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Follow-up Study for UX111 in MPS IIIA

This study is a long-term follow-up for individuals with Mucopolysaccharidosis IIIA (MPS IIIA), also known as Sanfilippo Syndrome Type A, who have previously received UX111 (formerly ABO-102) in an earlier clinical trial. The main goals are to continue checking the safety and how well UX111 works over time. No new UX111 will be given in this follow-up study. Some participants might receive additional immune-modulating (IM) therapy, decided by their doctor and caregiver. The study will track any side effects and changes in cognitive (thinking and learning) scores using the Bayley Scales of Infant and Toddler Development – Third Edition (BSITD-III) for up to five years. The study is currently unclear on its recruitment status and plans to enroll 41 participants.

Study design
This is an interventional, multicenter, long-term follow-up study. It plans to enroll 41 participants.
What's involved
You would need to have participated in a prior clinical trial where you received UX111. You or your legal guardian must be willing to complete consent and follow study procedures and visit schedules.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for up to five years to assess side effects and cognitive development.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04360265

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

Enrolling by Invitation
PHASE3All AgesInterventional
Ultragenyx Pharmaceutical Inc
~41 participants
Updated 2026-07-17 on ClinicalTrials.gov
What's tested:No Investigational ProductAdjuvant Immunomodulatory (IM) Therapy

At a glance

Recruiting sites
0 of 4 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs)
Measured over Up to Year 5
+1 more outcome measured

Conditions

Mucopolysaccharidosis IIIA
MPS IIIA
Sanfilippo Syndrome
Sanfilippo A

Explore related trials

Mucopolysaccharidosis Type IIIA

Where it's being run

4 sites across 3 states
Spain2
Ohio1
South Australia1

Study leadership

  • Medical Director · STUDY_DIRECTOR · Ultragenyx Pharmaceutical Inc

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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

What this trial measures

  • Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs)Up to Year 5
  • Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive Raw Score Over TimeUp to Year 5