Pivotal study of N-Acetyl-L-Leucine in CACNA1A Disorders
Research type
Research Study
Full title
Effects of N-Acetyl-L-Leucine on CACNA1A Disorders: A Phase III, randomized, placebo-controlled, double-blind, crossover study
IRAS ID
1013172
Contact name
Taylor Fields
Contact email
Sponsor organisation
IntraBio Inc
Eudract number
2025-523828-51
Clinicaltrials.gov Identifier
Research summary
There are currently no available approved, on-label treatments for any CACNA1A1 disorder. Therefore, there is a need for the development of novel and more effective therapies to treat these disorders. N-Acetyl-L- Leucine (study drug) has been studied in Phase II and III trials, in patients with (Ataxia-Telangiectasia) A-T and Niemann-Pick Type C disease (NPC). The study design for this Phase III study (in CACNA1A disorders) is the same as the previous Phase III studies.
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 CACNA1A disorders. 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 CACNA1A disorders. Patients will only be included in the study if they meet the eligibility criteria described in the study protocol, which is approved by the relevant ethics and regulatory authorities. To further minimize risks, patients will be monitored closely throughout the study. The study will occur in approximately 9 sites in Germany, Italy, Greece, Austria, Switzerland, the United Kingdom and the United States. The sites in the UK will be NHS sites.
All participants will provide informed consent. There will be a screening period of up to 56 days. This will be followed by the treatment period (approximately 168 days (from the first day of dosing [Visit 2] to the final day of dosing [at the end of Visit 6]).REC name
London - Hampstead Research Ethics Committee
REC reference
26/LO/0157
Date of REC Opinion
31 Mar 2026
REC opinion
Further Information Favourable Opinion