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

Phase 3Enrolling By InvitationNCT04360265

Ultragenyx Pharmaceutical Inc41 enrolled

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).

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

OTHER

Masking

NONE

Enrollment

Conditions

Mucopolysaccharidosis IIIA MPS IIIA Sanfilippo Syndrome Sanfilippo A

Interventions

No Investigational ProductOTHER

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

Adjuvant Immunomodulatory (IM) TherapyDRUG

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.

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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

Locations (4)

Nationwide Children's Hospital

Columbus, Ohio, United States

Women's and Children's Hospital

North Adelaide, South Australia, Australia

Vall d'Hebron Barcelona Campus

Barcelona, Spain

Hospital Clínico Universitario de Santiago

Santiago de Compostela, Spain

Outcomes

Primary Outcomes

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 Outcomes

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

Data from ClinicalTrials.gov

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