Health Trials

About this trial

A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of CACNA1A. The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.

Eligibility criteria

Qualifiers

Written informed consent signed by the patient and/or their legal representative / parent/ impartial witness.

Male or female aged ≥4 years with a genetically-confirmed diagnosis of a CACNA1A Disorder (including patients with loss-of-function and fain-of-function mutations, e.g. Episodic Ataxia Type 2 (EA2), Familial Hemiplegic Migraine Type 1, Spinocerebellar Ataxia type C (SCA6), Developmental and Epileptic encephalopathy 42 (DEE42), Congenital ataxia or cerebellar hypoplasia due to a CACNA1A mutation) at the time of signing informed consent.

intrauterine device (IUD);

surgical sterilization of the partner (vasectomy for 6 months minimum);

Disqualifiers

Acetyl-Leucine (DL-, L-, D-) or derivatives.

Excipients the IB1001 sachet (namely isomalt, hypromellose, and strawberry flavor).

Excipients the placebo sachet (namely isomalt, hypromellose, strawberry flavor, citric acid, microcrystalline cellulose, lactose, denatonium benzoate).

Simultaneous participation in another clinical study or participation in any clinical study involving administration of an investigational medicinal product (IMP; 'study drug') for at least 42 days prior to Visit 1. At the discretion of the Investigator, Medical Monitor, and Sponsor, the washout period for specific IMPs may be longer based on the pharmacological activity and pharmacokinetics of the drug.

Trial design

Treatments tested in this trial

  • N-Acetyl-L-Leucine
  • Placebo

Treatment groups

60 Participants
are divided into 2 treatment groups

Locations

10
Austria
Medical University of Innsbruck Innsbruck Austria
Germany
University of Cologne50937, Cologne Germany
Greece
University of Athens Athens Greece
Italy
University of Milan Milan Italy

Sponsors and collaborators

IntraBio Inc

Lead sponsor

Source ClinicalTrials.gov