 protocols (and are thereby ineligible to enroll in another trial of vamorolone therapy), during the time a new drug application for vamorolone is under preparation and review.",[26],[26,409,497,28],"VBP-15","AVAILABLE","2026-01-16",{"date":501,"type":34},"2026-01-20",{"name":503,"class":41},"Santhera Pharmaceuticals",{"id":505,"slug":506,"hasResults":12,"nctId":507,"briefTitle":508,"officialTitle":509,"acronym":4,"eligibilityCriteria":510,"healthyVolunteers":12,"sex":17,"minAge":75,"maxAge":379,"enrollmentInfo":511,"targetDuration":4,"studyType":53,"phases":513,"briefSummary":514,"conditions":515,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":178,"lastUpdatePostDateStruct":516,"startDateStruct":518,"completionDateStruct":520,"leadSponsor":522,"locationsCount":233},"100432080","phase-1-open-label-study-of-wve-n531-in-patients-with-duchenne-muscular-dystrophy-forward-53-100432080","NCT04906460","Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)","An Open-label Phase 1b\u002F2 Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy","Inclusion Criteria:\n\nPart A and Part B:\n\n1. Part A patients may be screened for Part B upon completion of a washout period of ≥18 weeks from last dose in Part A. New patients may also be screened for Part B\n2. Diagnosis of DMD based on clinical phenotype.\n3. Documented mutation in the DMD gene associated with DMD that is amenable to exon 53 intervention\n4. Score of ≥1 on item 1 or 2 of the shoulder component of the Performance of the Upper Limb (PUL) (Part B ).\n5. Ambulatory or non-ambulatory male\n6. Stable pulmonary and cardiac function, as measured by the following: (Part B):\n\n1\\. Reproducible percent predicted forced vital capacity (FVC) ≥50%; 2. Left ventricular ejection fraction (LVEF) \\>55% in patients \\\u003C10 years of age and \\>45% in patients ≥10 years of age, as measured (and documented) by echocardiogram (ECHO) and\u002For cardiac magnetic resonance imaging (MRI), within 6 months prior to enrollment into the study.\n\n7.Adequate muscle at Screening to perform open muscle biopsies, preferably deltoid.\n\n8\\. Currently on a stable corticosteroid therapy regimen, defined as initiation of systemic corticosteroid therapy that occurred ≥6 months prior to Screening and no changes in dose ≤3 months prior to Screening visit (Part B ).\n\nPart C\n\n1. New patients to be screened for Part C.\n2. Diagnosis of DMD based on clinical phenotype.\n3. Documented mutation in the DMD gene associated with DMD that is amenable to exon 53 intervention\n4. Score of ≥1 on item 1 or 2 of the shoulder component of the Performance of the Upper Limb (PUL) .\n5. Ambulatory male\n6. Stable pulmonary and cardiac function, as measured by the following:\n\n1\\. Reproducible percent predicted forced vital capacity (FVC) ≥50%; 2. Left ventricular ejection fraction (LVEF) \\>55% in patients as measured (and documented) by echocardiogram (ECHO) and\u002For cardiac magnetic resonance imaging (MRI), within 6 months prior to enrollment into the study.\n\n7\\. Adequate muscle at Screening to perform open muscle biopsies, preferably deltoid.\n\n8\\. Currently on a stable corticosteroid therapy regimen, defined as initiation of systemic corticosteroid therapy that occurred ≥6 months prior to Screening and no changes in dose ≤3 months prior to Screening visit .\n\nExclusion Criteria:\n\n1. Clinically significant medical finding on the physical examination other than DMD that, in the judgment of the Investigator, will make the patient unsuitable for participation in, and\u002For completion of the study procedures.\n2. Part B and Part C: Major surgery within 3 months prior to Day 1 or planned major surgery for any time during the study.\n3. Part B: Diagnosis of active alcohol, cannabinoid, or other substance use disorder (except nicotine) within 6 months prior to the Screening visit\n4. Part C: Any recreational substance use (including prescribed cannabinoids), with the exception of nicotine, irrespective of legality, within 2 months prior to Screening and\u002For unwilling to refrain from such use for the duration of the study.",{"count":512,"type":22},26,[80,55],"This is a Phase 1b\u002F2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD). To participate in the study, patients must have a documented mutation of the DMD gene that is amenable to exon 53 skipping intervention. This study has 3 parts, Part A, Part B, including Part B Extension Arm, and Part C. Part A is completed. Part B is completed. Following completion of Part B, all patients elected to continue to receive study drug in the optional Part B open-label Extension Arm. Part C has been added to