RGX-111 Gene Therapy in Patients With Mucopolysaccharidosis Type I (MPS I)
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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:
Clinical trial phase:
Phase I
Phase II
Official title:
Related specialties
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