A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102

Mucopolysaccharidosis III-A Clinical Trial

Official title: 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)

Status Enrolling by invitation

Clinical Trial Summary

The main objective of this study is to evaluate the long-term safety and tolerability of ABO-102 in participants with MPS IIIA.

Clinical Trial Description

This is a multicenter, long-term follow-up study of patients with MPS IIIA who have completed a prior clinical trial involving the administration of ABO-102 (NCT02716246 and NCT04088734). No investigational product will be provided. At approved sites immunosuppression (IS) therapy may be administered to selected participants. The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IS therapy. Not all participants may receive IS therapy. This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022. ;

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis III
• Mucopolysaccharidosis III-A

• NCT number: NCT04360265
• Study type: Interventional
• Source: Ultragenyx Pharmaceutical Inc
• Status: Enrolling by invitation
• Phase: Phase 3
• Start date: September 28, 2020
• Completion date: June 2027