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NCT06447025PHASE2Recruiting

An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia

Larimar Therapeutics, Inc.

Start Date

1/25/2024

Completion Date

1/1/2027

Summary

An open label study designed to evaluate the safety, PK, PD, and clinical effects of long-term daily administration of CTI-1601 enrolling adolescent and adult patients with FRDA who have participated in a prior clinical study of CTI-1601 as well as children (age 2 years and older), adolescents and adults with FRDA who have not participated in a prior clinical study of CTI-1601.

Detailed Description

An open-label study designed to evaluate the long-term safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of subcutaneous (SC) administration of CTI-1601, also known as nomlabofusp, in subjects with Friedreich's ataxia (FRDA). The objectives of this study are: * To evaluate the safety and PK of long-term subcutaneous (SC) administration of CTI-1601 in subjects with FRDA * To evaluate the PD and clinical effects of long-term subcutaneous (SC) administration of CTI-1601 in subjects with FRDA on: * Tissue FXN concentrations * Clinical evaluations of FRDA * Gene expression and select lipids

Eligibility Criteria

Age Range: 2 years to 60 years

Inclusion Criteria: * Subjects with FRDA who have or have not previously completed participation in a study of CTI-1601 are eligible to participate in this study unless the subject experienced one or more of the following in a previous CTI-1601 study: a) serious adverse event (SAE) related to study drug; b) significant AE, defined as Grade 3 or higher according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0 (or higher), related to study drug; c) some other event, related to participation in a previous study with CTI-1601, 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); d) Withdraw from participation in a previous study of CTI-1601 for any reason. * Subject has a HbA1c less than or equal to 7.0%. * Subject must demonstrate sufficient dexterity and visual acuity to prepare and self-administer SC injections of CTI-1601 QD or is able to identify a caregiver who will be trained and committed to prepare and administer the daily injections. If subject is taking permitted concomitant medication(s), subject must have been on a stable dose and frequency of medication(s) over the past 28 days prior to the initiation of Screening; however, subjects taking niacin and resveratrol must have been on a stable dose and frequency for 90 days prior to the initiation of Screening \- Subjects who are currently receiving omaveloxolone or intend to receive omaveloxolone are permitted in the study but must either receive CTI-1601 for 3 months prior to their first dose of omaveloxolone or receive omaveloxolone for 3 months prior to their first dose of CTI-1601. Exclusion Criteria: Subjects are excluded from the study if any of the following exclusion criteria are met: * Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only one allele) for FRDA. * Subject has any condition, disease, or situation, including a cardiac condition or disease, that in the opinion of the PI, could confound the results of the study or put the subject at undue risk, making participation inadvisable. * Subject used any investigational drug (other than CTI-1601) or device within 90 days prior to Screening. * Subject requires use of amiodarone. * Subject used erythropoietin, etravirine, or gamma interferon within 90 days prior to Screening. * Subject use of biotin supplementation that exceeds 30 mcg/day, either as part of a multivitamin or as a standalone supplement, within 7 days prior to the first dose of study drug. Biotin supplementation ≤30 mcg/day is permitted if taken at a stable dose and frequency for at least 28 days prior to Screening and there is a commitment from the subject to maintain the biotin dose throughout the study (due to interference with assays). * Subject uses more than 3 grams of acetaminophen daily. * Subject receives medication that requires SC injection in the abdomen or thigh. * Subject is unable to discontinue medications that have not been at a stable dose and frequency for at least 28 days prior to Screening. * Subject has a Screening echocardiogram (ECHO) LVEF \< 45%. * Male subject has a QTcF \> 450 milliseconds or female subject has a QTcF \> 470 milliseconds on an ECG.

Interventions

DRUG

CTI-1601

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Conditions

Friedreich Ataxia

Locations

University of California Los Angeles

Los Angeles, California 90095

United States

Fixel Institute for Neurological Disease, University of Florida Health

Gainesville, Florida 32608

United States

Morsani Center for Advanced Health Care, University of South Florida Health

Tampa, Florida 33612

United States

University of Iowa

Iowa City, Iowa 52242

United States

Uncommon Cures

Chevy Chase, Maryland 20815

United States

Clinilabs Drug Development, Corp.

Eatontown, New Jersey 07724

United States

Ohio State University United States

Columbus, Ohio 43210

United States

Children's Hospital of the University of Pennsylvania (CHOP)

Philadelphia, Pennsylvania 19104

United States