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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 : November 5, 2019
Sponsor:
Information provided by (Responsible Party):
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).

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Casimersen Drug: Golodirsen Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 260 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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


Arm Intervention/treatment
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.
Drug: Casimersen
Casimersen solution for IV infusion
Other Name: SRP-4045

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.
Drug: Golodirsen
Golodirsen solution for IV infusion
Other Name: SRP-4053




Primary Outcome Measures :
  1. 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
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.


Information from the National Library of Medicine

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


Locations
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United States, Arizona
Neuromuscular Research Center
Phoenix, Arizona, United States, 85028
United States, California
University of California Los Angeles
Los Angeles, California, United States, 90095
United States, Florida
NW Florida Clinical Research Group, LLC
Gulf Breeze, Florida, United States, 32561
United States, Illinois
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Iowa
University of Iowa Childrens Hospital
Iowa City, Iowa, United States, 52242
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Maryland
Johns Hopkins University School of Medicine
Baltimore, Maryland, United States, 21205
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Abigail Wexner Research Institute at Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
United States, Texas
Children's Medical Center Dallas
Dallas, Texas, United States, 75207
Belgium
UZ Gent
Gent, Oost-Vlaanderen, Belgium, 9000
UZ Leuven
Leuven, Belgium, 3000
Italy
Fondazione Policlinico Universitario A Gemelli
Milano, Italy, 20123
Spain
Hospital de La Santa Creu i Sant Pau
Barcelona, Cataluña, Spain, 08041
United Kingdom
Great Ormond Street Hospital (GOSH)
London, United Kingdom, WC1N 1EH
Royal Victoria Infirmary
Newcastle Upon Tyne, United Kingdom, NE1 4LP
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
Investigators
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Study Director: Medical Director Sarepta Therapeutics, Inc.

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Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03532542     History of Changes
Other Study ID Numbers: 4045-302
2017-004625-32 ( EudraCT Number )
First Posted: May 22, 2018    Key Record Dates
Last Update Posted: November 5, 2019
Last Verified: November 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sarepta Therapeutics, Inc.:
Duchenne muscular dystrophy
Exon Skipping
DMD
Exon 53
Exon 45
Duchenne
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked