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Clinical trial

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)

ID
Name
LTFU-ABO-102
Description
The main objective of this study is to evaluate the long-term safety and tolerability of ABO-102 in participants with MPS IIIA.
Trial arms
Trial start
2020-09-28
Estimated PCD
2027-06-01
Trial end
2027-06-01
Phase
Early phase I
Treatment
No Investigational Product
No investigational product will be administered in this follow-up trial.
Arms:
Cohort 1, Cohort 2
Adjuvant Immunosuppression (IS) Therapy
The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IS therapy.
Arms:
Cohort 1
Size
33
Primary endpoint
Adverse Events
Up to Year 5
Eligibility criteria
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 this study. * Any other situation or medical condition that precludes the participant from undergoing procedures required in this study.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'OTHER', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 33, 'type': 'ESTIMATED'}}
Updated at
2024-02-07

1 organization

3 products

1 indication

Product
Adjuvant Immunosuppression
Indication
MPS III-A
Organization
Ultragenyx Pharmaceutical
Product
Adjuvant Immunosuppression Therapy
Product
Adjuvant Immunosuppression (IS) Therapy