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NCT04088734 · ClinicalTrials.gov registry record · Phase 1
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
A Phase 1 study, sponsored by Ultragenyx Pharmaceutical.
- Terminated
- Registry status
- Phase 1
- Development phase
- 5
- Enrollment target
NCT04088734: Clinical Trial Phase 1 study, sponsored by Ultragenyx Pharmaceutical.
NCT04088734 is a Phase 1 study that was terminated before completion, run by Ultragenyx Pharmaceutical. The registered enrollment target is 5 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (92% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04088734, a Phase 1 study, was terminated before completion, sponsored by Ultragenyx Pharmaceutical.
- TERMINATED
- Registry status
- Phase 1
- Development phase
- 5 participants
- Enrollment target
Study Summary
Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein
Primary Outcome
An adverse event (AE) is any untoward medical occurrence or unintended change from the time informed consent form (ICF) is signed, including inter-current illness that occurs during the course of a clinical trial after treatment has started, whether considered related to treatment or not. TEAEs are those that occurred after the start of study drug. Related adverse events were categorized as possible, probable, or definitely.
Interventions
- DRUG ABO-102
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 5 participants |
| Start Date | 2019-09-18 |
| Est. Completion | 2022-03-10 |
| Phase | Phase 1 |
Why NCT04088734 stopped before completion
NCT04088734 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 5 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (92% lower).
The record links to 0 conditions, and to 1 intervention - of which ABO-102 is the first listed.
NCT04088734 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04088734 about?
NCT04088734 is a clinical study titled "Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease". Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein
What is the current status of trial NCT04088734?
This trial is currently terminated. It is a Phase 1 study. The enrollment target is 5 participants. The study started on 2019-09-18. Estimated completion is 2022-03-10.
What interventions are being tested in trial NCT04088734?
The interventions under investigation include: ABO-102 (DRUG).
Who is sponsoring clinical trial NCT04088734?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04088734's enrollment target sits among peer trials
5 1989th of 2000 higher than 6 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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