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NCT04360265Phase 3ENROLLING BY INVITATION

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

Study 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).

Conditions
Mucopolysaccharidosis IIIAMPS IIIASanfilippo SyndromeSanfilippo A
Interventions / Drugs
No Investigational ProductAdjuvant Immunomodulatory (IM) Therapy
View on ClinicalTrials.gov ↗
Sponsor
RARE
Ultragenyx Pharmaceutical
Rare Disease
D49/100
Study Details
Enrollment41 participants
Primary Completion—
Lead SponsorUltragenyx Pharmaceutical Inc
Data Retrieved2026-04-11 04:25:35.045756+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Current status: ENROLLING BY INVITATION.

Enrolled: 41 participants
Sponsor Catalyst Profile

Ultragenyx Pharmaceutical (RARE) is the sponsoring company for this trial. BiotechSign currently grades this company D (49/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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