A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients

Phase 1

• Conditions: Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping; 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: EUCTR2017-004625-32-ES
• Lead Sponsor: Sarepta Therapeutics, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2018-09-10
• Last Update Posted: 17 September 2018

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: 150

Inclusion Criteria

1. Completed a clinical trial evaluating casimersen or golodirsen, per protocol.
2. If sexually active, agrees to use a male condom during such activity for the entire duration of the study and for 90 days after the last dose of study drug. The sexual partner must also use a medically acceptable form of contraceptive (eg, female oral contraceptives) during this time frame.
3. Is able to understand and comply with all the study requirements and, if under 18 years of age, had as (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with all the study requirements.
4. Is willing to provide informed assent (if applicable) and has (a) parent(s) or legal guardian(s) who is (are) willing to provide written informed consent for the patient to participate in the study.
5. Is between 7 and 23 years of age, inclusive, at randomization
Are the trial subjects under 18? yes
Number of subjects for this age range: 135
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 15
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. A medical condition that could, in the Investigator’sopinion, adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results.
2. Any patient who, in the Investigator’s opinion, seems unable/unwilling to comply with the study procedures.

Study & Design

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