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NCT03759665 · ClinicalTrials.gov registry record · Phase 2
N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)
A Phase 2 study, sponsored by IntraBio.
- Completed
- Registry status
- Phase 2
- Development phase
- 30
- Enrollment target
NCT03759665: Completed Phase 2 study, sponsored by IntraBio.
NCT03759665 is a Phase 2 study that has completed, run by IntraBio. The registered enrollment target is 30 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (77% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03759665, a Phase 2 study, has completed, sponsored by IntraBio.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 30 participants
- Enrollment target
Study Summary
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). There are two phases to this study: the Parent Study, and the Extension Phase. The Parent Study evaluates the safety and efficacy of N-Acetyl-L-Leucine (IB1001) in the symptomatic treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of GM2 Gangliosidosis. The Extension Phase was considered exploratory.
Primary Outcome
The Clinical Impression of Change in Severity assessment will instruct the blinded rater to consider: compared to the first video, how has the severity of their performance on the 9 Hole Peg Test of the Dominant Hand (9HPT-D) or 8 Meter Walk Test (8MWT) changed (improved or worsened) in 6-weeks as observed in the second video? The Clinical Impression of Change in Severity is evaluated on a 7 point Likert scale (+3=significantly improved to -3= significantly worse). Change values were calculated
Interventions
- DRUG IB1001
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 30 participants |
| Start Date | 2019-06-07 |
| Est. Completion | 2023-01-09 |
| Phase | Phase 2 |
What the finished NCT03759665 record still lists
NCT03759665 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 30 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (77% lower).
The record links to 0 conditions, and to 1 intervention - of which IB1001 is the first listed.
NCT03759665 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03759665 about?
NCT03759665 is a clinical study titled "N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)". This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). There are two phases to this study: the Parent Study, and the...
What is the current status of trial NCT03759665?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 30 participants. The study started on 2019-06-07. Estimated completion is 2023-01-09.
What interventions are being tested in trial NCT03759665?
The interventions under investigation include: IB1001 (DRUG).
Who is sponsoring clinical trial NCT03759665?
This trial is sponsored by IntraBio, which has 6 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03759665's enrollment target sits among peer trials
30 1582nd of 2000 higher than 332 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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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