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

November 26, 2024 updated by: Larimar Therapeutics, Inc.

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

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).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

28

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New Jersey
      • Eatontown, New Jersey, United States, 07724
        • Clinilabs Drug Development Corporation

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Through study completion, an average of 93 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
At baseline and up to 29 days
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
At baseline and up to 29 days
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
At baseline and up to 29 days
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
At baseline and up to 29 days
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
At baseline and up to 58 days
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
At baseline and up to 29 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Larimar Therapeutics, Inc.

Investigators

  • Principal Investigator: Magdy Shenouda, M.D., Clinilabs, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 21, 2022

Primary Completion (Actual)

December 4, 2023

Study Completion (Actual)

December 4, 2023

Study Registration Dates

First Submitted

October 5, 2022

First Submitted That Met QC Criteria

October 11, 2022

First Posted (Actual)

October 14, 2022

Study Record Updates

Last Update Posted (Actual)

November 29, 2024

Last Update Submitted That Met QC Criteria

November 26, 2024

Last Verified

November 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CLIN-1601-200

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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