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

• Status: Enrolling by invitation
• Trial end date: 2027-06-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ultragenyx Pharmaceutical Inc
• Collaborator: Abeona Therapeutics, Inc

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.