AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
NCT ID: NCT05693142
Last Updated: 2025-11-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2/PHASE3
65 participants
INTERVENTIONAL
2023-01-04
2028-08-31
Brief Summary
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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
Detailed Description
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This is a multicenter, phase I/II/III, open-label study to evaluate the safety, tolerability, pharmacodynamics (microdystrophin protein levels), pharmacokinetic, and clinical efficacy of RGX-202 when administered IV as one-time dose to ambulant male participants with Duchenne. A comprehensive, short-term, prophylactic immunosuppression regimen will be administered during treatment to mitigate a potential immune response. This study is being conducted in three sequential parts: a phase I/II study (Part 1), a phase 3 pivotal study (Part 2) and a confirmatory study (Part 3). Part 1 will study a one-time dose of RGX-202 (1x10\^14 or 2x10\^14 GC/kg) in up to 15 participants with Duchenne. In part 1, the primary objective is to evaluate the safety and tolerability of RGX-202 through 52 weeks. Part 2 (Pivotal Expansion) will study a single dose of RGX-202 (2x10\^14 GC/kg) in approximately 30 participants. After the last Part 2 participant is dosed, enrollment into the confirmatory study (Part 3) will be initiated. The target enrollment for the confirmatory study (Part 3) is approximately 30 participants. Participants will be assessed at various time points for 104 weeks after receiving RGX-202. All participants will be given the opportunity to enroll in a separate long-term follow-study in accordance with the US federal government guidelines for the safety follow-up of patients receiving gene therapy.
Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Part 1: Cohort 1 and 1b: RGX-202 Dose 1
A single IV infusion of RGX-202 at a dose of 1×10\^14 GC/kg body weight
RGX-202
RGX-202 is a recombinant AAV8 containing a transgene encoding a novel microdystrophin
Part 1: Cohort 2, 2c;, and Part 2; and Part 3: RGX-202 Dose 2
A single IV infusion of RGX-202 at a dose of 2x10\^14 GC/kg body weight
RGX-202
RGX-202 is a recombinant AAV8 containing a transgene encoding a novel microdystrophin
Interventions
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RGX-202
RGX-202 is a recombinant AAV8 containing a transgene encoding a novel microdystrophin
Eligibility Criteria
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Inclusion Criteria
* 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.
* Must meet any of the following criteria:
* 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 is able to complete the TTSTAND per protocol-specific criteria.
* Participant has been on a stable dose of systemic glucocorticoids according to the standard of care for at least 12 weeks. Cohort 2c participants must be consistently on or off a stable dose of systemic glucocorticoids according to the standard of care for at least 12 weeks.
* Clinical laboratory test results, including hepatic and renal function, are within the normal range during screening, or if abnormal, are not clinically significant, in the opinion of the investigator.
* Documentation is provided at screening visit for participant's adherence to the local country's vaccination schedule. The parent(s) or legal guardian(s) must be willing to have their child receive a meningococcal vaccine, if not already vaccinated.
* Participant and parent(s)/legal guardian(s) are willing and able to comply with scheduled visits, study intervention administration plan, and study procedures.
* 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.
* DMD gene mutation with any mutation except for those with deletions or point mutations in exons 8, 9 and/or 10.
* Participant is able to complete the TTSTAND per protocol-specific criteria.
* Clinical laboratory test results, including hepatic and renal function, are within the normal range during screening, or if abnormal, are not clinically significant, in the opinion of the investigator.
* Documentation is provided at screening visit for participant's adherence to the local country's vaccination schedule. The parent(s) or legal guardian(s) must be willing to have their child receive a meningococcal vaccine, if not already vaccinated.
* Participant and parent(s)/legal guardian(s) are willing and able to comply with scheduled visits, study intervention administration plan, and study procedures.
* Is a male at least 1 year of age and ≥ 10 kg at the time of screening.
* Participants 1 to \<4 years of age must meet the following criteria:
* is able to walk 10 meters independently without assistive devices.
* must be consistently on or off a stable dose of systemic glucocorticoids according to the standard of care for at least 12 weeks.
* Participants 4 years and older must meet the following criteria:
* are able to walk 100 meters independently without assistive devices.
* have been on a stable dose of systemic glucocorticoids according to the standard of care for at least 12 weeks.
* have a NSAA total score ≥16.
Exclusion Criteria
* 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.
* Participant has impaired cardiac function defined as a left ventricular ejection fraction of \< 55% on screening cardiac assessments (echocardiogram or MRI).
* Participant is not a good candidate for the study, in the opinion of the investigator.
* Participant has any condition that would contraindicate treatment with immunosuppression.
* Participant has received givinostat within 3 months of study entry or 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.
* Participant has detectable AAV8 total binding antibodies in serum.
* Participant has impaired cardiac function defined as a left ventricular ejection fraction of \< 55% on screening cardiac assessments echocardiogram or MRI).
* Participant is not a good candidate for the study, in the opinion of the investigator.
1 Year
MALE
No
Sponsors
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REGENXBIO Inc.
INDUSTRY
Responsible Party
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Locations
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Arkansas Children's Hospital
Little Rock, Arkansas, United States
Stanford School of Medicine /Division of Neuromuscular Medicine
Palo Alto, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Rare Disease Research
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Iowa
Iowa City, Iowa, United States
University of Massachusetts Chan Medical School
Worcester, Massachusetts, United States
Cincinnati Children's
Cincinnati, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
The University of Texas Southwestern Medical Center
Dallas, Texas, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States
Children's Hospital of Richmond at Virginia Commonwealth University
Richmond, Virginia, United States
Alberta Children's Hospital
Calgary, Alberta, Canada
BC Children's Hospital
Vancouver, British Columbia, Canada
Children's Hospital London Health Science Centre
London, Ontario, Canada
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada
Countries
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Central Contacts
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Facility Contacts
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Amber Evans
Role: primary
Role: primary
Michele Yang
Role: primary
Hannah Kleiner
Role: backup
Lily Goggans
Role: primary
Maureen Ikpeoha
Role: backup
Nicole Geanous
Role: primary
Laura Knosp
Role: primary
Tyler Mola
Role: primary
Angela Edmondson
Role: primary
Beata Dyar
Role: primary
Elaine Most
Role: primary
Erika Paradiso
Role: primary
Falgun Patel
Role: primary
Israt Yasmeen, MBT
Role: primary
Nela Martic
Role: primary
Rhiannon Hicks
Role: primary
Laura Thompson
Role: primary
Related Links
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For additional information on how to participate (or be considered for the study) please follow this link.
Related Info
Other Identifiers
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RGX-202-1101
Identifier Type: -
Identifier Source: org_study_id