A Two-Part, Open-Label Systemic Gene Delivery Study To Evaluate The Safety And Expression Of RO7494222 (SRP-9001) In Subjects Under The Age Of Four With Duchenne Muscular Dystrophy (ENVOL)

Research summary

The purpose of this study is to assess the safety and expression of delandistrogene moxeparvovec (also referred to as RO7494222 or SRP-9001), a novel micro-dystrophin gene therapy, in participants under the age of 4 with Duchenne muscular dystrophy (DMD). Current treatment options for DMD include systemic corticosteroids which confer limited benefit while introducing significant side effects, and therapies aimed at restoring the production of endogenous dystrophin protein (e.g., exon-skipping) which are only available to a small proportion of patients with specific genetic mutations. Therefore, there continues to be a high unmet medical need for treatments that offer meaningful benefit to a wider range of patients with DMD. This study will enroll a very young population because therapeutic intervention at an early age (in some cases, before symptoms of DMD become evident) has the potential to improve the long-term prognosis of patients with DMD and change the course of their disease.

Principal Investigator

Prof Laurent Servais

Contact us

Email: strongclinicaltrials@paediatrics.ox.ac.uk

IRAS number

1006080