RGX-202, one of the alternative gene therapy candidates developed for Duchenne Muscular Dystrophy, continues to accept applications from candidate participants for phase 3 clinical trials.
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A phase 3 clinical trial is being conducted by REGENXBIO to assess the safety, tolerability, and effectiveness of RGX-202, an experimental gene therapy for Duchenne muscular dystrophy (DMD). To replace the dystrophin protein that is absent from the muscles of males with DMD, RGX-202 is engineered to create a novel microdystrophin protein.
About Phase 3 Study of RGX-202
This is an open-label, multi-center, single-dose trial called AFFINITY DUCHENNE®. This implies that RGX-202, the research medication, will be administered once to all eligible individuals at various study locations. A one-time intravenous medication infusion and roughly 26 clinic visits are required for the two-year research. Following the administration of RGX-202, visits will increase in frequency and gradually decrease. Participants will be urged to sign up for a three-year, long-term follow-up study at the conclusion of the two-year period in order to track their health and the impact of RGX-202.
The medication will be given as an intravenous (in the vein) infusion once. The North Star Ambulatory Assessment [NSAA], timed function tasks (Time to Stand [TTStand], Time to Walk/Run 10 Meters [TTWR], and Time to Climb 4 STairs [TTClimb]), safety evaluations, and muscle biopsy to measure microdystrophin levels will all be used to assess the impact of RGX-202. The Peabody Developmental Motor Scale, Third Edition (PDMS-3) and the stride velocity (SV95C), a digital health technology assessment, will be used to evaluate participants under the age of four.
RGX-202 Phase 3 Clinical Trial – NCT05693142 –
This is a multicenter, open-label dose evaluation clinical study to assess the safety, tolerability, and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in participants with Duchenne.
Study Start (Estimated): 2023-01-04
Primary Completion (Estimated): 2026-02
Study Completion (Estimated): 2028-08
Number of Participants (Estimated): 65
Locations: USA, Canada.
Ages Eligible for Study: 1 Year and older (Child, Adult, Older Adult )
RGX-202 Phase 3 Participation Criteria
People must fulfill the inclusion requirements in order to be eligible. If a person has another ailment or is undergoing treatment that could prevent them from receiving gene therapy, they might not be able to participate. The complete set of inclusion and exclusion criteria can be found by clicking on this study link – NCT05693142 –. For qualified participants and caregivers, travel assistance is offered.
How Can I Contact with Regenxbio?
Please contact a patient navigator, email the REGENXBIO Patient Advocacy team at [email protected], or visit the trial website (www.regenxbiodmdtrials.com) for more information about the study or to inquire about participation.
Learn More: The Result of RGX-202 Affinity Duchenne® Trial Phase 1/2
