= Fully recruited
= Not yet recruiting
= Enrolling by invitation
This study will evaluate the safety and efficacy of gene transfer therapy in boys aged between 4 and 7 with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.
This is a randomized, double-blind, placebo-controlled 2-part study of systemic gene delivery of SRP-9001 in approximately 120 male DMD ambulatory subjects aged between 4 and 7 years of age. All patients will have the opportunity to receive intravenous (IV) SRP-9001 in either Part 1 or Part 2. The study will consist of 4 periods as follows:
• A Baseline Period (pre-infusion) which begins when eligibility is confirmed and ends on the day prior to the Day 1 infusion during which baseline assessments will be completed.
• Approximately 60 subjects will receive IV SRP-9001 and approximately 60 subjects will receive placebo (saline, 0.9% sodium chloride solution) in the Infusion Period in Part 1. In the Infusion Period in Part 2, subjects who received placebo in Part 1 will receive IV SRP-9001, and subjects who received SRP-9001 in Part 1 will receive placebo. All subjects, parents/caregivers, Investigators, and site staff, with the exception of the unblinded site pharmacist, will be blinded to subject treatment (SRP-9001 or placebo).
• A 104-week Follow-up Period (post Part 1infusion) during which safety and efficacy parameters will be evaluated in Part 1 and Part 2. Subjects will be expected to attend both remote and in-person visits to complete required procedures/assessments. Additional, unscheduled visits are allowed per the Investigator’s clinical judgement. For subjects who complete the study, the last study visit will occur at Part 2 Week 52. For subjects who prematurely discontinue post-infusion follow-up, an end of study/early termination visit will be required; however, each subject
• Muscle biopsies are only required for those patients that enrol at a recruiting trial site carrying out biopsies (in the UK these location are Newcastle and London - GOSH).
• All patients who enrol at a trial site which is also an MRI site, must also participate in an MRI sub-study (In the UK this will be the following trial sites: Newcastle, Liverpool - Alder Hey, and London - GOSH.
PROMIS is a family of instruments developed and validated to assess health-related quality of life. Parents will be asked "Taking into account all aspects of your child's observable symptoms, physical ability, ability to perform daily activities and overall health, how would you rate the change in clinical status for your child since the study start? using the following rating scale 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse, and 7=very much worse."
Other inclusion or exclusion criteria could apply.