RGX-111 Gene Therapy in Patients With MPS I
A Phase I Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-111 in Subjects With MPS I
Doctors at Children’s Hospital of Philadelphia and the University of Pennsylvania seek patients with Mucopolysaccharidosis type I (MPS I) to trial therapeutic gene therapy. The therapy uses a harmless virus to deliver a deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system, to correct its deficiency in MPS I patients.
Study participants will receive the investigatory treatment via a single infusion to the central nervous system while under anesthesia. Follow up will require lab testing every few weeks for 2 years, with visits less frequent in the second year.
- Be 6years of age or older
- Have documented evidence of early- stage neurocognitive deficit due to MPS I
- Be able to receive immunosuppressive therapy in conjunction with the investigative therapy
THE STUDY INVOLVES:
- Screening to confirm acceptance to the study.
- Injection of the therapy, under anesthesia, guided by CT into the spinal fluid.
- Close observation immediately after the treatment.
- Follow up for 2 years with weekly, then monthly check-ins, spacing out to twice a year.
LOCATIONS & CONTACTS:
The trial will take place at the Children’s Hospital of Philadelphia, PA. Map.
Patient Advocacy | 866-860-0117 | [email protected]
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