Clinical Trial

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

Enrolling by Invitation Phase 3
View on ClinicalTrials.gov →
Summary
The main objective of this study is to evaluate the safety/tolerability and efficacy of UX111 (previously known as ABO-102) in participants with Mucopolysaccharidosis IIIA (MPS IIIA).
Protocol Amendment History 20 amendments
This ClinicalTrials.gov record has been amended 20 times since 2020-04-21; most recent amendment 2026-07-16.
Status change: Recruiting → Enrolling by Invitation 2022-08-11
Status change: Not Yet Recruiting → Recruiting 2020-10-26
Trial Details
NCT Number NCT04360265
Lead Sponsor Ultragenyx Pharmaceutical Inc
Conditions Mucopolysaccharidosis IIIA, MPS IIIA, Sanfilippo Syndrome, Sanfilippo A
Enrollment 41 participants
Start Date 2020-09-28
Primary Completion 2027-08 (estimated)
Study Completion 2027-08 (estimated)
Updated on ClinicalTrials.gov 2026-07-17