ClinicalTrials.gov record NCT06447025

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 7, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
Age2 years old to 60 years old
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 2 open-label study gives daily subcutaneous CTI-1601 to people with Friedreich's ataxia aged 2 to 60. It includes participants who previously received CTI-1601 and those new to the drug. Participants are followed for up to 24 months to collect safety, pharmacokinetics/pharmacodynamics, tissue frataxin, and clinical measures of motor and neurologic function.

What it is studying. The study tests long-term subcutaneous CTI-1601 (nomlabofusp) in people with Friedreich's ataxia to collect data on safety and tolerability, how the drug behaves in the body (pharmacokinetics), biological effects (pharmacodynamics and tissue frataxin levels), and effects on motor and neurologic function.

What the researchers hope to learn. To evaluate long-term safety and tolerability of CTI-1601; to characterize its pharmacokinetics and pharmacodynamics; to measure changes in tissue frataxin concentrations; and to collect clinical data on motor and neurologic function, gene expression, and select lipids.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • CTI-1601

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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 description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants will receive a daily subcutaneous injection of CTI-1601 (weight‑based dosing for children; up to a 50 mg fixed dose for adults) and attend regular clinic visits over a period of up to 24 months. Visits include heart monitoring (ECG and echocardiogram), blood samples for drug levels and safety, motor function tests (for example 9‑hole peg test and timed 25‑foot walk), questionnaires (fatigue and global impression scales), cheek swabs and skin punch biopsies to measure tissue frataxin, and safety assessments such as the Columbia Suicide Severity Rating Scale. Participants or their caregivers will be trained to prepare and give daily injections. Study staff will collect samples and monitor for side effects at scheduled time points through study completion.

How the study is structured. Phase 2, open-label, single-group interventional study. Participants receive once-daily subcutaneous CTI-1601 (weight-based dosing for children and a fixed dose for adults as specified in the protocol) and are followed with regular safety checks, blood sampling for PK/PD, tissue sampling for frataxin, and clinical assessments over up to 24 months. There is no placebo or masking.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Friedreich ataxia

Clinical trials26 trials listed for Friedreich ataxiaEvery trial the registry lists for Friedreich ataxia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Friedreich ataxia researchLatest monthly update: September 2026. Includes: Heart change stages. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jan 25, 2024
Primary completion
January 2027 (estimated)
Study completion
January 2027 (estimated)
Target enrollment
85 participants (estimated)
Last updated on registry
Oct 9, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Larimar Therapeutics, Inc.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (11)

Terms used on this page · explanations by MedicalUpdates.ai

open label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
PK
How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
serious adverse event
Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
study drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
Sponsor
The organisation or person that initiates the study and has authority over it. The sponsor may be a company, hospital, university, government agency or individual, and is not necessarily the organisation that funds the study.
concomitant medication
Other medicines a participant takes during the study. Some must be reported; some are restricted.
stable dose
A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
exclusion criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
prior to Screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
echocardiogram
An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
QTcF
A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
pharmacodynamics
What a drug does to the body, for example how it changes a blood marker or a symptom, and how that relates to the dose.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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