[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Italfarmaco\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":117},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,43,68,91],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100523284","phase-3-study-on-efficacy-and-safety-of-givinostat-versus-hydroxyurea-in-patients-with-polycythemia-vera-100523284",false,"NCT06093672","Study on Efficacy and Safety of Givinostat Versus Hydroxyurea in Patients With Polycythemia Vera","Randomized, Open-label, Multicenter Phase 3 Study to Assess the Efficacy and Safety of GIVinostat Versus Hydroxyurea IN JAK2V617F-positive High-risk Polycythemia Vera Patients: the GIV-IN PV TRIAL","GIV-IN-PV","Core Treatment - Inclusion Criteria:\n\n* Patients must have been diagnosed with PV according to the 2016 WHO criteria before randomization\n* Patients must have JAK2V617F-positive disease\n* Patients with PV must meet the definition of HR for thrombosis (i.e., HR) within 3 years before screening as follows:\n\n  * Age ≥ 60 years, and\u002For\n  * Prior thrombosis.\n* Patients must be in need of treatment at screening, defined by the presence of at least one of the following:\n\n  * HCT ≥ 45% or HCT \\\u003C 45% with at least 1 phlebotomy performed in the 3 months before screening, or\n  * WBC count \\> 10 × 109\u002FL, or\n  * PLT count \\> 400 × 109\u002FL.\n* Patients must have normalized HCT (i.e., HCT \\\u003C 45%) at randomization\n\nExtended Treatment - Inclusion Criteria\n\n* Patients must have completed the Week 48 visit of the DSC\u002F08\u002F2357\u002F32 core treatment phase and:\n\n  1. if the patient received givinostat, a complete hematological response (CHR) at Week 48 shall be achieved\n  2. if the patient received HU, did not achieve a CHR (see above for the definition) at Week 48\n\n     Core Treatment phase - Exclusion Criteria\n* Patients pre-treated with HU with a documented history of resistance or intolerance to HU defined by the original ELN criteria\n* Patients with a QTcF value of \\> 450 msec for males and \\> 460 msec for females at the Screening visit (as the mean of 3 consecutive readings 5 minutes apart in the event a first ECG demonstrates a prolonged QTcF interval); congenital or acquired history of QTc prolongation or ventricular arrhythmias, at the Screening visit\n* Splanchnic thrombosis and\u002For thrombosis of the cerebral venous sinuses and\u002For splenectomy in the medical history\n* Patients with clinically significant cardiovascular disease\n* Patients with myocardial infarction, stroke or unstable angina within the 6 months prior to screening.\n* Patients with inadequate liver or renal function at screening\n* Uncontrolled hypertriglyceridemia at screening, i.e., triglycerides ˃ 1.5 × ULN\n* Previous treatment with a JAK2 or HDAC inhibitor or 32-phosphorus (radioactive isotope) therapy.\n* Patients being treated concurrently with any investigational agent or prior participation in an interventional clinical study within the 30 days prior to screening or within 5 half-lives of the investigational product, whichever is longer.\n* Pregnant or nursing women\n\nExtended treatment phase - Exclusion criteria\n\n* For patients randomized to givinostat in the core treatment phase - Patients with a QTcF value at Week 48 of \\> 500 msec\n* For patients randomized to HU in the core treatment phase:\n\n  * PLT count ≤ 150 × 109\u002FL at Week 48\n  * ANC \\\u003C 1.2 × 109\u002FL at Week 48\n  * Uncontrolled hypertriglyceridemia at Week 48\n  * Patients with a QTcF value at Week 48 of \\> 450 msec for males and \\> 460 msec for female","ALL","18 Years",{"count":20,"type":21},220,"ESTIMATED","INTERVENTIONAL",[24],"PHASE3","The goal of this clinical trial is to compare the efficacy and safety of givinostat to hydroxyurea in Jak2V617F-positive high risk polycythemia vera patients.",[27],"Polycythemia Vera",[27,29],"Givinostat","RECRUITING","2026-03-24",{"date":33,"type":34},"2026-03-25","ACTUAL",{"date":36,"type":34},"2024-03-26",{"date":38,"type":21},"2026-07",{"name":40,"class":41},"Italfarmaco","INDUSTRY",90,{"id":44,"slug":45,"hasResults":11,"nctId":46,"briefTitle":47,"officialTitle":48,"acronym":4,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":50,"minAge":51,"maxAge":4,"enrollmentInfo":52,"targetDuration":4,"studyType":22,"phases":54,"briefSummary":56,"conditions":57,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":59,"lastUpdatePostDateStruct":60,"startDateStruct":62,"completionDateStruct":64,"leadSponsor":66,"locationsCount":67},"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.","MALE","7 Years",{"count":53,"type":21},206,[55,24],"PHASE2","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.",[58],"Duchenne Muscular Dystrophy","2026-01-19",{"date":61,"type":34},"2026-01-21",{"date":63,"type":34},"2017-10-24",{"date":65,"type":21},"2029-12",{"name":40,"class":41},39,{"id":69,"slug":70,"hasResults":11,"nctId":71,"briefTitle":72,"officialTitle":73,"acronym":4,"eligibilityCriteria":74,"healthyVolunteers":11,"sex":50,"minAge":75,"maxAge":76,"enrollmentInfo":77,"targetDuration":4,"studyType":22,"phases":79,"briefSummary":80,"conditions":81,"keywords":82,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":83,"lastUpdatePostDateStruct":84,"startDateStruct":86,"completionDateStruct":88,"leadSponsor":89,"locationsCount":90},"100575234","phase-2-pharmacokinetics-and-safety-of-givinostat-in-dmd-patients-ages-from-at-least-2-years-to-less-then-6-years-old-100575234","NCT06769633","Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old","A Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years Old","Inclusion Criteria - Core Phase:\n\n1. Male children aged ≥2 to \\\u003C6 years at screening (subjects ≥6 years of age at screening will not be enrolled into the study)\n2. Written consent provided by parent\u002Flegal guardian and subject written assent, if applicable (according to local regulation)\n3. A genetic diagnosis of DMD\n4. Corticosteroid treatment considerations:\n\n   1. For subject receiving a stable dose or oral systemic corticosteroids:\n\n      No significant change in dose or dosing regimen (except for adjustments due to body weight change) for a minimum of 3 months immediately prior to the start of the study drug or\n   2. For subjects without current corticosteroid treatment:\n\nMust not start corticosteroids in the Core Phase of the study (ie, first 48 weeks).\n\nInclusion Criteria - Extension Phase:\n\n1. Must have participated in the Core Phase study (48 weeks) and have attended the End of Treatment Visit\n2. Give informed consent and \u002For assent in writing signed by the parent\u002Flegal guardian and\u002For subject (according to local regulation)\n3. In stable oral systemic corticosteroids treatment with no significant change in dose or dosing regimen (except for adjustments due to body weight change). For subjects without corticosteroids during the Core Phase, the treatment can be started based on the Investigator's clinical medical judgement.\n\nExclusion Criteria - Core Phase\n\n1. Exposure to another investigational drug within 3 months prior to the start of the study drug\n2. Exposure to any dystrophin restoration product (eg, Ataluren, Exon skipping) within 6 months prior to the start of study drug\n3. Received any gene therapy (eg, AAV Micro-dystrophin delivery) within 12 months prior to start of study drug\n4. Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to the start of the study drug (eg, growth hormone). Note: Vitamin D, calcium, and any other supplements will be allowed.\n5. Have had surgery that might have an effect on muscle strength or function within 3 months prior to start of the study drug or planned surgery at any time during the study\n6. The presence of other clinically significant disease, which, in the Investigator's opinion, could adversely affect subject's safety, making it unlikely to complete the study or to be compliant with study-specific requirements that could impair the assessment of study results\n7. Diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD, based on Investigator clinical medical judgement\n8. Platelet count, white blood cells, and\u002For haemoglobin counts \\\u003C lower limit of normal (LLN) at screening (Note: for abnormal screening laboratory test results \\[\\\u003CLLN\\], the platelet count, white blood cell, and haemoglobin will be repeated once; if the repeat test result is still \\\u003CLLN, the subject will be excluded)\n9. Current or history of liver disease or impairment, including but not limited to a baseline elevated total bilirubin (ie, \\>1.5 × upper limit of normal \\[ULN\\]), unless secondary to Gilbert disease or pattern consistent with Gilbert disease\n10. Inadequate renal function, as defined by serum Cystatin C result \\>2 × ULN (Note: if the value is \\>2 × ULN, the serum Cystatin C will be repeated once; if the repeated test result is still \\>2 × ULN, the subject will be excluded)\n11. Fasting triglycerides \\>300 mg\u002FdL (3.42 mmol\u002FL) at screening (Note: if the value is \\>300 mg\u002FdL, the triglycerides will be repeated once; if the repeated test result is still \\>300 mg\u002FdL in fasting, the subject should be excluded)\n12. Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening\n13. Baseline corrected QT interval using Fridericia's formula (QTcF) \\>450 msec (as the mean of 3 consecutive readings taken 5 minutes apart) or history of additional risk factors for torsades de pointes (ie, heart failure, hypokalaemia, or family history of long QT syndrome)\n14. Psychiatric illness or social situations rendering the potential subject unable to understand and comply with the muscle function tests and\u002For with the study protocol procedures, based on the Investigator's clinical medical judgement\n15. Hypersensitivity to any component of study drug\n16. Sorbitol intolerance or malabsorption or have the hereditary form of fructose intolerance.