Clinical Research Directory
Browse clinical research sites, groups, and studies.
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
Sponsor: Ultragenyx Pharmaceutical Inc
Summary
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).
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)
Key Details
Gender
All
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
41
Start Date
2020-09-28
Completion Date
2027-08
Last Updated
2026-01-21
Healthy Volunteers
No
Interventions
No Investigational Product
No investigational product will be administered in this follow-up trial.
Adjuvant Immunomodulatory (IM) Therapy
The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IM therapy. Not all participants may receive IM therapy.
Locations (4)
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