Mucopolysaccharidosis II
Conditions
Keywords
MPS II, Gene Therapy, Hunter
Brief summary
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.
Detailed description
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.
Interventions
OTHERLong-term Follow-Up
No intervention
Sponsors
REGENXBIO Inc.
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Healthy volunteers
No
Inclusion criteria
* To be eligible, a participant must have previously received RGX-121 in a separate parent trial. * Participant or participant's legal guardian(s) is/(are) willing and able to provide written, signed informed consent
Exclusion criteria
* Participant has not received RGX-121 previously in a separate parent trial.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Long-term Safety of RGX-121 | 5 years inclusive of parent study | To evaluate the safety of RGX-121 serious and adverse events as assessed by Common Terminology Criteria for Adverse Events (CTCAE Version 5.0). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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) | 5 years inclusive of parent study | 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. |
| 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) | 5 years inclusive of parent study | 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. |
| Evaluate the long-term effect of RGX-121 on neurodevelopmental parameters of cognitive, motor, behavioral, and adaptive function (Mullen Scales of Early Learning) | 5 years inclusive of parent study | 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. |
| Evaluate the long-term effect of RGX-121 on neurodevelopmental parameters of cognitive, motor, behavioral, and adaptive function (Vineland Adaptive Behavior Scales, 2nd Edition) | 5 years inclusive of parent study | 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 |
| Evaluate the long-term impact of RGX-121 on Plasma Biomarkers (I2S) | 5 years inclusive of parent study | 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). |
| Evaluate the long-term impact of RGX-121 on CSF Biomarkers (I2S) | 5 years inclusive of parent study | To evaluate the long-term impact of RGX-121 on CSF Biomarker Iduronate-2-sulfatase (I2S) through lumbar puncture. |
| Evaluate the long-term impact of RGX-121 on CSF Biomarkers (GAGs) | 5 years inclusive of parent study | To evaluate long-term impact of RGX-121 on CSF Biomarker Glycosaminoglycans (GAGs) through lumbar puncture. |
| Long-term effect of RGX-121 on Urine Biomarker (GAGs) | 5 years inclusive of parent study | To evaluate the long-term impact of RGX-121 on Urine Biomarker Glycosaminoglycans (GAGs) through urine analysis. |
Countries
Brazil, United States
Outcome results
None listed