TerminatedPhase 1/2

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

Stopped: Terminated due to lack of efficacy seen in patients with advanced MPS IIIA disease. The patients will be followed up annually for safety until five years post dosing

United States, Australia, Spain5 participantsStarted 2019-09-18

Plain-language summary

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

Who can participate

Age range

2 Years – 18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. No detectable or significantly reduced SGSH enzyme activity by leukocyte assay and
. Genomic DNA analysis demonstrating homozygous or compound heterozygous mutations in the SGSH gene

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Incidence, Type and Severity of Related Treatment-Emergent Adverse Events (TEAEs) by Time Frame

Timeframe: From the first dose of study drug to <30 days postdose, Day 30, 60, 90, 180 and Month 12

Incidence, Type and Severity of Serious Adverse Events (SAEs) by Time Frame

Timeframe: From signing of informed consent through Day 60, 90, 180 and up to Day 454 (> 12 months)

Change From Baseline (BL) in Multiples of Normal of Liver and Spleen Volumes After Treatment

Timeframe: Baseline, Day 30, 180, Month 12

Change From BL in Cerebrospinal Fluid (CSF) Heparan Sulfate Levels After Treatment

Timeframe: Baseline, Day 30, Day 180, Month 12

Trial details

NCT IDNCT04088734

SponsorUltragenyx Pharmaceutical Inc

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-03-10

Results submitted2023-06-09

View on ClinicalTrials.gov More MPS IIIA trials