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

Phase 1

Completed

• Conditions: Friedreich Ataxia
• Interventions: Biological: Placebo; Biological: CTI-1601

• Registration Number: NCT04176991
• Lead Sponsor: Larimar Therapeutics, Inc.

Brief Summary

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

Detailed Description

Single Ascending Dose (SAD), Double-Blind, Placebo Controlled Study.

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

Secondary Objectives:

1. To evaluate the pharmacokinetics (PK) of CTI-1601 following increasing single doses of subcutaneously (SC) administered CTI-1601.

2. To evaluate the pharmacodynamics (PD) of CTI-1601 following increasing single doses of SC administered CTI-1601.

CTI-1601 or Placebo - Dose/Mode of Administration: Single Dose/Subcutaneous

Study Timeline

• Study First Submitted: 2019-11-14
• Study First Submitted QC: 2019-11-22
• Study First Posted: 2019-11-26
• Study Start: 2019-12-11
• Primary Completion: 2020-10-31
• Study Completion: 2020-10-31
• Status Verified: 2020-11
• Last Update Submitted: 2020-11-09
• Last Update Posted: 2020-11-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 28

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).

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	-
CTI-1601	CTI-1601	-

Primary Outcome Measures

Name	Time	Method
Number of Participants with Treatment-Emergent Adverse Events	Through study completion, an average of 70 days	Overall summary of the Participants with Treatment Emergent Adverse Events
Number of Treatment Emergent Adverse Events by System Organ Classification and Preferred Term	Through study completion, an average of 70 days	Overall summary of Participants with Treatment Emergent Adverse Events by System Organ Classification (MedDRA version 22.0)

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetics - Area under the concentration-time curve after a single dose	Up to 48 hours	Summary assessment of changes in the area under the concentration-time curve after a single dose
Pharmacokinetics - Time to reach maximum plasma concentration after a single dose	Up to 48 hours	Summary assessment of changes in time to reach maximum plasma concentration after a single dose
Pharmacokinetics - Apparent total plasma clearance	48 hours	Summary assessment of changes in the apparent total plasma clearance
Pharmacokinetics - Terminal half-life estimation	48 hours	Summary assessment of changes in the terminal half-life estimation
Pharmacokinetics - Apparent volume of distribution	48 hours	Summary assessment of changes in the apparent volume of distribution
Changes in Gene Expression Profiling	At baseline and up to 10 days	Summary assessment of changes in gene expression levels
Pharmacokinetics - Maximum observed plasma concentration after a single dose	Up to 48 hours	Summary assessment of changes in the maximum observed plasma concentration after a single dose
Pharmacokinetics - Area under the concentration-time curve from time 0 to infinity	48 hours	Summary assessment of changes in the area under the concentration-time curve from time 0 to infinity
Pharmacokinetics - Area under the concentration-time curve from time 0 to the last measurable time point	48 hours	Summary assessment of changes in the Area under the concentration-time curve from time 0 to the last measurable time point
Changes from Baseline in Frataxin Levels in Buccal Cells	At baseline and up to 10 days	Summary assessment of changes in frataxin levels in buccal cells
Changes from Baseline in Frataxin Levels in Whole Blood	At baseline and up to 10 days	Summary assessment of changes in frataxin levels in whole blood

Trial Locations

Locations (1)

Clinilabs Drug Development Corporation; 🇺🇸 Eatontown, New Jersey, United States