the study and will enroll new patients.",[26],{"date":517,"type":34},"2025-12-15",{"date":519,"type":34},"2021-09-28",{"date":521,"type":22},"2027-04-24",{"name":523,"class":41},"Wave Life Sciences USA, Inc.",{"id":525,"slug":526,"hasResults":12,"nctId":527,"briefTitle":528,"officialTitle":528,"acronym":4,"eligibilityCriteria":529,"healthyVolunteers":268,"sex":327,"minAge":403,"maxAge":4,"enrollmentInfo":530,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":532,"conditions":533,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":536,"lastUpdatePostDateStruct":537,"startDateStruct":539,"completionDateStruct":541,"leadSponsor":543,"locationsCount":233},"100440770","biomarker-development-for-muscular-dystrophies-100440770","NCT05019625","Biomarker Development for Muscular Dystrophies","Inclusion Criteria:\n\n* Subjects with DM1 or DM2 based on genetic testing and\u002For clinical criteria (some subjects who have positive genetic testing may be asymptomatic, while other subjects who show characteristic clinical features may have declined to have genetic testing done). Control non-DM subjects are unknown to have DM or any other muscular dystrophy by history and may have had no genetic testing.\n* Able to provide informed consent or assent for participation in the study.\n* Demographic characteristics for single biofluid collection: Males and females age 5 years and older.\n* Demographic characteristics for serial biofluid and muscle function testing: Males and females age 14 years and older with DM1.\n* Demographic characteristics for biofluid and muscle biopsy: Males and females, ages 18-65 years.\n\nDemographic characteristics for single biofluid collection, ultrasound, and myography: Males and females age 14 years and older.\n\nExclusion Criteria:\n\n* Medical history of any of the following. State of immunosuppression; coagulopathy; pre-existing liver or kidney disease; documented HIV positive; documented hepatitis B and\u002For C positive.\n* Medications and other drugs. Use of anti-platelet drugs within 7 days prior to blood draw or biopsy; use of anticoagulants within 60 days prior to blood draw or biopsy; active drug or alcohol use or dependence that, in the opinion of the biopsy surgeon, would interfere with post-procedure wound care.\n* Other. Inability or unwillingness of the subject to give written informed consent.",{"count":531,"type":22},465,"Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. The investigators are interested in finding less invasive methods that reduce the need for muscle biopsies. The purpose of this research is to learn about the possibility of detecting and measuring the activity and severity of muscular dystrophies by examining a urine sample and a blood sample, and some muscles in the arms and legs using tests called ultrasound and electrical impedance myography; both tests are painless and non-invasive. The information that is gathered from this study may help to evaluate, prevent, diagnose, treat, and improve the understanding of human muscle diseases.",[534,26,333,535],"Myotonic Dystrophy","Facioscapulohumeral Muscular Dystrophy","2025-11-19",{"date":538,"type":34},"2025-11-24",{"date":540,"type":34},"2015-02-20",{"date":542,"type":22},"2028-06",{"name":544,"class":288},"Massachusetts General Hospital",{"id":546,"slug":547,"hasResults":12,"nctId":548,"briefTitle":549,"officialTitle":549,"acronym":4,"eligibilityCriteria":550,"healthyVolunteers":12,"sex":327,"minAge":403,"maxAge":4,"enrollmentInfo":551,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":553,"conditions":554,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":536,"lastUpdatePostDateStruct":555,"startDateStruct":556,"completionDateStruct":558,"leadSponsor":560,"locationsCount":561},"100440562","extracellular-rna-biomarkers-of-duchenne-muscular-dystrophy-100440562","NCT05016908","Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy","Inclusion Criteria:\n\n* Subjects with DMD or BMD based on genetic testing. Control subjects are unknown to have any other muscular dystrophy by history and may have had no genetic testing.\n* Able to provide informed consent or assent for participation in the study.\n* Demographic characteristics for biofluid collection: Males age 5 years and older with DMD or BMD; males and females ages 18 years and older without muscular dystrophy.\n\nExclusion Criteria:\n\n* Medical history of any of the following: State of immunosuppression; coagulopathy; pre-existing liver or kidney disease; documented HIV positive; documented hepatitis B and\u002For C positive.\n* Use of anti-platelet drugs within 7 days prior to blood draw; use of anticoagulants within 60 days prior to blood draw.