Phase 2 Friedreich Ataxia Trial, Recruiting NCT06447025 Sponsor: Larimar Therapeutics, Inc. Condition: Friedreich Ataxia
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Phase 2 Friedreich Ataxia Trial, Recruiting

NCT06447025
Recruiting Phase 2

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

  • You have Friedreich's ataxia (FRDA) and either have never been in a previous CTI-1601 study, or you completed one without experiencing serious side effects, severe reactions, or withdrawing early.
  • Your HbA1c (a blood sugar level test) is 7.0% or below.
  • You are able to give yourself a daily injection under the skin, or you have a caregiver who is willing to be trained to do this for you each day.
  • If you take any allowed medications, you have been on the same dose for at least the past 28 days before screening starts (or 90 days for niacin or resveratrol specifically).
  • If you take or plan to take omaveloxolone (another medication), you must have been on CTI-1601 for 3 months first, or on omaveloxolone for 3 months before starting CTI-1601.

Who may not be able to join:

  • Your genetic test shows you have a specific type of Friedreich's ataxia called "compound heterozygous" (where only one copy of the gene has the typical repeat expansion) (confirm with trial site).
  • You have any health condition — including a heart condition — that the study doctor believes could affect the results or put you at risk.
  • You have taken another experimental drug or used an experimental device within the past 90 days.
  • You need to take a heart medication called amiodarone.
  • You have used erythropoietin, etravirine, or gamma interferon within the past 90 days.
  • You have taken more than 30 mcg per day of biotin (a B-vitamin supplement) in the 7 days before your first dose.
  • You take more than 3 grams of acetaminophen (e.g., Tylenol/paracetamol) per day.
  • You already receive injections under the skin of your abdomen or thigh for another medication.
  • You are taking medications that have not been at a stable dose for at least 28 days before screening and cannot stop them.
  • Your heart pumping function (called LVEF, measured by an ultrasound of the heart) is below 45%.
  • Your heart's electrical activity reading (ECG) shows a specific measurement called QTcF that is too high — over 450 milliseconds for males or over 470 milliseconds for females.

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 28 July 2026
Phase 2: approximately ~30% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

Contact this trial

Principal Investigator: Larimar Therapeutics, Inc., Larimar Therapeutics, Inc.

Phone: 844-511-9056

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Trial details

Status
Recruiting
Phase
Phase 2
Sponsor
Larimar Therapeutics, Inc.
Registry
ClinicalTrials.gov
Start date
25 January 2024
Est. completion
1 January 2027

Where this trial is recruiting

🇺🇸 United States

Primary endpoints

Number of subjects with treatment-emergent adverse events (TEAEs) by System Organ Class (SOC), Preferred Term (PT) and Maximum Severity; Change from baseline in electrocardiogram (ECG) parameters including, but not limited to, HR, RR interval, PR interval, QRS duration, QT interval, and QTcF interval; Change from baseline in left ventricular ejection fraction (LVEF); Change from baseline in left ventricular end-diastolic volume (LVEDV); Number of subjects with any suicidal ideation or behavior (Categories 1-10) of the Columbia Suicide Severity Rating Scale (C-SSRS); Change from baseline at eac...

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov