RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain.
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.
For additional information on how to participate (or be considered for the study), please follow this link: https://mytomorro.ws/affinity-ct-gov
The participant's legal guardian(s) is (are) willing and able to provide written, signed informed consent prior to any study-related procedures; and, where applicable, the minor participant has provided written or verbal assent according to local requirements.
Is a male at least 4 years of age and less than 12 years of age at consent or 1 to <4 years of age at the time of dosing and ≥ 10 kg at the time of screening.
DMD gene mutation in exons 18 and above, and a clinical picture consistent with typical DMD with the exception of a participant (Cohort 1b) with DMD gene mutation in exons 12-17.
Participant is able to walk 100 meters independently without assistive devices. Cohort 2c participant must be able to walk 10 meters independently without assistive devices. Cohort 1b participant must be able to walk with or without assistive devices.
Participant has any condition that would contraindicate treatment with immunosuppression.
Participant has received ataluren (a protein restoration therapy) or an exon-skipping therapy for the treatment of DMD within 6 months of study entry or is unable to refrain from taking ataluren or exon-skipping therapy for a duration of 5 years from the time of RGX-202 administration.
Participant has received any investigational or commercial gene therapy product over his lifetime.
Participant is currently taking any other investigational intervention (other than corticosteroids) or has taken any other investigational intervention (other than corticosteroids) within 3 months prior to the scheduled Day 1 intervention. If your corticosteroid is vamorolone, the participant will be asked to temporarily convert his daily dosing to prednisolone/prednisone during a short period of time around RGX-202 administration. He will be allowed to revert back to his baseline vamorolone regimen at the original per kilogram dose at which he entered the study and should remain on this for 24 months unless the investigator determines that this is not clinically indicated or possible.
Parallel
RGX-202
GeneticRGX-202 is a recombinant AAV8 containing a transgene encoding a novel microdystrophin
Part 1 Safety measured by incidence of Adverse Events and Serious Adverse Events
Evaluate incidences of AEs and SAEs
Part 2 and 3 Pharmacodynamic
Proportion of participants whose RGX-202 microdystrophin protein expression determined in their muscle biopsy is ≥ 10%
Time to Stand (TTSTAND)
Values will include time (seconds) and velocity (tasks/second)
Time to Walk/Run 10 meters (TTWR)
Values will include time (seconds) and velocity (meters/second)
Time to Climb 4 Stairs (TTCLIMB)
Values will include time (seconds) and velocity (tasks/second)
North Star Ambulatory Assessment (NSAA)
Performance-based assessment of muscle strength and function using a 17-item scale, with all items rated 0,1, or 2, with higher score indicating better performance. The NSAA total score is the sum of the 17 items, ranging from 0 to 34. NSAA linearized score ranges from 0 to 100.
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