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

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

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 If you are interested in participating in the study or want to learn more, please contact: Contact us
This study is now recruiting. Learn more about enrolling here.

Description

This study will evaluate the safety andtolerability of RGX-111 in patients with Mucopolysaccharidosis Type I with aneurocognitive deficit that are at least 4 months old. This study involvesadministration of the RGX-111, general anesthesia, lumbar puncture, blooddraws, MRI, ultrasound, electrocardiogram, echocardiogram, hearing test, andchart review. If you have questions or would like to learn more about thestudy, please contact the Principal Investigator, Dr. Ficicioglu at (215)590-3376. 

Eligibility and criteria
IRB Number:
19-016202
Eligible age range:
4 months - 99
Clinical trial phase:
Phase I
Phase II
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
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