\n17. Body weight \\\u003C10 kg at screening.\n\nExclusion Criteria - Extension Phase\n\n1. Platelet count, white blood cells, and\u002For haemoglobin \\\u003CLLN at EOT\u002FV12 (Note: for abnormal laboratory test results \\[\\\u003CLLN\\], the platelet count, white blood cell, and haemoglobin will be repeated once; if the repeat test result is still \\\u003CLLN, the subject will be excluded)\n2. Current liver disease or impairment, including but not limited to an elevated total bilirubin (ie, \\>1.5 × ULN), unless secondary to Gilbert disease or pattern consistent with Gilbert disease\n3. Inadequate renal function, as defined by serum Cystatin C result \\>2 × ULN (Note: if the value is \\>2 × ULN, the serum Cystatin C will be repeated once; if the repeated test result is still \\>2 × ULN, the subject will be excluded)\n4. Fasting triglycerides \\>300 mg\u002FdL (3.42 mmol\u002FL; Note: if the value is \\>300 mg\u002FdL, the triglycerides will be repeated once; if the repeated test result is still \\>300 mg\u002FdL in fasting condition, the subject should be excluded)\n5. 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\n6. Evidence of psychiatric illness or social situations rendering the potential subject unable to understand and comply with the muscle function tests and\u002For with the study protocol procedures, based on the Investigator's clinical medical judgement.","2 Years","6 Years",{"count":78,"type":21},18,[55],"This is a Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in younger DMD Patients.\n\n* Planned screening duration: approximately 4 weeks\n* Planned Core Treatment duration: approximately 48 weeks\n* Planned Extension Treatment duration: approximately 96 weeks\n* Planned Follow Up duration: approximately 4 weeks (± 7 days)\n* Total duration of study participation: up to 151 weeks (ie, 37-38 months)",[58],[58,29],"2025-07-09",{"date":85,"type":34},"2025-07-10",{"date":87,"type":34},"2025-01-02",{"date":65,"type":21},{"name":40,"class":41},9,{"id":92,"slug":93,"hasResults":11,"nctId":94,"briefTitle":95,"officialTitle":96,"acronym":97,"eligibilityCriteria":98,"healthyVolunteers":11,"sex":50,"minAge":99,"maxAge":100,"enrollmentInfo":101,"targetDuration":4,"studyType":22,"phases":103,"briefSummary":104,"conditions":105,"keywords":106,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":108,"lastUpdatePostDateStruct":109,"startDateStruct":111,"completionDateStruct":113,"leadSponsor":115,"locationsCount":116},"100510947","phase-3-efficacy-safety-and-tolerability-of-givinostat-in-non-ambulant-patients-with-duchenne-muscular-dystrophy-100510947","NCT05933057","Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy","Randomised, Double-blind, Placebo-controlled, Multicentre Study to Evaluate the Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy","ULYSSES","Inclusion Criteria:\n\nPatients must satisfy all the following criteria:\n\n1. Children and adolescent males aged ≥ 9 to \\\u003C18 years at screening (patients ≥ 18 years of age at screening will not be enrolled into the study)\n2. Are able to give informed assent and\u002For consent in writing signed by the patient and\u002For parent\u002Flegal guardian (according to local regulations)\n3. A genetic diagnosis of DMD\n4. Non-ambulant, defined as being wheelchair bound and:\n\n   1. Unable to perform the 10-meter walk\u002Frun test (10MWT), or\n   2. Unable to complete the 10MWT in 30 seconds or less, without any support or devices\n5. Performance of the Upper Limb test (PUL version 2.0) entry item scores 3 to 6\n6. If on medication for DMD-associated cardiomyopathy (eg, ACE inhibitor, β-blocker, diuretics), stable for ≥1 month immediately prior to start of study treatment, if any\n7. Stable corticosteroids, defined as:\n\n   1. Receiving systemic corticosteroids for a minimum of 6 months immediately prior to start of study treatment\n   2. No significant change in dose or dosing regimen (except for adjustments due to body weight change) for a minimum of 6 months immediately prior to start of study treatment\n8. Willing to use adequate contraception. Effective contraceptive methods must be used from randomisation visit through 3 months after the last dose of study drug, and include the following:\n\n   1. True abstinence (ie, absence of any sexual intercourse), when in line with the preferred and usual lifestyle of the patient. Periodic abstinence (eg, calendar, ovulation, post-ovulation, and symptothermal methods) and withdrawal are not acceptable methods of contraception\n   2. Condom with spermicide and the female partner must use an effective method of contraception, such as an oral, transdermal, injectable or implanted hormonal contraceptive; intrauterine device; bilateral tubal occlusion, or a diaphragm or a barrier method of contraception in conjunction with spermicidal jelly such as for example cervical cap with spermicide jelly.