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Enrolling by Invitation

NCT Number: NCT06491927

Long Term Follow-up for RGX-202

RGX-202-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-202 in participants who have received RGX-202 (an investigative gene therapy designed to deliver a transgene for novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain) in a separate parent study.

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Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Sex eligibility

Male

Study type

Observational

Primary location

Arkansas Children's Hospital, Little Rock, Arkansas, United States

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About this study

This is a multicenter, prospective, observational study to evaluate the long-term safety and efficacy of investigational RGX-202. Eligible participants are those who have undergone evaluation in a previous (parent) clinical study following a single intravenous infusion of RGX-202 for the treatment of DMD. Enrollment in the current long-term follow-up (LTFU) study will occur after the participant has completed or discontinued from the parent study. Participants will be followed in this study cumulatively for up to 5 years after RGX-202 administration (inclusive of the parent study). No investigational treatment will be administered under this protocol. The total study duration for each participant may vary depending on when he enrolls in the current study following RGX-202 administration in the parent study.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • The parent(s) or legal guardian(s) of the participant has/(have) provided written informed consent and (where applicable) Health Insurance Portability and Accountability Act (HIPAA) authorization after the nature of the study has been explained, prior to any research-related procedures; and, where applicable, the minor participant has provided written or verbal assent according to local requirements.
  • Must have undergone evaluation in a previous clinical study following a single IV infusion of RGX-202 for the treatment of DMD and either completed or withdrawn early from that study.
  • Participant and parent(s)/legal guardian(s) are willing and able to comply with scheduled visits, and study procedures.
  • Sexually active participants must be willing to use a medically accepted method of contraception from the time of the screening visit through 5 years after RGX-202 administration.

Exclusion criteria

  • No exclusion criteria apply in this observational follow up study.

Treatment and study plan

No intervention

Genetic

Observational study

Primary outcomes

  1. Evaluation of long-term safety of RGX-202

    Time frame: Baseline, 5 years inclusive of parent study

    Incidences of AEs and SAEs over time

Secondary outcomes

  1. Time to Stand (TTSTAND)

    Time frame: Baseline, 5 years inclusive of parent study

    Values will include time (seconds) and velocity (tasks/second)

  2. Time to Run/Walk (TTRW)

    Time frame: Baseline, 5 years inclusive of parent study

    Values will include time (seconds) and velocity (meters/second)

  3. Time to Climb (TTCLIMB)

    Time frame: Baseline, 5 years inclusive of parent study

    Values will include time (seconds) and velocity (tasks/second)

  4. North Start Ambulatory Assessment (NSAA)

    Time frame: Baseline, 5 years inclusive of parent study

    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 .

  5. Peabody Developmental Motor Scale, Third Edition (PDMS-3); Body Control Subtest

    Time frame: Baseline, 5 years inclusive of parent study

    The PDMS-3 is a norm-referenced developmental assessment that measures motor skills of young children. The Body Control subtest measures the child's ability to maintain balance and postural reactions in a variety of positions. Motor skills appropriate for the child's developmental level are administered and rated 0, 1, or 2, with higher score indicating better performance. Body Control subtest total raw scores range from 0 to 112. Age equivalent and scaled scores will also be generated. PDMS-3 Body Control subtests is included for participants age <4 at study entry to the parent study.

  6. Peabody Developmental Motor Scale, Third Edition (PDMS-3); Body Transport Subtest

    Time frame: Baseline, 5 years inclusive of parent study

    The PDMS-3 is a norm-referenced developmental assessment that measures motor skills of young children. The Body Transport subtest measures the child's ability to move from one place to another, including walking, running, jumping forward, and skipping. Motor skills appropriate for the child's developmental level are administered and rated 0, 1, or 2, with higher score indicating better performance. Body Transport subtest total raw score ranges from 0 to 63. Age equivalent and scaled scores will also be generated. PDMS-3 Body Control subtests is included for participants age <4 at study entry to the parent study.

  7. Stride velocity 95th centile (SV95C)

    Time frame: Baseline, 5 years inclusive of parent study

    Assessment of peak ambulatory performance captured by wearable activity monitoring device. For velocity measures, higher values indicate greater function.

  8. Pharmacokinetics (PK)

    Time frame: Baseline, 5 years inclusive of parent study

    Vector genome concentrations as measured by polymerase chain reaction [PCR] to RGX-202 deoxyribonucleic acid [DNA] in muscle and serum.

  9. Vector Shedding

    Time frame: Baseline, 5 years inclusive of parent study

    Vector genome concentrations as measured by polymerase chain reaction [PCR] to RGX-202 deoxyribonucleic acid [DNA] in urine, feces, and saliva.

Sponsors and collaborators

Lead sponsor

REGENXBIO Inc.

Industry

Registry information

Official study title

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-202 Gene Therapy in Males With Duchenne Muscular Dystrophy (DMD)

Important dates

Study start
2024
Primary completion
2029
Study completion
2029
First posted
Jul 9, 2024
Registry last updated
Mar 24, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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