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Experimental Friedreich's ataxia drug tested in kids – but trial halted early

NCT ID NCT06681766

Disease control Sponsor: Larimar Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 10, 2026 · Updated 1 time

Summary

This early-stage trial tested a drug called nomlabofusp (CTI-1601) in 18 adolescents and children with Friedreich's ataxia, a rare genetic disease that affects movement and coordination. The goal was to check safety and how the body processes the drug. However, the study was terminated, so results may be limited.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
nomlabofusp (CTI-1601)
What this could lead to
If it works, this could point toward a treatment that helps manage Friedreich's ataxia by delivering a missing protein.
What could go wrong
This was a very early (phase 1) trial focused on safety, not effectiveness. It was terminated, so results may not be available. The study was small (18 participants) and limited to specific age and weight groups.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

18 people

The number who actually took part.

Started

Dec 2024

Finished

Apr 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Subject has genetically confirmed diagnosis of FRDA manifested by homozygous GAA repeat expansions, with repeat sizing (if available) included on the diagnosis report. 2. Male or female subjects ≥ 2 to \< 18 years of age at screening. 3. Subjects must weigh ≥ 10.0 kg. 4. Subject must be able to traverse a distance of 25 feet with or without some assistive device (e.g., cane, walker, crutches, self-propelled wheelchair) and meet the following requirements: 1. Be able to sit upright with thighs together and arms crossed without requiring support on more than 2 sides; 2. Be able to transfer from bed to chair independently or with assistance if, in the opinion of the investigator, the degree of physical disability does not result in undue risk to the subject while participating in the study; and 3. Perform basic age-appropriate daily care, such as feeding themselves and personal hygiene, with minimal assistance. Exclusion Criteria: 1. Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only 1 allele) for FRDA. 2. Subject has any condition, disease, or situation, including a cardiac condition or disease, that in the opinion of the investigator could confound the results of the study or put the subject at undue risk, making participation inadvisable. 3. Subjects currently receiving or having received omaveloxolone within 30 days prior to Screening.

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Conditions

The condition(s) this trial relates to.

Friedreich ataxia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Friedreich ataxia

Contacts and locations

Locations

  • Uncommon Cures

    Chevy Chase, Maryland, 20815, United States

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