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 IIIA MPS IIIA Sanfilippo Syndrome Sanfilippo A

Interventions / Drugs

No Investigational Product Adjuvant Immunomodulatory (IM) Therapy

View on ClinicalTrials.gov ↗

Sponsor

RARE

Ultragenyx Pharmaceutical

Rare Disease

C63/100

Study Details

Enrollment41 participants

Primary Completion—

Lead SponsorUltragenyx Pharmaceutical Inc

Data Retrieved2026-04-11 04:25:35.045756+00:00

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