\n* Inability or unwillingness of the subject to give written informed consent.",{"count":552,"type":22},100,"Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. The investigators are interested in finding less invasive methods that reduce the need for muscle biopsies. The purpose of this research is to learn about the possibility of detecting and measuring the activity and severity of muscular dystrophies by examining a urine sample and a blood sample.",[26],{"date":538,"type":34},{"date":557,"type":34},"2019-11-30",{"date":559,"type":22},"2027-11",{"name":544,"class":288},2,{"id":563,"slug":564,"hasResults":12,"nctId":565,"briefTitle":566,"officialTitle":266,"acronym":4,"eligibilityCriteria":567,"healthyVolunteers":12,"sex":17,"minAge":269,"maxAge":4,"enrollmentInfo":568,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":569,"conditions":570,"keywords":571,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":572,"lastUpdatePostDateStruct":573,"startDateStruct":575,"completionDateStruct":577,"leadSponsor":579,"locationsCount":42},"100523244","wearable-technology-to-evaluate-hyperglycemia-and-hrv-in-dmd---longitudinal-aim-100523244","NCT06093100","Wearable Technology to Evaluate Hyperglycemia and HRV in DMD - Longitudinal Aim","Inclusion and Exclusion Criteria:\n\nInclusion criteria\n\n* Male- ≥10 years\n* Clinical phenotype of DMD confirmed with muscle biopsy or genotype.\n* Informed consent for individuals ≥18 years\n* Parent\u002Fguardian informed consent and child assent for individuals \\\u003C 18 years\n* Able to undergo non-sedated CMR\n\nExclusion Criteria\n\n* Refusal to participate\n* Diagnosis of diabetes prior to the study and\u002For taking insulin or other anti-diabetic drug therapy in \\\u003C 4 weeks prior to treatment\n* Inability to fast for 10 hours\n* Use of a pacemaker, implantable cardioverter-defibrillator (ICD), or other implanted device\n* Unable to comply with study procedures, in the opinion of the investigator.",{"count":206,"type":22},"Duchenne Muscular Dystrophy (DMD) is an X-linked disorder that causes muscle wasting, cardiopulmonary failure, and premature death. Heart failure is a leading cause of death in DMD, but substantial knowledge gaps exist regarding predisposing risk factors. In the general population, hyperglycemia, insulin resistance, and decreased heart rate variability (HRV; reflecting autonomic dysfunction) are associated with cardiomyopathy (CM). It is unclear whether these factors are associated with DMD-CM. Closing this knowledge gap may lead to novel screening and therapeutic strategies to delay progression of DMD related CM. Despite risk factors for hyperglycemia, including the use of glucocorticoids, low muscle mass, obesity, and reduced ambulation, little is known regarding glucose abnormalities in DMD. Some of these same risk factors, along with the distance needed to travel for specialty care, present significant barriers to research participation and clinical care for individuals with DMD. Remote wearable technology may improve research participation in this vulnerable population. Therefore, this study will leverage remote wearable technologies to overcome these barriers and define the relationship between dysglycemia and DMD-CM.\n\nIn this Aim of the study, the investigators will assess the utility of remote wearable technology to predict changes in traditional metrics of metabolism and cardiac function. In this pilot study, 10 individuals with DMD will undergo cardiac magnetic resonance imaging (CMR) and oral glucose tolerance tests (OGTTs) at baseline and two years. The investigators will remotely assess glycemia (using continuous glucose monitors), HRV (using extended Holter monitors), and activity (using accelerometers) every 6 months over the 2 years and evaluate if changes in wearable metrics predict changes in CMR and OGTT.",[26],[26,274,275,276,277],"2025-11-04",{"date":574,"type":34},"2025-11-06",{"date":576,"type":34},"2024-07-10",{"date":578,"type":22},"2030-12",{"name":287,"class":288},{"id":581,"slug":582,"hasResults":12,"nctId":583,"briefTitle":584,"officialTitle":584,"acronym":4,"eligibilityCriteria":585,"healthyVolunteers":268,"sex":17,"minAge":403,"maxAge":586,"enrollmentInfo":587,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":589,"conditions":590,"keywords":591,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":595,"lastUpdatePostDateStruct":596,"startDateStruct":598,"completionDateStruct":600,"leadSponsor":602,"locationsCount":603},"100169857","magnetic-resonance-imaging-and-biomarkers-for-muscular-dystrophy-100169857","NCT01484678","Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy","Inclusion