Back to resultsA Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)
Primary Purpose
Duchenne Muscular Dystrophy
Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Eteplirsen
Sponsored by
About this trial
This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring DMD, Duchenne, Eteplirsen, Dystrophy, Dystrophin, Exon Skipping, Exon 51, Ambulatory, Pediatric
Eligibility 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.
Sites / Locations
- UZ-Gent
- Hopital Trousseau, Bâtiment lemariey
- Fondazione Policlinico Universitario Agostino Gemelli, UOC Neuropsichiatria Infantile
- UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Eteplirsen
Arm Description
Participants will receive eteplirsen via IV infusions, once weekly, for up to 284 weeks.
Outcomes
Primary Outcome Measures
Number of Participants Experiencing Adverse Events (AEs)
A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.
Number of Participants Experiencing Death Due to Adverse Events
A summary of all deaths regardless of causality is located in the 'Reported Adverse Events' section.
Number of Participants Experiencing Adverse Events of Special Interest (AESIs)
AESIs were defined as any AE that was of scientific and medical concern specific to study treatment, for which ongoing and rapid communication by the Investigator to the sponsor was appropriate. AESIs included findings potentially indicative of hepatic and renal abnormalities, hypersensitivity, and thrombocytopenia. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.
Secondary Outcome Measures
Full Information
NCT ID
NCT03985878
First Posted
June 11, 2019
Last Updated
July 25, 2023
Sponsor
Sarepta Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT03985878
Brief Title
A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)
Official Title
An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Terminated
Why Stopped
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.
Study Start Date
June 26, 2019 (Actual)
Primary Completion Date
August 31, 2022 (Actual)
Study Completion Date
August 31, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sarepta Therapeutics, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
DMD, Duchenne, Eteplirsen, Dystrophy, Dystrophin, Exon Skipping, Exon 51, Ambulatory, Pediatric
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Eteplirsen
Arm Type
Experimental
Arm Description
Participants will receive eteplirsen via IV infusions, once weekly, for up to 284 weeks.
Intervention Type
Drug
Intervention Name(s)
Eteplirsen
Other Intervention Name(s)
AVI-4658, EXONDYS 51, EXONDYS
Intervention Description
Eteplirsen IV infusion once weekly.
Primary Outcome Measure Information:
Title
Number of Participants Experiencing Adverse Events (AEs)
Description
A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.
Time Frame
Up to 162 weeks
Title
Number of Participants Experiencing Death Due to Adverse Events
Description
A summary of all deaths regardless of causality is located in the 'Reported Adverse Events' section.
Time Frame
Up to 162 weeks
Title
Number of Participants Experiencing Adverse Events of Special Interest (AESIs)
Description
AESIs were defined as any AE that was of scientific and medical concern specific to study treatment, for which ongoing and rapid communication by the Investigator to the sponsor was appropriate. AESIs included findings potentially indicative of hepatic and renal abnormalities, hypersensitivity, and thrombocytopenia. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.
Time Frame
Up to 162 weeks
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Sarepta Therapeutics, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
UZ-Gent
City
Gent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
Hopital Trousseau, Bâtiment lemariey
City
Paris
ZIP/Postal Code
75021
Country
France
Facility Name
Fondazione Policlinico Universitario Agostino Gemelli, UOC Neuropsichiatria Infantile
City
Rome
ZIP/Postal Code
00168
Country
Italy
Facility Name
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre
City
London
State/Province
England
ZIP/Postal Code
WC1N 1EH
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)
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