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Clinical Trials in Spain / 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 Spain 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)

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

More Ultragenyx Pharmaceutical Inc trials in Spain

Long-term Extension of GTX-102 in Angelman Syndrome Active, not recruiting
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency Active, not recruiting
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD Active, not recruiting
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) Active, not recruiting
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease 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 Spain