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This study examines the safety of single ascending doses of the drug CTI-1601 compared with a placebo in people with Friedreich's ataxia. It enrolled patients aged 18 and over with a genetically confirmed diagnosis of Friedreich's ataxia (homozygous GAA repeat expansion). The study was conducted in Phase 1 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: 1. Subject has genetically confirmed Friedreich's ataxia diagnosis, homozygous GAA repeat expansions, with repeat sizing (if available) included on diagnostic report. 2. Subject is male or female, 18 years of age or older at screening. 3. Subject must have a mFARS\_neuro score ≥ 20 and be able to traverse a distance of 25 feet with or without some assistive device (cane, walker, crutches, self-propelled wheelchair) and (a) be able to sit upright with thighs together and arms crossed without requiring support on more than two sides; (b) be able to transfer from bed to chair independently or with minimal assistance if, in the opinion of the investigator, the degree of physical disability does not result in undue risk to the subject while participating in the study; and (c) perform basic daily care, such as feeding themselves and personal hygiene, with minimal assistance. 4. Subjects must weigh \> 40 kilograms (kg). Exclusion Criteria: 1. Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only one allele) for Friedreich's ataxia. 2. Subject requires use of amiodarone. 3. Subject used erythropoietin, etravirine, or gamma interferon within 3 months prior to screening. 4. Subject use of investigational drug (other than CTI-1601) or device within 90 days prior to screening. 5. Subject use of daily biotin supplementation that exceeds 30 mcg/day, either as part of a multivitamin or as a standalone supplement, within 7 days prior to study drug administration and/or throughout the entire study. 6. Subject has clinically significant arrhythmia on electrocardiogram (ECG), or evidence of predisposition to significant ventricular arrhythmia on ECG, or evidence of active and unstable coronary artery disease. 7. Male subject who has an ECG QTcF \> 450 milliseconds or female subject who has an ECG QTcF \> 470 milliseconds. 8. Subject has a screening echocardiogram ejection fraction \<45 percent. 9. Subject has a history of aspiration, aspiration pneumonia, or recurrent episodes of pneumonia (greater than or equal to 2 episodes of pneumonia) within the last 12 months. 10. Subjects with known or suspected chronic use of cannabinoid products.