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Trial record 1 of 1 for:    NCT03985878
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A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)

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: NCT03985878
Recruitment Status : Terminated (Participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment. There are no safety concerns with Eteplirsen.)
First Posted : June 14, 2019
Last Update Posted : October 3, 2022
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Study Description
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Brief Summary:
The purpose of this extension study is to evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male participants with DMD who have successfully completed the 96-week eteplirsen Study 4658-102.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Eteplirsen Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
Actual Study Start Date : June 26, 2019
Actual Primary Completion Date : August 31, 2022
Actual Study Completion Date : August 31, 2022

Resource links provided by the National Library of Medicine

Drug Information available for: Eteplirsen

Arms and Interventions
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Arm Intervention/treatment
Experimental: Eteplirsen
Participants will receive eteplirsen via IV infusions, once weekly, for up to 284 weeks.
 Drug: Eteplirsen
Eteplirsen IV infusion once weekly.
Other Names:
  • AVI-4658
  • EXONDYS 51
  • EXONDYS


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants with Adverse Events (AEs) [ Time Frame: Up to 288 weeks ]
  2. Number of Deaths Due to Adverse Events [ Time Frame: Up to 288 weeks ]
  3. Number of Participants with Adverse Events of Special Interest (AESIs) [ Time Frame: Up to 288 weeks ]
    AESIs will be defined as any AE that is of scientific and medical concern specific to study treatment, for which ongoing and rapid communication by the Investigator to the sponsor is appropriate. AESIs will include findings potentially indicative of hepatic and renal abnormalities, hypersensitivity, and thrombocytopenia.


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 5 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Participant successfully completes 96 weeks of treatment in Study 4658-102.

Exclusion Criteria:

  • Participant has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the participant, or make it unlikely that the course of treatment or follow-up would be completed, or impair the assessment of study results.

Other inclusion/exclusion criteria apply.

Contacts and Locations
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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): NCT03985878


Locations
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Belgium
UZ-Gent
Gent, Belgium, 9000
France
Hopital Trousseau, Bâtiment lemariey
Paris, France, 75021
Italy
Fondazione Policlinico Universitario Agostino Gemelli, UOC Neuropsichiatria Infantile
Rome, Italy, 00168
United Kingdom
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre
London, England, United Kingdom, WC1N 1EH
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
Investigators
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Study Director: Medical Director Sarepta Therapeutics, Inc.
More Information
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Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03985878    
Other Study ID Numbers: 4658-102-OLE
2019-000337-39 ( EudraCT Number )
First Posted: June 14, 2019    Key Record Dates
Last Update Posted: October 3, 2022
Last Verified: September 2022

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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.:
DMD
Duchenne
Eteplirsen
Dystrophy
Dystrophin
 Exon Skipping
Exon 51
Ambulatory
Pediatric
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