MPS IIIA Disease Monitoring Program
Mucopolysaccharidosis Type IIIA (MPS IIIA) Disease Monitoring Program
1 other identifier
observational
55
0 countries
N/A
Brief Summary
The main objective of this observational study is to evaluate the long-term safety and effectiveness of rebisufligene etisparvovec for 10 years after rebisufligene etisparvovec administration.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started Nov 2026
Longer than P75 for all trials
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 25, 2026
CompletedFirst Posted
Study publicly available on registry
October 2, 2026
CompletedStudy Start
First participant enrolled
November 1, 2026
ExpectedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2038
Study Completion
Last participant's last visit for all outcomes
October 1, 2038
October 2, 2026
September 1, 2026
11.9 years
September 25, 2026
October 1, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Number of Participants with Serious Adverse Events (SAEs) Assessed as Related to Rebisufligene Etisparvovec by the Investigator and Targeted Adverse Events (Targeted AEs) Associated with Identified and Potential Risks of UX111 or AAV8 Gene Therapy
10 Years
Bayley Scales of Infant and Toddler Development Cognitive Raw Score Over Time
10 Years
Study Arms (2)
Group 1: Rebisufligene Etisparvovec in Prior Clinical Study
Participants administered rebisufligene etisparvovec (Fayuvi, UX111) in prior clinical study involving rebisufligene etisparvovec
Group 2: Rebisufligene Etisparvovec in Post-Marketing Setting
Participants administered rebisufligene etisparvovec (Fayuvi, UX111) in a post-marketing setting
Interventions
No investigational/study product will be administered in this DMP.
Eligibility Criteria
The DMP population comprises patients with MPS IIIA who received rebisufligene etisparvovec in a clinical trial or have been prescribed (or plan to receive) rebisufligene etisparvovec in a post marketing setting.
You may qualify if:
- Confirmed diagnosis of MPS IIIA
- Patient who:
- Group 1: Received rebisufligene etisparvovec in a prior interventional clinical trial.
- May be enrolled upon completion of Study ABT-001 (Cohort 4) or end of study (EOS) or early termination (ET) visit from the prior clinical trial (Study ABT-003 or Study LTFU-ABO-102). OR
- Group 2: Received (or plans to receive) rebisufligene etisparvovec in a postmarketing setting.
- Provide informed consent after the nature of the study has been explained, and prior to any research-related procedures. If a minor or an adult with cognitive limitations, willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related procedures.
You may not qualify if:
- Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Director
Ultragenyx Pharmaceuticals Inc.
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 25, 2026
First Posted
October 2, 2026
Study Start (Estimated)
November 1, 2026
Primary Completion (Estimated)
October 1, 2038
Study Completion (Estimated)
October 1, 2038
Last Updated
October 2, 2026
Record last verified: 2026-09