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.
Study Type
OBSERVATIONAL
Enrollment
66
Observational study
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Stanford School of Medicine /Division of Neuromuscular Medicine
Palo Alto, California, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
The University of Texas Southwestern Medical Center
Dallas, Texas, United States
Evaluation of long-term safety of RGX-202
Incidences of AEs and SAEs over time
Time frame: Baseline, 5 years inclusive of parent study
Time to Stand (TTSTAND)
Values will include time (seconds) and velocity (tasks/second)
Time frame: Baseline, 5 years inclusive of parent study
Time to Run/Walk (TTRW)
Values will include time (seconds) and velocity (meters/second)
Time frame: Baseline, 5 years inclusive of parent study
Time to Climb (TTCLIMB)
Values will include time (seconds) and velocity (tasks/second)
Time frame: 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 .
Time frame: 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.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Virginia Commonwealth University
Richmond, Virginia, United States
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Baseline, 5 years inclusive of parent study