Clinical trial
Long-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
Name
4045-302
Description
The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).
Trial arms
Trial start
2018-08-02
Estimated PCD
2023-07-26
Trial end
2023-07-26
Status
Terminated
Phase
Early phase I
Treatment
Casimersen
Casimersen solution for IV infusion
Arms:
Casimersen
Other names:
SRP-4045, AMONDYS 45
Golodirsen
Golodirsen solution for IV infusion
Arms:
Golodirsen
Other names:
SRP-4053, VYONDYS 53
Size
171
Primary endpoint
Number of Patients With Serious Adverse Events (SAEs)
Up to 30 days after the last infusion of study drug (assessed up to 148 weeks)
Eligibility criteria
Inclusion Criteria:
* 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.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 171, 'type': 'ACTUAL'}}
Updated at
2023-08-21
1 organization
2 products
1 indication
Organization
Sarepta TherapeuticsProduct
CasimersenIndication
Duchenne muscular dystrophyProduct
Golodirsen