[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Solid Biosciences Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":133},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,54,80,108],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":29,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":42,"lastUpdatePostDateStruct":43,"startDateStruct":46,"completionDateStruct":48,"leadSponsor":50,"locationsCount":53},"100606807","phase-1-a-study-of-sgt-212-gene-therapy-in-friedreichs-ataxia-100606807",false,"NCT07180355","A Study of SGT-212 Gene Therapy in Friedreich's Ataxia","A Phase 1b First-in-Human, Open-Label, Dose-Finding Trial to Evaluate the Safety and Tolerability of SGT-212 Delivered Via Dual Intradentate Nucleus (IDN) and Intravenous (IV) Administration to Participants With Friedreich's Ataxia (FA)","FALCON","Inclusion Criteria:\n\n* Has history of FA symptom onset ≤25 years of age\n* Has a clinical and genetic diagnosis of FA\n* Has a staging score of ≥1 but \\\u003C6 on the Friedreich's Ataxia Rating Scale (FARS) Functional Disability Staging Score\n* Is willing to agree to the following rules for use of omaveloxolone (Skyclarys):\n\n  1. For a candidate who is currently taking omaveloxolone, has been on a stable dose for 12 weeks, expects to continue taking omaveloxolone at that dose throughout the study, and is willing to stop taking omaveloxolone at the direction of the Investigator or Sponsor's Medical Monitor if evidence of transaminitis or synthetic liver dysfunction is detected during the study\n  2. For a candidate who is not actively taking omaveloxolone, at least 12 weeks have passed since the last dose and the candidate agrees not to resume omaveloxolone during the 18-month period after SGT-212 infusion NOTE: The use of any other approved or investigational medicinal product for the treatment of FA should be discussed with the study team.\n\nExclusion Criteria:\n\n* Antibodies against adeno-associated virus serotype 9 (AAV9)\n* Has a modified FARS (mFARS) score \\\u003C20\n* Has a body weight ≤25 kilogram (kg) or has body mass index (BMI) ≥33 kg\u002Fm\\^2\n* Has a contraindication to endomyocardial biopsy (EMB) or cardiac catheterization\n* Is unable to undergo cardiac and brain MRI with contrast, including hypersensitivity to gadolinium contrast agent, presence of a non-MRI-compatible cardiac pacemaker, presence of a non-MRI-compatible implantable cardiac defibrillator, or physical condition (e.g., contractures)\n* Has uncontrolled diabetes as defined by a hemoglobin (Hb) A1c \\>9%\n* Has participated in recent interventional clinical studies or received any investigational therapy administered within 3 months or 5 half-lives (whichever is longer) prior to Screening\n* Has received gene therapy at any time\n* Has contraindications to receiving corticosteroids\n* Has any contraindication to the surgical procedures involved with IDN infusion of SGT-212\n* Has any known cardiac disease not related to FA including known obstructive coronary artery disease (CAD)\n* Other Inclusion\u002FExclusion criteria to be applied as per protocol.","ALL","18 Years","40 Years",{"count":21,"type":22},10,"ESTIMATED","INTERVENTIONAL",[25],"PHASE1","This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA). It will be delivered via dual intradentate nucleus (IDN) and intravenous (IV) administration to participants with FA.\n\nAll participants will receive SGT-212 and will be enrolled in the study for approximately 5 years.",[28],"Friedreich's Ataxia (FA)",[30,31,32,33,34,35,36,37,38,39,40],"SGT-212","Gene Therapy","Frataxin","Repeat Expansion","Neuromuscular","Cardiac","Ataxia","Movement Disorder","Rare disease","AAV","AAVhu68","RECRUITING","2026-06-17",{"date":44,"type":45},"2026-06-22","ACTUAL",{"date":47,"type":45},"2025-10-22",{"date":49,"type":22},"2032-02-29",{"name":51,"class":52},"Solid Biosciences Inc.","INDUSTRY",3,{"id":55,"slug":56,"hasResults":11,"nctId":57,"briefTitle":58,"officialTitle":59,"acronym":4,"eligibilityCriteria":60,"healthyVolunteers":11,"sex":61,"minAge":62,"maxAge":63,"enrollmentInfo":64,"targetDuration":4,"studyType":23,"phases":66,"briefSummary":68,"conditions":69,"keywords":71,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":42,"lastUpdatePostDateStruct":73,"startDateStruct":74,"completionDateStruct":76,"leadSponsor":78,"locationsCount":79},"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.","MALE","0 Years","17 Years",{"count":65,"type":22},60,[25,67],"PHASE2","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.",[70],"Duchenne Muscular Dystrophy",[72,31],"DMD",{"date":44,"type":45},{"date":75,"type":45},"2024-05-06",{"date":77,"type":22},"2031-05-06",{"name":51,"class":52},15,{"id":81,"slug":82,"hasResults":11,"nctId":83,"briefTitle":84,"officialTitle":85,"acronym":4,"eligibilityCriteria":86,"healthyVolunteers":11,"sex":61,"minAge":87,"maxAge":88,"enrollmentInfo":89,"targetDuration":4,"studyType":23,"phases":91,"briefSummary":93,"conditions":94,"keywords":95,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":100,"lastUpdatePostDateStruct":101,"startDateStruct":103,"completionDateStruct":104,"leadSponsor":106,"locationsCount":107},"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.","7 Years","11 Years",{"count":90,"type":22},80,[92],"PHASE3","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.",[70],[96,97,98,99],"SGT-003","Duchenne Muscular Dystrophy (DMD)","adeno-associated virus (AAV)","IMPACT DUCHENNE","2026-06-01",{"date":102,"type":45},"2026-06-02",{"date":47,"type":45},{"date":105,"type":22},"2034-01",{"name":51,"class":52},5,{"id":109,"slug":110,"hasResults":11,"nctId":111,"briefTitle":112,"officialTitle":113,"acronym":114,"eligibilityCriteria":115,"healthyVolunteers":11,"sex":17,"minAge":87,"maxAge":4,"enrollmentInfo":116,"targetDuration":4,"studyType":23,"phases":118,"briefSummary":119,"conditions":120,"keywords":122,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":100,"lastUpdatePostDateStruct":127,"startDateStruct":128,"completionDateStruct":130,"leadSponsor":132,"locationsCount":107},"100604328","phase-1-a-study-of-sgt-501-gene-therapy-in-catecholaminergic-polymorphic-ventricular-tachycardia-cpvt-100604328","NCT07148089","A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)","A Phase 1b, Multicenter, Open-Label, Dose Finding Study to Investigate the Safety and Tolerability of a Single Intravenous Dose of SGT-501 in Patients With Catecholaminergic Polymorphic Ventricular Tachycardia","ARTEMIS","Inclusion Criteria:\n\nType of Participant and Disease Characteristics:\n\n* Clinical diagnosis of CPVT, based on documented history of polymorphic or bidirectional non-sustained ventricular tachycardia with exercise or ventricular ectopy in a pattern consistent with CPVT on EST.\n* Central Screening laboratory determination of a RYR2 variant that is pathogenic or likely pathogenic for CPVT.\n* Documented history of life-threatening ventricular arrhythmic event defined as: survived sudden cardiac arrest, sudden cardiac arrest with appropriate implantable cardioverter defibrillator (ICD) shock, arrhythmic syncope, or sustained ventricular tachycardia (30 seconds or more) with or without ICD shock.\n* On stable dose (defined as no change in dose by more than 50% for at least 1 month prior to Screening) of standard-of-care therapy defined as a beta-blocker and\u002For flecainide.\n* Documented prior history of EST demonstrating a ventricular arrythmia score (VAS) score of ≥ 2.\n* For the first 2 participants in each cohort only: a properly functioning ICD device in place. Following review of data from Cohorts 1 and 2, the Data Safety and Monitoring Board (DSMB) will determine if this criterion is required for participants in Cohort 3.\n* Must be up to date with meningococcal vaccination per national guidelines or willing to receive meningococcal vaccine to achieve this.\n* Other inclusion criteria to be applied as per protocol.\n\nExclusion Criteria:\n\n* Abnormal liver function: gamma-glutamyl transferase (GGT) \\> 1.5 × upper limit of normal \\[ULN\\] or total bilirubin \\> ULN).\n* Abnormal renal function defined by estimated glomerular filtration rate \\\u003C 60 milliliter \u002Fminute (mL\u002Fmin)\u002F1.73-square meter (m\\^2) using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) Formula.\n* Clinically significant abnormalities of coagulation including international normalized ratio or activated partial thromboplastin time \\> 1.2 × ULN or platelets \\\u003C 150,000 cells\u002Fcubic millimeter (mm\\^3).\n* Potential concomitant cardiomyopathy or inherited arrhythmia as evidenced by pathogenic or likely pathogenic mutation other than RYR2 obtained on cardiac panel during Screening.\n* Current or prior treatment with an approved or investigational gene transfer drug.\n* Exposure to another investigational drug within 90 days prior to Screening or 5 half-lives since last administration, whichever is longer.\n* Contraindication or unwillingness to receive required immunosuppression regimen.\n* Body mass index ≥ 30 kilograms per square meter (kg\u002Fm\\^2).\n* Other exclusion criteria to be applied as per protocol.",{"count":117,"type":22},18,[25],"This is a Phase 1b, Multicenter, Open-Label, Dose Finding Study to Investigate the Safety and Tolerability of a Single Intravenous Dose of SGT-501 in participants with catecholaminergic polymorphic ventricular tachycardia (CPVT). The first-in-human (FIH) safety study will focus on obtaining safety data in adult participants. Cohort 1 and Cohort 2 (optional for dose exploration) will include participants ≥ 18 years of age. Cohort 3 will include participants ≥ 7 to \\\u003C 18 years of age and will be initiated following data and safety monitoring board (DSMB) recommendations. Participants will be monitored for 5 years post-administration of SGT-501 including the active treatment period (1 year) and long-term follow-up (LTFU) (4 years) period.",[121],"Catecholaminergic Polymorphic Ventricular Tachycardia",[123,124,125,126,35,31],"Catecholaminergic polymorphic ventricular tachycardia (CPVT)","SGT-501","Ryanodine Receptor 2 (RYR2)","adeno-associated virus serotype 8 (AAV8)",{"date":102,"type":45},{"date":129,"type":45},"2026-02-23",{"date":131,"type":22},"2031-05",{"name":51,"class":52},""]