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

NCT04360265 · Status: ENROLLING_BY_INVITATION · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 41

Last updated 2026-04-24

No results posted yet for this 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 IIIA
  • MPS IIIA
  • Sanfilippo Syndrome
  • Sanfilippo A

Interventions

OTHER

No Investigational Product

No investigational product will be administered in this follow-up trial.

DRUG

Adjuvant Immunomodulatory (IM) Therapy

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 IM therapy.

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceutical Inc

Study Design

Allocation
NON_RANDOMIZED
Purpose
OTHER
Masking
NONE
Model
PARALLEL

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-09-28
Primary Completion
2027-08-31
Completion
2027-08-31
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Spain

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04360265 on ClinicalTrials.gov