Clinical trial NCT06491927: Long Term Follow-up for RGX-202
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)
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Duchenne Muscular Dystrophy
Keywords: Gene therapy
DMD
Duchenne
Duchenne Muscular Dystrophy
Lead sponsor: REGENXBIO Inc.
Sponsor class: INDUSTRY
First posted: 2024-07-09
Enrollment: 19
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Received RGX-202 in a separate parent study.

Brief 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 (a 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.

Detailed description: This is a multicenter, prospective, observational study to evaluate the long-term safety and efficacy of 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Evaluation of long-term safety of RGX-202
timeFrame: 5 years inclusive of parent study
Secondary outcomes:
measure: Efficacy measured by change in Functional Assessment
timeFrame: 5 years inclusive of parent study
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
Study officials:
References: