clinical trial · NCT02716246

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

$RARE →·phase3·recruiting·n = 36

MPS IIIASanfilippo SyndromeSanfilippo AMucopolysaccharidosis IIIUX111Prophylactic Immunomodulatory (IM) TherapyOptimized Prophylactic IM TherapyAdjuvant IM Therapy

brief summary

The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.

started

Apr 25, 2016

primary completion

Jul 1, 2027

completion

Jul 1, 2027

last updated

Apr 24, 2026

detailed description

Open-label, single dose, dose-escalation clinical trial of UX111 (scAAV9.U1a.hSGSH) injected intravenously through a peripheral limb vein. A limited course of prophylactic immunomodulatory (IM) therapy will be administered. At approved sites adjuvant IM therapy may be administered to selected participants. The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IM therapy. Not all participants may receive adjuvant IM therapy.

This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.

official title

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA

full record on clinicaltrials.gov ↗

sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol