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

Contact

If you are interested in participating in the study or want to learn more, please get in touch.

This study is no longer recruiting.

Description

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.

Eligibility and criteria

IRB Number:

19-016202

Eligible age range:

4 months - 99 years

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 - (NCT03580083)

What to expect

Related specialties

Metabolic Disease Program

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