A research study of a new investigational medicinal product for thetreatment of Duchenne Muscular Dystrophy patients who have completedstudy 4658-102

Phase 1

• Conditions: Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping; MedDRA version: 20.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2019-000337-39-GB
• Lead Sponsor: Sarepta Therapeutics, Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-04-23
• Last Update Posted: 3 November 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: 15

Inclusion Criteria

1. Patient has successfully completed 96 weeks of treatment in Study
4658-102.
2. Parent(s) or legal guardian(s) who is/are able to understand and
comply with the study requirements.
3. Patient and/or their parent(s)/legal guardian(s) are willing and able
to provide signed informed consent.
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified