Status:
ENROLLING_BY_INVITATION
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
Lead Sponsor:
Ultragenyx Pharmaceutical Inc
Conditions:
Mucopolysaccharidosis IIIA
MPS IIIA
Eligibility:
All Genders
Phase:
PHASE3
Brief 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).
Detailed Description
This is a multicenter, long-term follow-up study of patients with MPS IIIA who have participated in a prior clinical trial involving the administration of UX111 (NCT02716246 and NCT04088734). No inves...
Eligibility Criteria
Inclusion
- Participants that have participated in a prior clinical trial in which they received UX111
- Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule
Exclusion
- Planned or current participation in another clinical trial that may confound the safety or efficacy evaluation of UX111 during this study
- Any other situation or medical condition that precludes the participant from undergoing procedures required in this study
Key Trial Info
Start Date :
September 28 2020
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
August 1 2027
Estimated Enrollment :
41 Patients enrolled
Trial Details
Trial ID
NCT04360265
Start Date
September 28 2020
End Date
August 1 2027
Last Update
October 20 2025
Active Locations (4)
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1
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
2
Women's and Children's Hospital
North Adelaide, South Australia, Australia
3
Vall d'Hebron Barcelona Campus
Barcelona, Spain, 08035
4
Hospital Clínico Universitario de Santiago
Santiago de Compostela, Spain