Clinical Research Alert: Phase 3 Study of RGX-202 in Boys with DMD

REGENXBIO Inc. is enrolling boys living with Duchenne muscular dystrophy (DMD) to participate in a phase 3 clinical trial to evaluate the safety, tolerability and efficacy of the investigational gene therapy RGX-202 to treat DMD. RGX-202 is designed to produce a novel microdystrophin protein to replace the missing dystrophin protein in the muscles of males with DMD.

The study

This study, known as AFFINITY DUCHENNE®, is an open-label, multi-center, single-dose study. This means that the study will take place at multiple study sites and that all eligible participants will receive a single dose of the study drug, RGX-202. The study will last approximately two years, and will require a one-time intravenous drug infusion and approximately 26 clinic visits. Visits will be more frequent immediately following RGX-202 administration and become less frequent over time. At the end of two years, participants will be encouraged to enroll in a three-year, long-term follow-up study to monitor the participant’s health and the effects of RGX-202.

The drug will be administered as a one-time, intravenous (in the vein) infusion. The effect of RGX-202 will be evaluated by various tests, including muscle biopsy to determine microdystrophin levels, safety assessments, and timed function tasks (Time to Stand [TTStand], Time to Walk/Run 10 Meters [TTWR], and Time to Climb 4 STairs [TTClimb], and the North Star Ambulatory Assessment [NSAA]). Participants younger than 4-years-old will be assessed using the Peabody Developmental Motor Scale, 3rd Edition (PDMS-3), and a digital health technology measurement, the stride velocity (SV95C).

Study criteria

To be eligible, individuals must meet the inclusion criteria. Individuals may not be eligible to participate if they are affected by another illness or receiving another treatment that might interfere with the ability to receive gene therapy.

Please visit this study link for the full listing of inclusion and exclusion criteria.

Travel support is available for eligible participants and caregivers.

Interested in participating?

To learn more about the study or inquire about participation, please visit REGENXBIO’s trial website (www.regenxbiodmdtrials.com), email the REGENXBIO Patient Advocacy team at duchenne@regenxbio.com, or connect with a patient navigator.