Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
Doctors at the Nationwide Children’s Hospital in Columbus, OH, seek patients with Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome to trial an investigatory gene therapy, a virus engineered to transfer a functional SGSH gene to the patient, so that their bodies can process heparan sulfate normally.
Participants will undergo a single IV infusion of the investigatory gene therapy, along with a course of steroids, then be assessed at 6, 12, 18, and 24 months.
- Be 6 months of age or older
- Have confirmed diagnosis of MPS IIIA
- Meet specific genetic screening criteria regarding their type of MPS mutation
THE STUDY INVOLVES:
- Screening to confirm eligibility.
- Participants receive a single infusion of the investigatory gene therapy, as well as a steroid prescription.
- Lab and physical assessments will continue 24 months at 6-month intervals.
LOCATIONS AND CONTACTS:
The study site is at the Nationwide Children’s Hospital in Columbus, OH. Map.
Juan Ruiz, MD, PhD | +34 685895069 (international) | [email protected]
Abeona Therapeutics, Inc
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