RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome)

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The purpose of this study is to look at the safety and tolerability of RBX-121, a one-time gene therapy, in subjects with MPS II (Hunter Syndrome). You may be eligible for this study if you are a male between the ages of 4 months to 5 years old. Reimbursement for travel and research procedures will be provided. This study involves blood draws, interviews, urine collection, genetic testing, general anesthesia, lumbar punctures, MRI, gene therapy, lumbar puncture, ultrasound, ECG/echocardiogram and physical and neurological examinations. If you have questions or would like to learn more about the study, please contact the study team at [email protected].

Who Do I Contact?

If you are interested in participating in the study or want to learn more please contact our study team at .

ClinicalTrials.gov Identifier

NCT03566043

View full trial information on ClinicalTrials.gov

Eligibility & Criteria

IRB #:
18-015617
Official Title:
A Phase I/II Multicenter, Open-Label Study To Evaluate The Safety, Tolerability, and Pharmacodynamics Of RGX-121 In Pediatric Subjects with MPS II (Hunter Syndrome)
Study Phase:
Phase I
Phase II
Eligible Age Range:
4 Months - 5 Years
Gender:
Male
Study Categories:
Leader
Can Ficicioglu

Can Ficicioglu, MD, PhD