NCT04360265

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

A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)

• Status: Enrolling By Invitation
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 41 (estimated)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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).

Detailed description

This is a multicenter, long-term follow-up study of patients with MPS IIIA who have participated in a prior clinical trial involving the administration of UX111 (NCT02716246 and NCT04088734). No investigational product will be provided. At approved sites adjuvant immunomodulatory (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 IM therapy. This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.

Conditions

• Mucopolysaccharidosis IIIA
• MPS IIIA
• Sanfilippo Syndrome
• Sanfilippo A

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2020-09-28
• Primary completion: 2027-08-01
• Completion: 2027-08-01
• First posted: 2020-04-24
• Last updated: 2026-01-21

Locations

4 sites across 3 countries: United States, Australia, Spain

Regulatory

• FDA-regulated drug study