An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

Sarepta Therapeutics

Status and phase: Terminated; Phase 3

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: Casimersen; Drug: Golodirsen

Study type: Interventional

Funder types: Industry

Identifiers

NCT03532542

2017-004625-32 (EudraCT Number)

4045-302

Details and patient eligibility

About

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).

Enrollment: 171 patients

Sex: Male

Ages: 7 to 23 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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171 participants in 2 patient groups

Casimersen

Experimental group

Description:

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.

Treatment:

Drug: Casimersen

Golodirsen

Experimental group

Description:

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.

Treatment:

Drug: Golodirsen