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)
Phase 1
- Conditions
- Mucopolysaccharidosis type IIIAMedDRA version: 20.1Level: PTClassification code: 10056890Term: Mucopolysaccharidosis III Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- CTIS2023-510392-66-00
- Lead Sponsor
- ltragenyx Pharmaceutical Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 34
Inclusion Criteria
Participants that have completed a prior clinical trial involving the administration of ABO-102., 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 Criteria
Planned or current participation in another clinical trial that may confound the safety and efficacy evaluation of ABO-102 during the duration of this study., Any other situation or medical condition that precludes the participant from undergoing procedures required in this study.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method