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

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ClinicalTrials.gov Identifier: NCT04176991
Recruitment Status : Completed
First Posted : November 26, 2019
Last Update Posted : November 12, 2020
Sponsor:
Collaborators:
Veristat, Inc.
Metrum Research Group, LLC
Information provided by (Responsible Party):
Larimar Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE November 14, 2019
First Posted Date  ICMJE November 26, 2019
Last Update Posted Date November 12, 2020
Actual Study Start Date  ICMJE December 11, 2019
Actual Primary Completion Date October 31, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 22, 2019)
  • Number of Participants with Treatment-Emergent Adverse Events [ Time Frame: 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 [ Time Frame: 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)
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 22, 2019)
  • Pharmacokinetics - Area under the concentration-time curve after a single dose [ Time Frame: Up to 48 hours ]
    Summary assessment of changes in the area under the concentration-time curve after a single dose
  • Pharmacokinetics - Maximum observed plasma concentration after a single dose [ Time Frame: Up to 48 hours ]
    Summary assessment of changes in the maximum observed plasma concentration after a single dose
  • Pharmacokinetics - Time to reach maximum plasma concentration after a single dose [ Time Frame: Up to 48 hours ]
    Summary assessment of changes in time to reach maximum plasma concentration after a single dose
  • Pharmacokinetics - Area under the concentration-time curve from time 0 to infinity [ Time Frame: 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 [ Time Frame: 48 hours ]
    Summary assessment of changes in the Area under the concentration-time curve from time 0 to the last measurable time point
  • Pharmacokinetics - Apparent total plasma clearance [ Time Frame: 48 hours ]
    Summary assessment of changes in the apparent total plasma clearance
  • Pharmacokinetics - Terminal half-life estimation [ Time Frame: 48 hours ]
    Summary assessment of changes in the terminal half-life estimation
  • Pharmacokinetics - Apparent volume of distribution [ Time Frame: 48 hours ]
    Summary assessment of changes in the apparent volume of distribution
  • Changes from Baseline in Frataxin Levels in Buccal Cells [ Time Frame: 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 [ Time Frame: At baseline and up to 10 days ]
    Summary assessment of changes in frataxin levels in whole blood
  • Changes in Gene Expression Profiling [ Time Frame: At baseline and up to 10 days ]
    Summary assessment of changes in gene expression levels
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
Official Title  ICMJE 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
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Friedreich Ataxia
Intervention  ICMJE
  • Biological: CTI-1601
    CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia
  • Biological: Placebo
    Placebo Comparator
Study Arms  ICMJE
  • Experimental: CTI-1601
    Intervention: Biological: CTI-1601
  • Placebo Comparator: Placebo
    Intervention: Biological: Placebo
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: November 9, 2020)
28
Original Estimated Enrollment  ICMJE
 (submitted: November 22, 2019)
32
Actual Study Completion Date  ICMJE October 31, 2020
Actual Primary Completion Date October 31, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04176991
Other Study ID Numbers  ICMJE CLIN-1601-101
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Larimar Therapeutics, Inc.
Study Sponsor  ICMJE Larimar Therapeutics, Inc.
Collaborators  ICMJE
  • Veristat, Inc.
  • Metrum Research Group, LLC
Investigators  ICMJE Not Provided
PRS Account Larimar Therapeutics, Inc.
Verification Date November 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP