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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT04176991
• Date of registration: 14/11/2019
• Prospective Registration: Yes
• Primary sponsor: Larimar Therapeutics, Inc.
• Public title: Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
• Scientific title: A Phase 1 Single Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
• Date of first enrolment: December 11, 2019
• Target sample size: 28
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT04176991
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1

Countries of recruitment

United States

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Friedreich Ataxia

Intervention(s)

Biological: CTI-1601

Biological: Placebo

Primary Outcome(s)

Number of Participants with Treatment-Emergent Adverse Events [Time Frame: Through study completion, an average of 70 days]

Number of Treatment Emergent Adverse Events by System Organ Classification and Preferred Term [Time Frame: Through study completion, an average of 70 days]

Secondary Outcome(s)

Pharmacokinetics - Area under the concentration-time curve from time 0 to infinity [Time Frame: 48 hours]

Pharmacokinetics - Terminal half-life estimation [Time Frame: 48 hours]

Pharmacokinetics - Apparent total plasma clearance [Time Frame: 48 hours]

Pharmacokinetics - Area under the concentration-time curve from time 0 to the last measurable time point [Time Frame: 48 hours]

Changes in Gene Expression Profiling [Time Frame: At baseline and up to 10 days]

Pharmacokinetics - Time to reach maximum plasma concentration after a single dose [Time Frame: Up to 48 hours]

Pharmacokinetics - Apparent volume of distribution [Time Frame: 48 hours]

Pharmacokinetics - Maximum observed plasma concentration after a single dose [Time Frame: Up to 48 hours]

Changes from Baseline in Frataxin Levels in Buccal Cells [Time Frame: At baseline and up to 10 days]

Changes from Baseline in Frataxin Levels in Whole Blood [Time Frame: At baseline and up to 10 days]

Pharmacokinetics - Area under the concentration-time curve after a single dose [Time Frame: Up to 48 hours]

Secondary ID(s)

CLIN-1601-101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Metrum Research Group, LLC

Veristat, Inc.