Trial Profile

A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients With Middle and Advanced Phases of MPS IIIA Disease

Status: Discontinued

Phase of Trial: Phase I/II

Latest Information Update: 27 Jul 2023

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At a glance

Drugs Rebisufligene etisparvovec (Primary)
Indications Mucopolysaccharidosis III
Focus Adverse reactions; Therapeutic Use
Sponsors Abeona therapeutics; Ultragenyx Pharmaceutical

Most Recent Events

06 Apr 2022 Status changed from recruiting to discontinued.
17 Sep 2020 Planned End Date changed from 1 Dec 2022 to 1 Dec 2023.
17 Sep 2020 Planned primary completion date changed from 1 Dec 2022 to 1 Dec 2023.

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