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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| RGX-202 Recipients | Subjects who have received RGX-202 in a separate parent study. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| No Intervention | Genetic | Observational study |
|
| Measure | Description | Time Frame |
|---|---|---|
| Evaluation of long-term safety of RGX-202 | Incidences of AEs and SAEs over time | Baseline, 5 years inclusive of parent study |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Stand (TTSTAND) | Values will include time (seconds) and velocity (tasks/second) | Baseline, 5 years inclusive of parent study |
| Time to Run/Walk (TTRW) | Values will include time (seconds) and velocity (meters/second) |
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Inclusion Criteria:
Exclusion Criteria:
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Received RGX-202 in a separate parent study.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | United States | ||
| Stanford School of Medicine /Division of Neuromuscular Medicine |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| Baseline, 5 years inclusive of parent study |
| Time to Climb (TTCLIMB) | Values will include time (seconds) and velocity (tasks/second) | Baseline, 5 years inclusive of parent study |
| 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 . | Baseline, 5 years inclusive of parent study |
| 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. | Baseline, 5 years inclusive of parent study |
| 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. | Baseline, 5 years inclusive of parent study |
| Stride velocity 95th centile (SV95C) | Assessment of peak ambulatory performance captured by wearable activity monitoring device. For velocity measures, higher values indicate greater function. | Baseline, 5 years inclusive of parent study |
| Pharmacokinetics (PK) | Vector genome concentrations as measured by polymerase chain reaction [PCR] to RGX-202 deoxyribonucleic acid [DNA] in muscle and serum. | Baseline, 5 years inclusive of parent study |
| Vector Shedding | Vector genome concentrations as measured by polymerase chain reaction [PCR] to RGX-202 deoxyribonucleic acid [DNA] in urine, feces, and saliva. | Baseline, 5 years inclusive of parent study |
| Palo Alto |
| California |
| 94304 |
| United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| The University of Texas Southwestern Medical Center | Dallas | Texas | 75390 | United States |
| Virginia Commonwealth University | Richmond | Virginia | 23298 | United States |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |