A clinical study in 3 parts with a microdystrophin (called GNT0004), a new gene therapy in boys with Duchenne disease who can still walk. The study will start with finding the proper treatment dose (part 1).After that, a comparative study versus placebo will start to assess the safety and the effectiveness of the proper dose of this therapy (part 2).In the end, a follow up period will continue to investigate the treatment safety and efficacy over longer time (part 3).

Phase 1

• Conditions: Duchenne Muscular Dystrophy; MedDRA version: 20.0Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2020-002093-27-FR
• Lead Sponsor: Genethon

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-06-24
• Last Update Posted: 5 April 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: 51

Inclusion Criteria

1Male
2Being included in the GNT-014-MDYF study
36 to 10 years (inclusive)
4Positive gene testing with detailed genotyping confirmation of DMD, ie DMD mutations expected to abolish the production of dystrophin
Are the trial subjects under 18? yes
Number of subjects for this age range: 51
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

1Presence of neutralizing antibodies against AAV8
2Cardiomyopathy based on physical/cardiological examination and echocardiography with Left Ventricular Ejection Fraction (LVEF) below 55% and/or fractional shortening (SF) below 28%
3Any respiratory assistance needed including non-invasive daytime or nocturnal ventilation
4Inability to perform the planned respiratory functions tests

Study & Design

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