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Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease | Clinical Trials | Clareo Health

TERMINATEDPHASE1/PHASE2

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients With Middle and Advanced Phases of MPS IIIA Disease

NCT04088734•Ultragenyx Pharmaceutical Inc

View on ClinicalTrials.gov

Summary

Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein

Detailed Description

This is an open-label, single dose clinical trial. All participants will receive 3 X 10\^13 vg/kg of ABO-102 delivered one time through a venous catheter inserted into a peripheral limb vein. The target population includes MPS IIIA participants with a DQ lower than 60 in middle and advanced phases of the disease. Similar numbers of MPS IIIA participants with age equivalent above and below 18 months of age will be enrolled to ensure a representation of middle and advanced phases of the disease. This study was previously posted by Abeona Therapeutics, Inc and was transferred to Ultragenyx in August 2022.

Eligibility

Age

2 - 18 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Diagnosis of MPS IIIA confirmed by the following methods:
•1. No detectable or significantly reduced SGSH enzyme activity by leukocyte assay and
•2. Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the SGSH gene
•Cognitive Development Quotient (DQ) lower than 60 (calculated by Bayley Scales of Infant and Toddler Development - Third Edition)
•Must be ambulatory, though may receive assistance with ambulation
•Age range of 2 years up to 18 years (excluded)

Exclusion Criteria

•Inability to participate in the clinical evaluation as determined by Principal Investigator
•Identification of two nonsense or null variants on genetic testing of the SGSH gene
•At least one S298P mutation in the SGSH gene
•Has evidence of an attenuated phenotype of MPS IIIA
•Presence of a concomitant medical condition that precludes lumbar puncture or use of anesthetics
•Active viral infection based on clinical observations
•Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer, or precludes the child from participating in the protocol assessments and follow up
•Participants with total anti-AAV9 antibody titers greater than or equal to 1:100 as determined by ELISA binding immunoassay
•Participants with a positive response for the ELISPOT for T-cell responses to AAV9
•Serology consistent with exposure to HIV, or serology consistent with active hepatitis B or C infection
+13 more criteria

Locations (3)

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Adelaide Women's and Children's Hospital

North Adelaide, South Australia, Australia

Hospital Clínico Universitario de Santiago

Santiago de Compostela, Spain

Key Dates

Start Date

September 18, 2019

Primary Completion Date

March 10, 2022

Completion Date

March 10, 2022

Last Updated

July 25, 2023

Enrollment

ACTUAL participants

Conditions

MPS IIIASanfilippo SyndromeSanfilippo AMucopolysaccharidosis III

Interventions

ABO-102

DRUG

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: July 25, 2023Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

Inclusion Criteria

Exclusion Criteria

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