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N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)

NCT03759678 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 17

Last updated 2026-03-25

Study results available
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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.

Conditions

  • Ataxia Telangiectasia

Interventions

DRUG

IB1001

IB1001 (N-Acetyl-L-Leucine) is a modified amino-acid ester that is orally administered.

Sponsors & Collaborators

  • IntraBio Inc

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-01-08
Primary Completion
2025-02-11
Completion
2025-10-27
FDA Drug
Yes

Countries

  • United States
  • Germany
  • Spain
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03759678 on ClinicalTrials.gov