A pivotal study of N-Acetyl-L-Leucine in Ataxia-Telangiectasia
Research type
Research Study
Full title
Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Phase III, randomized, placebo-controlled, double-blind, crossover study
IRAS ID
1011174
Contact name
Taylor Fields
Contact email
Sponsor organisation
IntrBio Ltd.
Eudract number
2024-517706-29
Clinicaltrials.gov Identifier
Research summary
There are currently no available treatments for Ataxia-Telangiectasia (A-T). Therefore, there is a need for the development of novel and more effective therapies to treat these intractable diseases. N-Acetyl-L- Leucine (study drug) has been studied in Phase II trials, in patients with A-T and Niemann-Pick Type C disease (NPC), as well as in a Phase III study in patients with NPC. The study design for this Phase III study (in A-T) is the same as the IB1001-301 study (in NPC).
The goal of this study is to demonstrate that N-Acetyl-L-Leucine is efficacious in improving symptoms, functioning, and quality of life in patients with A-T. Phase II and III clinical trials have demonstrated rapidly improved neurological signs and symptoms, functioning, and quality of life following treatment with N-Acetyl-L- Leucine (IB1001) in paediatric and adult patients, with A-T and NPC. N-Acetyl-L-Leucine was observed to be safe and well-tolerated, with no drug-related serious adverse events, establishing a very favourable risk- benefit profile for IB1001.
Based on these findings, IntraBio is conducting a Phase III, placebo-controlled, double-blind, randomized, crossover study investigating the efficacy and safety of N-Acetyl-L-Leucine for the treatment of A-T. Patients will only be included in the study if they meet the inclusion and exclusion criteria described in the study protocol approved by the relevant ethics committees and the regulatory authorities and informed consent is obtained. To further minimize risks, patients will be monitored closely throughout the study. The study will occur in approximately 9 sites in Germany, Slovakia, Spain, Switzerland, the United Kingdom and the United States. The sites in the UK will be NHS sites. There will be a screening period of up to 56 days. This will be followed by the treatment period, which is expected to be approximately 168 days (from the first day of dosing [Visit 2] to the final day of dosing [at the end of Period 2/Visit 6]).
REC name
London - Hampstead Research Ethics Committee
REC reference
24/LO/0907
Date of REC Opinion
23 Jan 2025
REC opinion
Further Information Favourable Opinion