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NCT03759639 · ClinicalTrials.gov registry record · Phase 2
N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)
A Phase 2 study, sponsored by IntraBio.
- Completed
- Registry status
- Phase 2
- Development phase
- 33
- Enrollment target
NCT03759639: Completed Phase 2 study, sponsored by IntraBio.
NCT03759639 is a Phase 2 study that has completed, run by IntraBio. The registered enrollment target is 33 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (75% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03759639, a Phase 2 study, has completed, sponsored by IntraBio.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 33 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 Niemann-Pick type C disease (NPC). 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) for the symptomatic treatment of NPC. The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of NPC.
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).
Interventions
- DRUG IB1001
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 33 participants |
| Start Date | 2019-09-04 |
| Est. Completion | 2022-11-07 |
| Phase | Phase 2 |
What the finished NCT03759639 record still lists
NCT03759639 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 33 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (75% lower).
The record links to 0 conditions, and to 1 intervention - of which IB1001 is the first listed.
NCT03759639 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03759639 about?
NCT03759639 is a clinical study titled "N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)". 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 Niemann-Pick type C disease (NPC). There are two phases to this study: the Parent Study, and the Extension Phase. ...
What is the current status of trial NCT03759639?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 33 participants. The study started on 2019-09-04. Estimated completion is 2022-11-07.
What interventions are being tested in trial NCT03759639?
The interventions under investigation include: IB1001 (DRUG).
Who is sponsoring clinical trial NCT03759639?
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 NCT03759639's enrollment target sits among peer trials
33 1553rd of 2000 higher than 438 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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