Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy: A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long term safety follow up part, in ambulant boys

Ongoing corticosteroid therapy with prednisolone (or prednisone) according to standard of care (participants receiving deflazacort or vamorolone daily dose can switch to prednisolone (or prednisone) 4 weeks before screening)

• Enrolled in the GNT-014-MDYF study (a natural history study preceding the present gene therapy study)
• Able to achieve a score in the NSAA (North Star Ambulatory Assessment) scale 18 and Time to Rise From Floor (RFF) 7 sec at screening visit
• Up to date vaccination record including hepatitis A and B, PCV

• DMD patients with any mutations affecting: - exons 1 through 17, (and any mutations affecting other exons as per a country’s requirement which will be applicable to this specific country) for Part 1 participants - affecting exons 8 and/or 9 (and any mutations affecting other exons as per a country’s requirement which will be applicable to this specific country) for Part 2 participants
• Presence of neutralizing antibodies against AAV8
• Cardiomyopathy based on physical/cardiological examination and echocardiography (or cardiac MRI if available) with Left Ventricular Ejection Fraction (LVEF) below 55% and fractional shortening (SF) below 28%
• Any respiratory assistance needed including non-invasive daytime or nocturnal ventilation
• Inability to perform the planned respiratory functions tests

• Email: [email protected]
• Phone: 0169472828