Criteria for boys with DMD:\n\n1\\. Ambulatory and non-ambulatory males (ages 5-30 at baseline testing) previously diagnosed with DMD based on:\n\n* clinical features with onset of symptoms before age five\n* elevated serum creatine kinase level or\n* absence of dystrophin expression, as determined by immunostain or western blot (\\\u003C2%) and\u002For DNA confirmation of a dystrophin mutation \\*Subjects will not be excluded based on corticosteroid treatment or other clinical trials\n\nInclusion Criteria for adults with Becker MD:\n\n1. Ambulatory males (ages 18-62) without disease or injury to the lower extremities\n2. Specific recruitment of a subset of individuals with deletion mutations in the dystrophin gene involving either exon 51 or exon 45.\n\nInclusion Criteria for age matched controls for Becker MD subjects:\n\n1\\. Ambulatory males (ages 18-62) without disease or injury to the lower and\u002For upper extremities will be eligible to participate in this study\n\nExclusion Criteria:\n\n1. Males with a contraindication to an MR examination\n2. Males with unstable medical problems\n3. Males who are not able to cooperate during testing\n4. Males with a secondary condition that may impact muscle metabolism, muscle function or functional ability (i.e. cerebral palsy, endocrine disorders, mitochondrial disease)\n5. Daytime ventilation\n6. Implantable Cardioverter Defibrillator- (ICD) or pace maker\n7. Healthy boys\u002Fmen who participate in competitive sports specific training in excess of 8 hours per week","62 Years",{"count":588,"type":22},550,"The purpose of this research study is to determine the potential of magnetic resonance imaging, spectroscopy, and whole body imaging to monitor disease progression and to serve as an objective outcome measure for clinical trials in Muscular Dystrophy (MD).\n\nThe investigators will compare the muscles of ambulatory or non-ambulatory boys\u002Fmen with DMD with muscles of healthy individuals of the same age and monitor disease progression in those with DMD over a 5-10 year period. The amount of muscle damage and fat that the investigators measure will also be related to performance in daily activities, such as walking and the loss of muscle strength. In a small group of subjects the investigators will also assess the effect of corticosteroid drugs on the muscle measurements.\n\nAdditionally, the investigators will map the progression of Becker MD following adults with this rare disease. The primary objective is to conduct a multi-centered study to validate the potential of non-invasive magnetic resonance imaging and magnetic resonance spectroscopy to monitor disease progression and to serve as a noninvasive surrogate outcome measure for clinical trials in DMD and BMD. The secondary objective is to characterize the progressive involvement of the lower extremity, upper extremity, trunk\u002Frespiratory muscles in boys\u002Fmen with DMD and BMD guiding clinical trials.",[26,333],[26,333,592,593,594],"Magnetic Resonance Spectroscopy","Magnetic Resonance Imaging","Muscle","2025-10-13",{"date":597,"type":34},"2025-10-15",{"date":599,"type":34},"2020-09-01",{"date":601,"type":22},"2026-08-31",{"name":319,"class":288},3,{"id":605,"slug":606,"hasResults":12,"nctId":607,"briefTitle":608,"officialTitle":609,"acronym":4,"eligibilityCriteria":610,"healthyVolunteers":12,"sex":17,"minAge":611,"maxAge":612,"enrollmentInfo":613,"targetDuration":4,"studyType":53,"phases":614,"briefSummary":615,"conditions":616,"keywords":617,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":619,"lastUpdatePostDateStruct":620,"startDateStruct":622,"completionDateStruct":624,"leadSponsor":626,"locationsCount":628},"100470940","phase-4-once-weekly-infant-corticosteroid-trial-for-dmd-100470940","NCT05412394","Once Weekly Infant Corticosteroid Trial for DMD","Phase-2 Trial of 5mg\u002Fkg\u002FWeek Prednisolone in Young Boys With DMD","Inclusion Criteria:\n\n* Subjects ages 1 month through 30 months\n* Weakness consistent with Duchenne on exam, creatine kinase ≥ 20 times the upper limit of normal, and genetic mutation known to be causative for DMD.\n\nExclusion Criteria:\n\n* Prior treatment with Glucocorticosteroids","1 Month","30 Months",{"count":512,"type":22},[245],"The hypothesis tested here is that a lower dose of intermittent oral corticosteroids (5mg\u002Fkg\u002Fweek) will be equally effective to the 10mg\u002Fkg\u002Fweek dose.",[26],[26,28,618,303],"Steroid","2025-08-08",{"date":621,"type":34},"2025-08-13",{"date":623,"type":34},"2021-04-30",{"date":625,"type":22},"2026-12",{"name":627,"class":288},"Anne M. Connolly",4]