CompletedPhase 1

Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia

United States28 participantsStarted 2019-12-11

Plain-language summary

To evaluate the safety and tolerability of single ascending doses of CTI-1601 in participants with Friedreich's ataxia

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Subject has genetically confirmed Friedreich's ataxia diagnosis, homozygous GAA repeat expansions, with repeat sizing (if available) included on diagnostic report.
✓. Subject is male or female, 18 years of age or older at screening.
✓. 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.
✓. Subjects must weigh \> 40 kilograms (kg).

✕. Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only one allele) for Friedreich's ataxia.
✕. Subject requires use of amiodarone.
✕. Subject used erythropoietin, etravirine, or gamma interferon within 3 months prior to screening.
✕. Subject use of investigational drug (other than CTI-1601) or device within 90 days prior to screening.
✕. 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.
✕. 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.
✕. Male subject who has an ECG QTcF \> 450 milliseconds or female subject who has an ECG QTcF \> 470 milliseconds.
✕. Subject has a screening echocardiogram ejection fraction \<45 percent.

Number of Participants with Treatment-Emergent Adverse Events

Timeframe: Through study completion, an average of 70 days

Number of Treatment Emergent Adverse Events by System Organ Classification and Preferred Term

Timeframe: Through study completion, an average of 70 days

NCT IDNCT04176991

SponsorLarimar Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-10-31

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Subject has genetically confirmed Friedreich's ataxia diagnosis, homozygous GAA repeat expansions, with repeat sizing (if available) included on diagnostic report.
✓. Subject is male or female, 18 years of age or older at screening.
✓. 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.
✓. Subjects must weigh \> 40 kilograms (kg).

✕. Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only one allele) for Friedreich's ataxia.
✕. Subject requires use of amiodarone.
✕. Subject used erythropoietin, etravirine, or gamma interferon within 3 months prior to screening.
✕. Subject use of investigational drug (other than CTI-1601) or device within 90 days prior to screening.
✕. 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.
✕. 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.
✕. Male subject who has an ECG QTcF \> 450 milliseconds or female subject who has an ECG QTcF \> 470 milliseconds.
✕. Subject has a screening echocardiogram ejection fraction \<45 percent.