RGX-202 for Duchenne Muscular Dystrophy
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to assess the long-term safety and effectiveness of a gene therapy called RGX-202, designed to help individuals with Duchenne Muscular Dystrophy (DMD). RGX-202 delivers a special protein that mimics a natural one missing in people with DMD. The trial specifically includes participants who previously received RGX-202 in an earlier study. Those with DMD who participated in that study may find this follow-up trial suitable for continued progress monitoring. As a Phase 4 trial, the study focuses on understanding how the already FDA-approved RGX-202 benefits more patients, offering participants a chance to contribute to broader knowledge while continuing treatment.
Do I need to stop my current medications for this trial?
The trial information does not specify whether you need to stop taking your current medications. It is best to discuss this with the trial coordinators or your doctor.
What is the safety track record for RGX-202?
Earlier studies found that RGX-202 was well tolerated by participants. Specifically, 13 trial participants did not experience any serious side effects, nor were there side effects requiring special attention. This suggests that RGX-202 is safe for humans so far. However, RGX-202 remains under study and has not yet received FDA approval. Further research is necessary to fully confirm its safety and effectiveness.12345
Why are researchers enthusiastic about this study treatment?
RGX-202 is unique because it uses a gene therapy approach to address underlying genetic issues, potentially providing a more long-lasting solution compared to current standard treatments, which might include enzyme replacement therapies or small molecule drugs. Unlike traditional therapies that often require frequent dosing, RGX-202 could offer more sustained benefits with less frequent administration. Researchers are excited about this treatment because it targets the root cause of the condition at the genetic level, which may lead to improved outcomes and a better quality of life for patients.
What evidence suggests that RGX-202 might be an effective treatment for dystrophin-related conditions?
Research has shown that RGX-202 could help treat Duchenne muscular dystrophy (DMD). In early studies, patients who received RGX-202 had microdystrophin levels at 31.5% compared to those who did not receive the treatment. This is important because microdystrophin helps muscles function better. Additionally, RGX-202 demonstrated positive effects on disease progression, improving muscle function in participants. Overall, early results suggest RGX-202 might improve outcomes for people with DMD.14678
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Long-term Follow-up
Participants are monitored for long-term safety and efficacy of RGX-202 after receiving the gene therapy in a parent study
Safety Monitoring
Evaluation of long-term safety through incidences of AEs and SAEs
What Are the Treatments Tested in This Trial?
Interventions
- RGX-202
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Subjects who have received RGX-202 in a separate parent study.
Find a Clinic Near You
Who Is Running the Clinical Trial?
REGENXBIO Inc.
Lead Sponsor
Citations
1.
ir.regenxbio.com
ir.regenxbio.com/news-releases/news-release-details/regenxbio-reports-positive-biomarker-data-affinity-duchennerREGENXBIO REPORTS POSITIVE BIOMARKER DATA ...
In a patient aged 7 years old, RGX-202 microdystrophin expression was measured to be 31.5% compared to control. In all patients, RGX-202 was ...
2.
regenxbio.gcs-web.com
regenxbio.gcs-web.com/news-releases/news-release-details/regenxbio-reports-new-positive-functional-data-phase-iiiREGENXBIO REPORTS NEW POSITIVE FUNCTIONAL DATA ...
RGX-202 demonstrating consistent evidence of positively changing disease trajectory for Duchenne. All dose level 2 participants exceeded ...
3.
regenxbio.com
regenxbio.com/getattachment/235b264f-bf42-4278-b4f6-0f20735e0026/WMS-2025_RGX-202_Poster_vFinal.pdf?lang=en-US&ext=.pdfRGX-202, an Investigational Gene Therapy for the ...
• Designed for consistent, improved safety outcomes ... RGX-202: AFFINITY DUCHENNE New Phase I/II Interim Functional Data, June 2025.
RGX-202 Gene Therapy Improves DMD Trajectory ... - AJMC
RGX-202 shows promising efficacy in Duchenne muscular dystrophy, improving functional outcomes and biomarker expression in early trial results.
5.
parentprojectmd.org
parentprojectmd.org/regenxbio-initiates-pivotal-phase-of-affinity-duchenne-trial-of-rgx-202-gene-therapy-reports-positive-functional-data/REGENXBIO Initiates Pivotal Phase of AFFINITY ...
RGX-202 uses a modified AAV to deliver a shortened dystrophin gene, showing positive functional data and stable or improved function in the ...
6.
regenxbio.com
regenxbio.com/getmedia/e16fcfde-2e79-4749-b2b6-da308267afa0/MDA-AFFINITY-DUCHENNE-2025.pdf?ext=.pdfRGX-202, an investigational gene therapy for the treatment ...
RGX-202 is an investigational product that has not been approved by the FDA. No conclusions regarding safety and efficacy can be made. Page 2. 2. RGX- ...
7.
parentprojectmd.org
parentprojectmd.org/regenxbio-shares-positive-functional-data-from-phase-i-ii-affinity-duchenne-trial-of-rgx-202/REGENXBIO Shares Positive Functional Data from Phase I ...
Safety. RGX-202 was well tolerated in 13 study participants across both dose levels with no serious adverse events (SAEs) and no AEs of special ...
NCT05693142 | AFFINITY DUCHENNE: RGX-202 Gene ...
This is a multicenter, phase I/II/III, open-label study to evaluate the safety, tolerability, pharmacodynamics (microdystrophin protein levels), pharmacokinetic ...
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