NCT03759678

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

Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: IntraBio Inc · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

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.

Detailed description

The primary purpose of the study is to evaluate the safety and efficacy of N-Acetyl-L-Leucine (IB1001) in the treatment of A-T investigating the efficacy in terms of improving symptoms, functioning, and quality of life against the defined endpoints in patients with A-T. Patients will be assessed during three study phases: a baseline period, a 6-week treatment period, and a 6-week post-treatment washout period. If within 6 weeks prior to the initial screening visit, a patient has received any of the prohibited medications defined in the eligibility criteria (irrespective of the preceding treatment duration) a wash-out study-run in of 6 weeks is required prior to the first baseline assessment. All patients will receive the study drug during this study. For each individual patient, the study lasts for approximately 3.5 - 4 months during which there are 6 study visits to the study site. This Extension Phase allows patients who have completed the Parent Study to, at the discretion of the Principal Investigator (PI), continue treatment with N-Acetyl-L-Leucine (IB1001). Patients will receive treatment with IB1001 for two one-year treatment periods, separated by a 6-week washout. All patients will receive the study drug during the treatment period. For each individual patient, the Extension Phase lasts for approximately 25.5 months, during which there are 6 visits to the study site.

Conditions

• Ataxia Telangiectasia

Interventions

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

Timeline

• Start date: 2020-01-08
• Primary completion: 2025-02-11
• Completion: 2025-10-27
• First posted: 2018-11-30
• Last updated: 2026-03-25
• Results posted: 2026-03-25

Locations

4 sites across 4 countries: United States, Germany, Spain, United Kingdom

Regulatory

• FDA-regulated drug study