Pivotal Study of N-acetyl-L-leucine for CACNA1A (NCT07221292) | Clinical Trial Compass
Not Yet RecruitingPhase 3
Pivotal Study of N-acetyl-L-leucine for CACNA1A
United States, Germany, Switzerland60 participantsStarted 2026-09-01
Plain-language summary
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.
Who can participate
Age range4 Years
SexALL
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Inclusion criteria
✓. 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.
✓. Females of childbearing potential, defined as a premenopausal female capable of becoming pregnant, will be included if they are either sexually inactive (sexually abstinent for 14 days prior to the first dose and confirm to continue through 28 days after the last dose) or using one of the following highly effective contraceptives (i.e. results in \<1% failure rate when used consistently and correctly) 14 days prior to the first dose continuing through 28 days after the last dose:
✓. intrauterine device (IUD);
✓. surgical sterilization of the partner (vasectomy for 6 months minimum);
✓. combined (estrogen or progestogen containing) hormonal contraception associated with the inhibition of ovulation (either oral, intravaginal, or transdermal);
✓. progestogen only hormonal contraception associated with the inhibition of ovulation (either oral, injectable, or implantable);
✓. intrauterine hormone releasing system (IUS);
Exclusion criteria
✕
What they're measuring
1
Scale for the Assessment and Rating of Ataxia
Timeframe: End of Period I (week 12) vs. End of Period 2 (week 24)
✕. 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.
✕. Patients with a physical or psychiatric condition which, at the Investigator's discretion and in consultation with the Medical Monitor and Sponsor (as applicable), may put the patient at risk, may confound the study results, or may interfere with the patient's participation in the clinical study, i.e. reliably perform study assessments.
✕. Known or persistent use, misuse, or dependency of medication, drugs, or alcohol.
✕. Current or planned pregnancy or women who are breastfeeding.