RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome)
A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)
Doctors are seeking male participants aged 4 months to 5 years old for a trial gene therapy to treat severe Hunter’s Syndrome. This gene therapy will be delivered directly to the brain, using a harmless virus as a way to transport a normal gene to replace the mutated gene that causes Hunter’s Syndrome.
Study participants will undergo a single dose of the trial therapy and be closely observed for the first 24 weeks post the treatment, with re-assessments at 48, 78, and 104 weeks. The researchers hope to see patients begin to normally produce the iduronate-2-sulfatase enzymes lacking due to their disease.
- Be 4 months to 5 years old and male
- Must meet any of the following criteria:
- Has a documented diagnosis of MPS II and a has a neurocognitive testing score > 55 and ≤ 77 (Bayley or Kaufman), OR
- Has a documented diagnosis of MPS II AND has a decline of ≥ 1 standard deviation on serial neurocognitive testing (Bayley or Kaufman) and a testing score > 55, OR
- Has a relative diagnosed with severe MPS II who has the same IDS mutation as the subject AND in the opinion of a geneticist has inherited a severe form of MPS II
THE STUDY INVOLVES:
- Screening before the treatment
- Under anesthesia, a CT scan-guided injection to the spinal cord will deliver the treatment to the brain, inside the blood-brain barrier
- The patients will be observed closely for 24 weeks, then have visits at 48, 78, and 104 weeks.
LOCATIONS AND CONTACTS:
Trials will take place at the Children’s Hospital of Pittsburgh – UPMC: Program for Neurodevelopment in Rare Disorders. Map.
Contact: Jacob Wesley, Pharm D, MS | [email protected] | 240-552-8181
Contact: Jodi Martin | 412-692-6351 | [email protected]
Contact: Dr. Maria Escolar | [email protected]
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