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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 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. Identifier: NCT03985878
Recruitment Status : Enrolling by invitation
First Posted : June 14, 2019
Last Update Posted : April 14, 2020
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

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 DMD patients who have successfully completed the 96-week eteplirsen study: Study 4658-102 (NCT03218995)

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

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Study Type : Interventional  (Clinical Trial)
Estimated 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
Estimated Primary Completion Date : November 2026
Estimated Study Completion Date : February 2027

Arm Intervention/treatment
Experimental: Eteplirsen
Patients will receive eteplirsen via intravenous (IV) infusions, once weekly, for up to 284 weeks.
Drug: Eteplirsen
Eteplirsen IV infusion once weekly.

Primary Outcome Measures :
  1. Incidence of Adverse Events (AEs) [ Time Frame: Up to 288 weeks ]
  2. Incidence of Death Due to Adverse Events [ Time Frame: Up to 288 weeks ]
  3. Incidence of 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.

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

Inclusion Criteria:

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

Exclusion Criteria:

  • Patient has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the patient, 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.

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 identifier (NCT number): NCT03985878

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Gent, Belgium, 9000
Hopital Trousseau, Bâtiment lemariey
Paris, France, 75021
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.
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Study Director: Medical Director Sarepta Therapeutics, Inc.
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Responsible Party: Sarepta Therapeutics, Inc. 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: April 14, 2020
Last Verified: April 2020

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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.:
Exon Skipping
Exon 51
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