Pivotal Study of N-acetyl-L-leucine for CACNA1A
Trial statusNot yet recruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age4+
SponsorIntraBio Inc
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 groupsSponsors and collaborators
Source ClinicalTrials.gov