RGX-111 Gene Therapy in Patients With Mucopolysaccharidosis Type I (MPS I)

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This study will evaluate the safety and tolerability of RGX-111 in patients with Mucopolysaccharidosis Type I with a neurocognitive deficit that are at least 4 months old. This study involves administration of the RGX-111, general anesthesia, lumbar puncture, blood draws, MRI, ultrasound, electrocardiogram, echocardiogram, hearing test, and chart review.

ClinicalTrials.gov Identifier

NCT03580083

View full trial information on ClinicalTrials.gov

Eligibility & Criteria

IRB #:
19-016202
Official Title:
A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of Intracisternal RGX-111 Gene Therapy in Subjects with Mucopolysaccharidosis Type I
Study Phase:
Phase I
Phase II
Eligible Age Range:
4 - 99 Years
Gender:
All
Study Categories:
Leader
Can Ficicioglu

Can Ficicioglu, MD, PhD