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

Enrolling by invitation · Phase 3

Conditions studied: Mucopolysaccharidosis IIIA, MPS IIIA, Sanfilippo Syndrome, Sanfilippo A

In brief

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).

Key facts

Study ID

NCT04360265

Run by

Ultragenyx Pharmaceutical Inc

People needed

41

Starts

2020-09-28

Expected to finish

2027-08-01

Last updated by the study team

2026-07-17

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Participants that have participated in a prior clinical trial in which they received UX111
• Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule

You may not qualify if…

• Planned or current participation in another clinical trial that may confound the safety or efficacy evaluation of UX111 during this study
• Any other situation or medical condition that precludes the participant from undergoing procedures required in this study

Where it is running

• Nationwide Children's Hospital — Columbus, Ohio, United States
• Women's and Children's Hospital — North Adelaide, South Australia, Australia
• Vall d'Hebron Barcelona Campus — Barcelona, Spain
• Hospital Clínico Universitario de Santiago — Santiago de Compostela, Spain

Full record on ClinicalTrials.gov

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