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Duchenne Muscular DystrophyNCT06491927

Long Term Follow-up for RGX-202

An observational study of Duchenne Muscular Dystrophy, run by REGENXBIO Inc. at 5 sites in United States.

Enrolling by invitation

Enrolling by invitation. The study team selects and invites participants, usually from an existing group of patients.

Sites and contacts
Official record
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Trial summary

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… Read more about this study

Who is it for?
Any age, men only
Conditions the study is for
Duchenne Muscular Dystrophy
How many people
66 participants
Where
5 sitesUnited States
Study dates
8 May 2024to 1 Dec 2029
Phase
Observational
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • Men only
  • No healthy volunteers
  • Child, Adult, Older adult

Study population: Received RGX-202 in a separate parent study.

Who may be able to join

  • 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in a previous clinical study following a single IV infusion of RGX-202 for the treatment of DMD and either completed or withdrawn early from that study.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.

Who cannot join

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

Trial locations and contacts

5 sites in 1 country. Contact the study team or a site near you to ask about taking part.

5 sites in 1 country

United States5 sites

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

  • The University of Texas Southwestern Medical Center

    Dallas, Texas, 75390

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202

  • Stanford School of Medicine /Division of Neuromuscular Medicine

    Palo Alto, California, 94304

  • Virginia Commonwealth University

    Richmond, Virginia, 23298

Other trials in United States

About this study

Official 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)

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.

Read the detailed description

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.

Being tested
No Intervention
Keywords
Gene therapy, DMD, Duchenne, Duchenne Muscular Dystrophy
Medical subject headings
Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • No InterventionGenetic

    Observational study

Study groups in NCT06491927
Study groupRoleWhat they receive
RGX-202 RecipientsSubjects who have received RGX-202 in a separate parent study.
Receives: Genetic: No Intervention

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    Incidences of AEs and SAEs over time

    Measured: Baseline, 5 years inclusive of parent study

Secondary outcomes (9)

  1. Time to Stand (TTSTAND)

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

    Measured: Baseline, 5 years inclusive of parent study

  2. Time to Run/Walk (TTRW)

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

    Measured: Baseline, 5 years inclusive of parent study

  3. Time to Climb (TTCLIMB)

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

    Measured: Baseline, 5 years inclusive of parent study

  4. North Start 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 .

    Measured: Baseline, 5 years inclusive of parent study

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

    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.

    Measured: Baseline, 5 years inclusive of parent study

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

    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.

    Measured: Baseline, 5 years inclusive of parent study

  7. Stride velocity 95th centile (SV95C)

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

    Measured: Baseline, 5 years inclusive of parent study

  8. Pharmacokinetics (PK)

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

    Measured: Baseline, 5 years inclusive of parent study

  9. Vector Shedding

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

    Measured: Baseline, 5 years inclusive of parent study

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Jul 2024
  2. Study start8 May 2024
  3. Primary completion1 Dec 2029 (estimated)
  4. Study completion1 Dec 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Other
Time perspective
Prospective
Enrolment
66 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06491927
Sponsor's protocol ID
RGX-202-5101
Status last verified
Mar 2026
First submitted
1 Jul 2024
Last updated
24 Mar 2026
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

An observational study of Duchenne Muscular Dystrophy, run by REGENXBIO Inc. at 5 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: enrolling by invitation, as of the sponsor's last update on 24 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Evaluation of long-term safety of RGX-202 (time frame: Baseline, 5 years inclusive of parent study). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06491927.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Mar 2026.

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