ABO-102

DRUG3 trials

Sponsors

Ultragenyx Pharmaceutical Inc., Ultragenyx Pharmaceutical Inc

Conditions

MPS IIIAMucopolysaccharidosis IIIMucopolysaccharidosis type IIIASanfilippo ASanfilippo Syndrome

Phase 1

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

TerminatedNCT04088734

Ultragenyx Pharmaceutical IncMPS IIIA, Mucopolysaccharidosis III, Sanfilippo A +1

Start: 2019-09-18End: 2022-03-10Updated: 2023-07-25

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA

RecruitingCTIS2023-510032-37-00

Ultragenyx Pharmaceutical Inc.Mucopolysaccharidosis type IIIA

Start: 2017-04-18Target: 21Updated: 2025-12-12

Phase 3

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)

RecruitingCTIS2023-510392-66-00

Ultragenyx Pharmaceutical Inc.Mucopolysaccharidosis type IIIA

Start: 2020-11-23Target: 24Updated: 2025-10-20