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Clinical Trials in the USA / NCT04360265
Enrolling by invitation Phase 3

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

NCT04360265 · tracked via the Priya Life Science USA tracker
Sponsor
Ultragenyx Pharmaceutical Inc
Phase
Phase 3
Started
2020-09-28
Last updated
2026-07-17

Condition(s) studied

Mucopolysaccharidosis IIIAMPS IIIASanfilippo SyndromeSanfilippo A

Investigational drug(s) / intervention(s)

No Investigational ProductAdjuvant Immunomodulatory (IM) Therapy

No Investigational Product: No investigational product will be administered in this follow-up trial.

Adjuvant Immunomodulatory (IM) Therapy: The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IM therapy. Not all participants may receive IM therapy.

Study summary

The main objective of this study is to evaluate the safety/tolerability and efficacy of UX111 (previously known as ABO-102) in participants with Mucopolysaccharidosis IIIA (MPS IIIA).

Eligibility

Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
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

Primary outcome measure(s)

  • 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 Time — Up to Year 5

Trial sites (4)

FacilityCityRegionStatus
Nationwide Children's Hospital Columbus Ohio
Women's and Children's Hospital North Adelaide South Australia
Vall d'Hebron Barcelona Campus Barcelona Spain
Hospital Clínico Universitario de Santiago Santiago de Compostela Spain

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Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency Active, not recruiting
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta Active, not recruiting

Other trials for the same condition

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04360265 on ClinicalTrials.gov ↗ ← All trials in the USA