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Enrolling by invitation NCT04360265

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

Phase III Interventional Mucopolysaccharidosis IIIA MPS IIIA Sanfilippo Syndrome Sanfilippo A

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: No Investigational Product, Adjuvant Immunomodulatory (IM) Therapy.
Who it may be relevant to
Registry conditions: Mucopolysaccharidosis IIIA, MPS IIIA, Sanfilippo Syndrome, Sanfilippo A. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Australia, Spain
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)

Overview

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).

Detailed description

This is a multicenter, long-term follow-up study of patients with MPS IIIA who have participated in a prior clinical trial involving the administration of UX111 (NCT02716246 and NCT04088734). No investigational product will be provided. At approved sites adjuvant immunomodulatory (IM) therapy may be administered to selected participants. 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.

This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.

Interventions

  • Other No Investigational Product
    No investigational product will be administered in this follow-up trial.
  • Drug 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.

Primary outcome measures

  • Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs) [Time frame: Up to Year 5]
  • Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive Raw Score Over Time [Time frame: Up to Year 5]
Secondary outcome measures (6)
  • Cerebrospinal Fluid (CSF) Heparin Sulfate (HS) (Disaccharide) Exposure [Time frame: Baseline, Up to Month 36]
  • Percent Change From Baseline in Prior Trial in CSF HS [Time frame: Baseline, Up to Month 36]
  • BSITD-III Receptive Communication Raw Score Over Time [Time frame: Up to Year 5]
  • BSITD-III Expressive Communication Raw Score Over Time [Time frame: Up to Year 5]
  • Annualized Percentage Change from Baseline in Prior Trial in Total Cortical Volume [Time frame: Baseline, Up to Month 36]
  • Survival [Time frame: Up to Year 5]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Parallel assignment
Masking
Open label
Primary purpose
Other

Study locations

Spain · 2 centers
  • Vall d'Hebron Barcelona Campus — Barcelona
  • Hospital Clínico Universitario de Santiago — Santiago de Compostela
United States · 1 center
  • Nationwide Children's Hospital — Columbus
Australia · 1 center
  • Women's and Children's Hospital — North Adelaide

Identifiers

NCT: NCT04360265 · LTFU-ABO-102 · 2019-002979-34 · UX111-CL302 · 2023-510392-66-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