Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

Plain-language summary

This Phase 3 study evaluates the safety, tolerability and efficacy of the AAV-mediated gene transfer product UX111 (previously known as ABO-102) in participants with Mucopolysaccharidosis IIIA (MPS IIIA). Adjuvant immunomodulatory therapy is also part of the study. Enrollment is by invitation and limited to participants who received UX111 in a prior clinical trial and whose parents or legal guardians can complete the informed consent process and comply with study procedures and the visit schedule.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 3

Status

Enrolling by invitation

Sponsor

Ultragenyx Pharmaceutical Inc

Intervention

No Investigational Product, Adjuvant Immunomodulatory (IM) Therapy

Start date

9/28/2020

Estimated completion

8/1/2027

Countries

United States, Australia, Spain

Related genes

Locations

• Columbus, United States
• North Adelaide, Australia
• Barcelona, Spain
• Santiago de Compostela, Spain

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* 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 Criteria:

* 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