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A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia

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ClinicalTrials.gov Identifier: NCT05579691
Recruitment Status : Completed
First Posted : October 14, 2022
Last Update Posted : February 16, 2024
Sponsor:
Information provided by (Responsible Party):
Larimar Therapeutics, Inc.

Study Description
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Brief Summary:
To evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) administration of CTI-1601 over 28 days in subjects with Friedreich's ataxia (FRDA).

Condition or disease Intervention/treatment Phase
Friedreich Ataxia Biological: CTI-1601 Other: Placebo Phase 2

Detailed Description:

This is a double-blind, placebo-controlled, study evaluating two doses (25 mg and 50 mg) of CTI-1601.

This study will consist of at least 2 cohorts with 12 to 15 subjects participating in each cohort. Subjects will be dosed once daily (QD) for 14 days followed by dosing every other day (QOD) through Day 28.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 28 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose Exploration Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CTI-1601 in Adult Subjects With Friedreich's Ataxia
Actual Study Start Date : September 21, 2022
Actual Primary Completion Date : December 4, 2023
Actual Study Completion Date : December 4, 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: CTI-160l
CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia
 Biological: CTI-1601
CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia
Placebo Comparator: Placebo
Placebo Comparator
 Other: Placebo
Placebo Comparator


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants with Treatment Emergent Adverse Events [ Time Frame: Through study completion, an average of 93 days ]
    Overall summary of Participants with Treatment Emergent Adverse Events


Secondary Outcome Measures :
  1. Maximum observed plasma concentration (Cmax) of CTI-1601 after multiple doses [ Time Frame: At baseline and up to 29 days ]
    Summary assessment of changes in the maximum observed plasma concentration (Cmax) of CTI-1601 after multiple doses

  2. Area under the concentration time curve (AUC) of CTI-1601 from time 0 through the last measurable time point [ Time Frame: At baseline and up to 29 days ]
    Summary assessment of changes in the AUC of CTI-1601 from time 0 to the last measurable time point and during the dosing interval

  3. Time to maximum observed plasma concentration (tmax) of CTI-1601 after multiple doses [ Time Frame: At baseline and up to 29 days ]
    Summary assessment of the time to maximum observed plasma concentration (tmax) of CTI-1601 after multiple doses

  4. Time to last observed plasma concentration (tlast) of CTI-1601 after multiple doses [ Time Frame: At baseline and up to 29 days ]
    Summary assessment of the time to last observed plasma concentration (tlast) of CTI-1601 after multiple doses

  5. Changes from baseline in frataxin levels in buccal cells [ Time Frame: At baseline and up to 58 days ]
    Summary assessment of changes in frataxin levels in buccal cells

  6. Changes from baseline in frataxin levels in skin punch cells [ Time Frame: At baseline and up to 29 days ]
    Summary assessment of changes in frataxin levels in skin punch cells


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Subject has a genetically confirmed diagnosis of FRDA manifested by homozygous GAA repeat expansions, with repeat sizing (if available) included on the diagnosis report.
  2. Subject is biologically male or female, 18 years of age or older at screening.
  3. Subject must have a mFARS score ≥ 20 and be able to traverse a distance of 25 feet with or without some assistive device (e.g., 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 assistance if, in the opinion of the PI, 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. Subject must weigh > 40.0 kg.

Exclusion Criteria:

Subjects are excluded from the study if any of the following exclusion criteria are met:

  1. If the subject previously participated in a study of CTI-1601 (CLIN-1601-101 (NCT04176991) or CLIN-1601-102 (NCT04519567)) the subject may not enroll in this study if they experienced one or more of the following: (a) Serious Adverse Event (SAE) related to study drug; (b) Adverse Event (AE) defined as Grade 3 or higher according to the CTCAE version 5.0 (or higher), related to study drug; (c) some other event that supports the exclusion of the subject from participating in this study as determined by the Sponsor (i.e., an AE considered clinically significant by the Sponsor regardless of whether it met SAE criteria and regardless of CTCAE grade).
  2. Subject who is confirmed as compound heterozygous (GAA repeat expansion on only one allele) for FRDA.
  3. Subject used an investigational drug or device within 90 days prior to screening.
  4. Subject requires use of amiodarone.
  5. Subject used erythropoietin, etravirine, or gamma interferon 90 days prior to Screening.
  6. Subject use of biotin supplementation that exceeds 30.0 mcg/day, either as part of a multivitamin or as a standalone supplement, within 7 days prior to the first dose of study drug.
  7. Subject uses more than 3.0 grams of acetaminophen daily.
  8. Subject receives medication that requires SC injection in the abdomen or thigh.
  9. Subject received a vaccination within 14 days of administration of the first dose of study drug or is scheduled to receive a vaccination within 14 days after administration of the last dose of study drug. As an exception, influenza and tetanus vaccines must be administered more than 72 hours prior to the first dose of study drug or 72 hours after the administration of the last dose of study drug.
  10. Subject has a screening ECHO LVEF < 45%.
  11. Male subject has a QTcF > 450 milliseconds or female subject has a QTcF > 470 milliseconds on an ECG.
  12. Subject currently receiving or having received omaveloxolone within 30 days prior to Screening.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05579691


Locations
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United States, New Jersey
Clinilabs Drug Development Corporation
Eatontown, New Jersey, United States, 07724
Sponsors and Collaborators
Larimar Therapeutics, Inc.
Investigators
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Principal Investigator: Magdy Shenouda, M.D. Clinilabs, Inc.
More Information
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Publications:

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Responsible Party: Larimar Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT05579691    
Other Study ID Numbers: CLIN-1601-200
First Posted: October 14, 2022    Key Record Dates
Last Update Posted: February 16, 2024
Last Verified: February 2024

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Ataxia
Cerebellar Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
 Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases