recruiting

Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

key information

study id #: NCT02716246

condition: MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III

status: Recruiting

purpose:

Open-label, dose-escalation clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein

intervention: ABO-102

results: https://clinicaltrials.gov/ct2/show/results/NCT02716246

last updated: February 26, 2022