Trial
NCT04088734
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
Phase ITERMINATEDEFFICACY/FUTILITYConfidence: MEDIUM
Sponsor
Ultragenyx Pharmaceutical Inc
Collaborators
Abeona Therapeutics, Inc
Condition
MPS IIIA
Intervention
ABO-102
Why stopped
Terminated due to lack of efficacy seen in patients with advanced MPS IIIA disease. The patients will be followed up annually for safety until five years post dosing
Provenance
Dataset: 2026-07-02 • Source: ClinicalTrials.gov API v2 • Last update: 2023-07-25