An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
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|ClinicalTrials.gov Identifier: NCT03532542|
Recruitment Status : Enrolling by invitation
First Posted : May 22, 2018
Last Update Posted : August 5, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Duchenne Muscular Dystrophy||Drug: Casimersen Drug: Golodirsen||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||260 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Long-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen|
|Actual Study Start Date :||August 2, 2018|
|Estimated Primary Completion Date :||August 10, 2026|
|Estimated Study Completion Date :||August 10, 2026|
Patients amenable to exon 45 skipping who have completed a clinical trial evaluating casimersen will receive open-label casimersen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks.
Casimersen solution for IV infusion
Patients amenable to exon 53 skipping who have completed a clinical trial evaluating golodirsen will receive open-label golodirsen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks.
Golodirsen solution for IV infusion
- Number of Patients With Serious Adverse Events (SAEs) [ Time Frame: Up to 30 days after the last infusion of study drug (assessed up to 148 weeks) ]
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|Ages Eligible for Study:||7 Years to 23 Years (Child, Adult)|
|Sexes Eligible for Study:||Male|
|Gender Based Eligibility:||Yes|
|Accepts Healthy Volunteers:||No|
- Completed a clinical trial evaluating casimersen or golodirsen, per protocol.
- Is between 7 and 23 years of age, inclusive, at enrollment.
Other inclusion/exclusion criteria apply.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03532542
|Study Director:||Medical Director||Sarepta Therapeutics, Inc.|
|Responsible Party:||Sarepta Therapeutics, Inc.|
|Other Study ID Numbers:||
2017-004625-32 ( EudraCT Number )
|First Posted:||May 22, 2018 Key Record Dates|
|Last Update Posted:||August 5, 2022|
|Last Verified:||August 2022|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Duchenne muscular dystrophy
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked