A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patients

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: EUCTR2016-000951-29-IT
• Lead Sponsor: SAREPTA THERAPEUTICS, INC.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2018-02-07
• Last Update Posted: 12 February 2018

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: 12

Inclusion Criteria

1. Be a male between 6 months to 48 months of age, inclusive.
2. Have an established clinical diagnosis of DMD with a deletion mutation amenable to exon 51 skipping (e.g., deletions of exons 45-50, 47-50, 48-50, 49-50, 50, 52, 52-63).
3. Have a parent(s) or legal guardian(s) who is able to understand and comply with the study requirements and is willing to provide written informed consent for the patient to participate in the study.
Are the trial subjects under 18? yes
Number of subjects for this age range: 1
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. Has received any pharmacologic treatment that might have an effect on muscle strength or function within 12 weeks prior to dosing at Week 1 (e.g., growth hormone, anabolic steroids).
2. Has received previous or current treatment with any experimental treatment. Prior drisapersen therapy is permitted if a patient has not received drisapersen for 6 months prior to the Week 1 dose.
3. Has a clinically significant illness other than DMD, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, behavioural disease, or malignancy likely to impair the patient's ability to participate in this study.
4. Has a clinically significant laboratory abnormality that is either not expected or is of a greater severity than what is expected in DMD patients.
5. Has any other condition that, in the Investigator's opinion, could interfere with the patient's participation in the study.

Study & Design

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