\n\nExclusion Criteria:\n\nPatients will be excluded from the study if they satisfy any of the following criteria:\n\n1. Exposure to another investigational drug within 3 months prior to start of study treatment.\n2. Have exposure to any dystrophin restoration product (eg, Ataluren, Exon skipping) within 6 months prior to the start of study treatment\n3. Having received any gene therapy (eg, AAV Micro-dystrophin delivery) prior to start of study treatment\n4. Use of any pharmacologic treatment or supplement (other than corticosteroids), that might have had an effect on muscle strength or function within 3 months prior to the start of study treatment (eg, growth hormone); vitamin D, calcium and any other supplements will be allowed\n5. Use of testosterone, unless used as a replacement therapy for the treatment of delayed puberty. The testosterone dose and regimen should be stable within 6 months prior to the start of study treatment, and circulating testosterone levels should be within the normal ranges for the patient's age\n6. Elbow-flexion contractures \\>30° in the dominant arm\n7. Inability to perform consistent PUL 2.0 measurement within ±2 points without shoulder domain or within ±3 points with shoulder domain during paired testing at screening\n8. Forced Vital Capacity % of predicted \\\u003C40%\n9. Requirement for daytime ventilator assistance (Note: Night ventilator assistance and use of bi-level positive airway pressure therapy is allowed)\n10. Episode of respiratory failure within the 8 weeks prior to screening\n11. Symptomatic cardiomyopathy or heart failure and\u002For left ventricular ejection fraction \\\u003C45%\n12. Baseline corrected QT interval using Fredericia's formula (QTcF) \\>450 msec (as the mean of 3 consecutive readings 5 minutes apart) or history of additional risk factors for torsades de pointes (eg, heart failure, hypokalaemia, or family history of long QT syndrome)\n13. Major surgical procedure (including scoliosis surgery) planned within 1 year of the start of study treatment\n14. Poorly controlled asthma or underlying lung disease such as bronchitis, bronchiectasis, emphysema, recurrent pneumonia that in the opinion of the Investigator might impact respiratory function\n15. Platelets, white blood cells, and\u002For haemoglobin \\\u003C lower limit of normal (LLN) at screening (Note: for abnormal screening laboratory test results \\[\\\u003CLLN\\], the platelets count, white blood cell, and haemoglobin will be repeated once; if the repeat test result is still \\\u003CLLN, the patient should be excluded)\n16. Fasting triglycerides \\>300 mg\u002FdL (3.42 mmol\u002FL) at screening (Note: if the value is \\>300 mg\u002FdL, the triglycerides will be repeated once; if the repeated test result is still \\>300 mg\u002FdL, the patient should be excluded)\n17. Current or history of liver disease or impairment, including but not limited to a baseline elevated total bilirubin (ie, \\>1.5 × upper limit of normal \\[ULN\\]), unless secondary to Gilbert disease or pattern consistent with Gilbert disease\n18. Inadequate renal function, as defined by serum Cystatin C result \\>2 × ULN (Note: if the value is \\>2 × ULN, the serum Cystatin C will be repeated once; if the repeated test result is still \\>2 × ULN, the patient should be excluded)\n19. Positive test for hepatitis B surface antigen, hepatitis C antibody, or human immunodeficiency virus at screening\n20. Hypersensitivity to any component of study medication\n21. Sorbitol intolerance or malabsorption, or have the hereditary form of fructose intolerance\n22. Diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD, based on Investigator judgement\n23. Psychiatric illness or social situations rendering the potential patient unable to understand and comply with the muscle function tests and\u002For with the study protocol procedures, based on Investigator judgement\n24. Have contraindications to MRI scan (eg, claustrophobia, metal implants, or uncontrolled seizure disorder), based on Investigator's judgement.","9 Years","17 Years",{"count":102,"type":21},138,[24],"This is a randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy, safety, and tolerability of givinostat in non-ambulant male paediatric (aged 9 to \\\u003C18 years) patients with DMD. 138 patients will be randomised 2:1 to givinostat or placebo and will be treated for 18 months.\n\n* Planned screening duration: approximately 4 weeks (±14 days)\n* Planned treatment duration: 18 months (approximately 72 weeks)\n* Planned follow-up duration: 4 weeks (±7 days) (for patients not participating in the long-term safety study)\n* Total duration of study participation: up to 83 weeks (ie, 20-21 months)",[58],[58,107],"givinostat","2025-05-09",{"date":110,"type":34},"2025-05-11",{"date":112,"type":34},"2024-02-19",{"date":114,"type":21},"2028-02",{"name":40,"class":41},20,""]