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. If you have questions or would like to learn more about the study, please contact the Principal Investigator, Dr. Ficicioglu at 215-590-3376.

Who Do I Contact?

If you are interested in participating in the study or want to learn more please contact our study team at morrisa6 [at] chop.edu.

Eligibility & Criteria

IRB #:
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
Study Categories: