RGX-121-5101 is an observational long-term follow-up study to the RGX-121-101 or RGX-121-1102 studies where participants received RGX-121, a gene therapy intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study will evaluate the long-term safety and efficacy of RGX-121.
This is a prospective follow-up study to evaluate the long-term safety and efficacy after a single administration of RGX-121. Eligible participants are those who previously have enrolled in clinical study RGX-121-101 or RGX-121-1102 and received a single intracisternal (IC) or intracerebroventricular (ICV) infusion of RGX-121. Enrollment of each participant will occur the same day or after the participant has completed the end of study (EOS) visit or early termination visit (ET) from the previous (parent) study. Participants will be followed in this study cumulatively for up to 5 years after RGX-121 administration (inclusive of the parent study). No treatment will be administered under this observational protocol. The total study duration for each participant may vary depending on when they enroll in the current study following RGX-121 administration in the parent study.
Study Type
OBSERVATIONAL
Enrollment
27
No intervention
University of California, San Francisco (UCSF)- Benioff Children's Hospital
San Francisco, California, United States
Saint Peter's Healthcare System
New Brunswich, New Jersey, United States
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States
Hospital de Clinicas de Porto Alegre
Porto Alegre, Brazil
Long-term Safety of RGX-121
To evaluate the safety of RGX-121 serious and adverse events as assessed by Common Terminology Criteria for Adverse Events (CTCAE Version 5.0).
Time frame: 5 years inclusive of parent study
Evaluate the long-term effect of RGX-121 on neurodevelopmental parameters of cognitive, motor, behavioral, and adaptive function (Bayley Scales of Infant and Toddler Development, 3rd Edition)
To evaluate the long-term effect of RGX-121 on neurodevelopmental parameters as measured by the BSID-III. The BSID-III assesses children aged 1 to 42 months across several domains (cognitive, language, and motor domains) to give a holistic view of the participants' development and potential delays.
Time frame: 5 years inclusive of parent study
Evaluate the long-term effect of RGX-121 on neurodevelopmental parameters of cognitive, motor, behavioral, and adaptive function (Kaufman Assessment Battery for Children, 2nd Edition)
To evaluate the long-term effect of RGX-121 on neurodevelopmental parameters as measured by the KABC-II. The KABC-II is an individually administered measure of the cognitive processing abilities of children and adolescents aged 3 through 18.
Time frame: 5 years inclusive of parent study
Evaluate the long-term effect of RGX-121 on neurodevelopmental parameters of cognitive, motor, behavioral, and adaptive function (Mullen Scales of Early Learning)
To evaluate the long-term effect of RGX-121 on neurodevelopmental parameters as measured by the MSEL. The MSEL is a standardized developmentally integrated system that assesses language, motor, and perceptual abilities, and measures cognitive ability and motor development.
Time frame: 5 years inclusive of parent study
Evaluate the long-term effect of RGX-121 on neurodevelopmental parameters of cognitive, motor, behavioral, and adaptive function (Vineland Adaptive Behavior Scales, 2nd Edition)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
To evaluate the long-term effects of RGX-121 on neurodevelopmental parameters as measured by the VABS-II. The VABS-II assesses adaptive behavior in individuals from infancy to age 90 years. The VABS-II offers a way to measure personal and social self-sufficiency in real-life situations and to observe how these cognitive abilities impact the autonomy management process when put into practice
Time frame: 5 years inclusive of parent study
Evaluate the long-term impact of RGX-121 on Plasma Biomarkers (I2S)
To evaluate the long-term impact of RGX-121 on Plasma biomarker Iduronate-2-sulfatase (I2S) through plasma collection for participants not receiving ERT (discontinued or naïve).
Time frame: 5 years inclusive of parent study
Evaluate the long-term impact of RGX-121 on CSF Biomarkers (I2S)
To evaluate the long-term impact of RGX-121 on CSF Biomarker Iduronate-2-sulfatase (I2S) through lumbar puncture.
Time frame: 5 years inclusive of parent study
Evaluate the long-term impact of RGX-121 on CSF Biomarkers (GAGs)
To evaluate long-term impact of RGX-121 on CSF Biomarker Glycosaminoglycans (GAGs) through lumbar puncture.
Time frame: 5 years inclusive of parent study
Long-term effect of RGX-121 on Urine Biomarker (GAGs)
To evaluate the long-term impact of RGX-121 on Urine Biomarker Glycosaminoglycans (GAGs) through urine analysis.
Time frame: 5 years inclusive of parent study