Recruiting
Phase 2

CTI-1601

Sponsor:

Larimar Therapeutics, Inc.

Code:

NCT06447025

Conditions

Friedreich Ataxia

Eligibility Criteria

Sex: All

Age: 2 - 60

Healthy Volunteers: Not accepted

Interventions

CTI-1601

Study Details

Brief 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.

Conditions

Friedreich Ataxia

Study ID

NCT06447025

Start date

Jan 25, 2024

Status verified date

Oct, 2026

Completion date

Jan, 2027

Anticipated

Primary completion date

Jan, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 60

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

85 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: CTI-1601

Once daily subcutaneous injection of 50 mg CTI-1601 in subjects ≥ 18 years of age or a weight-based dose of 0.8 mg/kg up to a maximum of 50 mg in subjects ≥ 2 to 17 years of age.

Interventions

CTI-1601

CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in patients with Friedreich's ataxia

Primary outcome measure

  • Number of subjects with treatment-emergent adverse events (TEAEs) by System Organ Class (SOC), Preferred Term (PT) and Maximum Severity [ Time Frame: Up to 24 months ]
  • Change from baseline in electrocardiogram (ECG) parameters including, but not limited to, HR, RR interval, PR interval, QRS duration, QT interval, and QTcF interval [ Time Frame: Up to 24 months ]
  • Change from baseline in left ventricular ejection fraction (LVEF) [ Time Frame: Up to 24 months ]
  • Change from baseline in left ventricular end-diastolic volume (LVEDV) [ Time Frame: Up to 24 months ]
  • Number of subjects with any suicidal ideation or behavior (Categories 1-10) of the Columbia Suicide Severity Rating Scale (C-SSRS) [ Time Frame: Up to 24 months ]
  • Change from baseline at each collection timepoint in tissue frataxin concentrations normalized to total protein observed in buccal cells collected from cheek swabs and skin cells collected from skin punch biopsies [ Time Frame: Up to 24 months ]
  • Change from baseline in motor function as assessed by 9-hole peg test (9-HPT) [ Time Frame: Up to 24 months ]
  • Change from baseline in motor function as assessed by the timed 25-foot walk test (T25-FW) [ Time Frame: Up to 24 months ]
  • Change from baseline in neurologic function as assessed by the modified Friedreich's Ataxia Rating Scale (mFARS) total score [ Time Frame: Up to 24 months ]
  • Change from baseline in neurologic function as assessed by the upright stability subscale examination of the mFARS [ Time Frame: Through study completion, up to 24 months ]
  • Change in activities of daily living (ADLs) as assessed by the Friedreich's Ataxia Rating Scale Activities of Daily Living (FARS_ADL) [ Time Frame: Up to 24 months ]
  • Change from baseline in total fatigue score and all the subscale scores as assessed by the Fatigue Impact Scale (MFIS) [ Time Frame: Up to 24 months ]
  • Change from baseline in the assessment of disease as assessed by the Functional Staging for Ataxia [ Time Frame: Up to 24 months ]
  • Overall impression of change as assessed by the patient using the Patient Global Impression of Change (PGI-C) Scale [ Time Frame: Up to 24 months ]
  • Overall impression of change assessed by a clinician using the Clinical Global Impression of Change (CGI-C) [ Time Frame: Up to 24 months ]
  • Area under the concentration-time curve for the dosing interval (AUC0-tau) [ Time Frame: Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months ]
  • Area under the concentration-time curve from time 0 to the time of last quantifiable concentration (AUC0-t) [ Time Frame: Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months ]
  • Mean maximum observed concentration (Cmax) [ Time Frame: Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months ]
  • Mean time of maximum observed concentration (Tmax) [ Time Frame: Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months ]
  • Concentration reached immediately before the next dose is administered (Ctrough) [ Time Frame: Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months ]

Central Contacts and Locations

Central contacts

Larimar Therapeutics, Inc.

844-511-9056medicalinfo@larimartx.com

Locations

University of California, San Franciso Medical Center

Recruiting

San Francisco, California, United States, 94143

Contacts

Ria Guerrero

(415) 514-4832maria.guerrero2@ucsf.edu

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

Recruiting

Tampa, Florida, United States, 33612

Contacts

Kayla Zayas

813-974-5909Kaylazayas@usf.edu

Lucretia Campbell

813-974-5909lcampbel@usf.edu

University of Iowa Hospitals & Clinics

Recruiting

Iowa City, Iowa, United States, 52242

Contacts

Corey McDaniel, BS

319-335-7498corey-mcdaniel@uiowa.edu

Uncommon Cures

Recruiting

Chevy Chase, Maryland, United States, 20815

Contacts

Rachael Batabyal, MD

240-858-4938Rbatabyal@uncommoncures.com

Tam Roshan Lal, MB, ChB

240-858-4912troshanlal@uncommoncures.com

Clinilabs Drug Development, Corp.

Recruiting

Eatontown, New Jersey, United States, 07724

Contacts

Recruiting Department

(212) 994-4567

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

David Lynch, MD, PhD

215-590-2242lynchd@pennmedicine.upenn.edu

McKenzie Wells, MS

267-426-9608wellsm@chop.edu

ICON

Recruiting

Salt Lake City, Utah, United States, 84124

Contacts

801-269-8200

More Information

Sponsor

Larimar Therapeutics, Inc.

Last update posted

Oct 9, 2026

Last verified

Oct, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-23. This information was provided to ClinicalTrials.gov by Larimar Therapeutics, Inc. on 2026-10-09. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.