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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03532542
Recruitment Status : Enrolling by invitation
First Posted : May 22, 2018
Last Update Posted : August 5, 2022
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE May 10, 2018
First Posted Date  ICMJE May 22, 2018
Last Update Posted Date August 5, 2022
Actual Study Start Date  ICMJE August 2, 2018
Estimated Primary Completion Date August 10, 2026   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 16, 2022)		 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) ]
Original Primary Outcome Measures  ICMJE
 (submitted: May 10, 2018)		 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)] ]
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
Official Title  ICMJE Long-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
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 Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE
  • Drug: Casimersen
    Casimersen solution for IV infusion
    Other Names:
    • SRP-4045
    • AMONDYS 45
  • Drug: Golodirsen
    Golodirsen solution for IV infusion
    Other Names:
    • SRP-4053
    • VYONDYS 53
Study Arms  ICMJE
  • Experimental: 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.
    Intervention: Drug: Casimersen
  • Experimental: 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.
    Intervention: Drug: Golodirsen
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Estimated Enrollment  ICMJE
 (submitted: November 16, 2018)		 260
Original Estimated Enrollment  ICMJE
 (submitted: May 10, 2018)		 150
Estimated Study Completion Date  ICMJE August 10, 2026
Estimated Primary Completion Date August 10, 2026   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages  ICMJE 7 Years to 23 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   Bulgaria,   Canada,   Czechia,   France,   Germany,   Israel,   Italy,   Poland,   Spain,   Sweden,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03532542
Other Study ID Numbers  ICMJE 4045-302
2017-004625-32 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Sarepta Therapeutics, Inc.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Sarepta Therapeutics, Inc.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director Sarepta Therapeutics, Inc.
PRS Account Sarepta Therapeutics, Inc.
Verification Date August 2022

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP