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

Phase 3

Terminated

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Drug: Casimersen; Drug: Golodirsen

• Registration Number: NCT03532542
• Lead Sponsor: Sarepta Therapeutics, Inc.

Brief Summary

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-05-10
• Study First Submitted QC: 2018-05-10
• Study First Posted: 2018-05-22
• Study Start: 2018-08-02
• Primary Completion: 2023-07-26
• Study Completion: 2023-07-26
• Status Verified: 2023-08
• Last Update Submitted: 2023-08-16
• Last Update Posted: 2023-08-21

Recruitment & Eligibility

• Status: TERMINATED
• Sex: Male
• Target Recruitment: 171

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.

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Casimersen	Casimersen	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.
Golodirsen	Golodirsen	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.

Primary Outcome Measures

Name	Time	Method
Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)	Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)	A treatment-emergent adverse event (TEAE) was any untoward medical occurrence in a clinical study participant that did not necessarily have a causal relationship with the study drug. A TEAE could, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurred during or after administration of the study drug, whether or not considered related to the study drug. A TESAE was any TEAE that resulted in any of the following outcomes: death, a life-threatening event, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity, or an important medical event (that is, may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously mentioned outcomes). A summary of serious and all other non-serious TEAEs regardless of causality is located in the Reported Adverse Events module.

Trial Locations

Locations (50)

Children's Hospital of Wisconsin, Corporate Center Suite 540; 🇺🇸 Milwaukee, Wisconsin, United States

Neuromuscular Research Center; 🇺🇸 Phoenix, Arizona, United States

Children's Hospital Los Angeles; 🇺🇸 Los Angeles, California, United States

University of California Los Angeles; 🇺🇸 Los Angeles, California, United States

Stanford Neuroscience Health Center; 🇺🇸 Palo Alto, California, United States

Rady Children's Hospital- San Diego; 🇺🇸 San Diego, California, United States

UF Health: University of Florida Clinical Research Center; 🇺🇸 Gainesville, Florida, United States

NW Florida Clinical Research Group, LLC; 🇺🇸 Gulf Breeze, Florida, United States

Rare Disease Research, LLC; 🇺🇸 Atlanta, Georgia, United States

Ann and Robert H Lurie Childrens Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

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