Long-term CTI-1601 (nomlabofusp) treatment for Friedreich's ataxia

An Open-Label Extension Study to Assess the Long-Term Safety, Efficacy, Pharmacodynamics, Pharmacokinetics, and Tolerability of Subcutaneous CTI-1601 in Subjects With Friedreich's Ataxia

This trial tests daily subcutaneous CTI-1601 (nomlabofusp) in children (age 2+), adolescents, and adults with Friedreich's ataxia to see long-term safety, drug levels, biological effects, and clinical response.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 85 (estimated)
• Ages: 2 Years to 60 Years
• Sex: All
• Sponsor: Larimar Therapeutics, Inc. (Industry-sponsored)
• Locations: 8 sites (Los Angeles, California and 7 other locations)
• Trial ID: NCT06447025 on ClinicalTrials.gov

What this trial studies

This open-label Phase 2 program gives participants daily subcutaneous doses of CTI-1601 (nomlabofusp) over an extended period to monitor long-term safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD). The study measures tissue frataxin concentrations, gene expression and selected lipids, along with standardized clinical assessments of FRDA symptoms. Enrollment includes children aged 2 years and older, adolescents and adults, and both participants who previously received CTI-1601 in earlier studies and those who are treatment-naïve. Subjects with prior CTI-1601–related serious or significant adverse events, or other sponsor-determined exclusions, are not eligible to re-enroll.

Who should consider this trial

Good fit: Ideal candidates are people aged 2 years and older with genetically confirmed Friedreich's ataxia who can perform required clinic visits and do not have prior CTI-1601–related serious or significant adverse events.

Not a fit: Patients who previously had serious or grade 3+ CTI-1601–related adverse events, who have uncontrolled diabetes (HbA1c > 7.0%), or who cannot tolerate or perform subcutaneous dosing or required assessments are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, CTI-1601 could increase frataxin levels and potentially slow or improve neurologic symptoms in people with Friedreich's ataxia.

How similar studies have performed: Early-phase studies of CTI-1601 have been conducted and provided initial safety and PK data, but long-term clinical benefit in FRDA has not yet been established.

Eligibility criteria

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.

Where this trial is running

Los Angeles, California and 7 other locations

• University of California Los Angeles — Los Angeles, California, United States (Recruiting)
• Fixel Institute for Neurological Disease, University of Florida Health — Gainesville, Florida, United States (Active_not_recruiting)
• Morsani Center for Advanced Health Care, University of South Florida Health — Tampa, Florida, United States (Recruiting)
• University of Iowa — Iowa City, Iowa, United States (Recruiting)
• Uncommon Cures — Chevy Chase, Maryland, United States (Recruiting)
• Clinilabs Drug Development, Corp. — Eatontown, New Jersey, United States (Recruiting)
• Ohio State University United States — Columbus, Ohio, United States (Active_not_recruiting)
• Children's Hospital of the University of Pennsylvania (CHOP) — Philadelphia, Pennsylvania, United States (Recruiting)

Study contacts

• Study coordinator: Larimar Therapeutics, Inc.

Conditions Friedreich Ataxia