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NCT03759678 · ClinicalTrials.gov registry record · Phase 2
N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)
A Phase 2 study of Ataxia-Telangiectasia and Louis Bar Syndrome, sponsored by IntraBio.
- Active
- Registry status
- Phase 2
- Development phase
- 39
- Enrollment target
- 5
- Study locations
NCT03759678: Active Phase 2 study of Ataxia-Telangiectasia and Louis Bar Syndrome, sponsored by IntraBio.
NCT03759678 is a Phase 2 study of Ataxia-Telangiectasia and Louis Bar Syndrome that is active but no longer recruiting, run by IntraBio. The registered enrollment target is 39 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (71% lower). The trial reports 5 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03759678, a Phase 2 study of Ataxia-Telangiectasia and Louis Bar Syndrome, is active but no longer recruiting, sponsored by IntraBio.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 39 participants
- Enrollment target
- 5
- Study locations
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 Ataxia-Telangiectasia (A-T). 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 A-T. The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of A-T.
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
Conditions Studied
Interventions
- DRUG IB1001
Study Locations (5)
Other
- University of Giessen - Giessen
- Ludwig Maximilian University of Munich - München
California
- University of California - Los Angeles - Los Angeles
Spain
- Hospital Universitario La Paz - Madrid
Cambridgeshire
- Royal Papworth Hospital NHS Foundation Trust - Cambridge
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 39 participants |
| Start Date | 2020-01-08 |
| Est. Completion | 2025-07-01 |
| Phase | Phase 2 |
What the registry record for NCT03759678 still lists
NCT03759678 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 39 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (71% lower).
The record links to 2 conditions, with Ataxia-Telangiectasia appearing as the primary indexed condition, and to 1 intervention - of which IB1001 is the first listed.
NCT03759678 lists 5 locations in 4 states (Other, California, Spain).
Frequently Asked Questions
What is clinical trial NCT03759678 about?
NCT03759678 is a clinical study titled "N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)". 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 Ataxia-Telangiectasia (A-T). There are two phases to this study: the Parent Study, and the Extension Phase. The P...
What is the current status of trial NCT03759678?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 39 participants. The study started on 2020-01-08. Estimated completion is 2025-07-01.
What conditions does trial NCT03759678 study?
This clinical trial studies the following conditions: Ataxia-Telangiectasia, Louis Bar Syndrome.
What interventions are being tested in trial NCT03759678?
The interventions under investigation include: IB1001 (DRUG).
Who is sponsoring clinical trial NCT03759678?
This trial is sponsored by IntraBio, which has 6 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03759678 being conducted?
This trial has 5 study locations across California, Spain, Cambridgeshire. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Where NCT03759678's enrollment target sits among peer trials
39 1459th of 2000 higher than 534 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
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Source ClinicalTrials.gov registry export · 2026-08-08
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