ClinicalTrials.gov record NCT07778836

A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia

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

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp 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 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
Age12 years old to 40 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 3, randomized, double-blind, placebo-controlled trial tests daily subcutaneous nomlabofusp in people with Friedreich's ataxia. Participants receive daily injections for 72 weeks and have assessments at baseline and at Weeks 12, 24, 36, 48, and 72. Main measures include Upright Stability Score (USS) subscale E, total modified Friedreich's Ataxia Rating Scale (mFARS), and Clinical Global Impression of Severity (CGI-S); safety and tolerability are also evaluated.

What it is studying. The study tests the efficacy and safety of subcutaneous nomlabofusp compared with placebo in people with Friedreich's ataxia. It measures change from baseline at Week 72 in the USS Subscale E and total mFARS scores, and in the CGI-S score, plus safety and tolerability over the treatment period.

What the researchers hope to learn. To evaluate whether nomlabofusp changes neurological function as measured by USS subscale E and total mFARS at Week 72, to assess CGI-S scores, and to assess the safety and tolerability of daily subcutaneous nomlabofusp.

What this study is about

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

To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia

Interventions being studied

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

Drug
  • Nomlabofusp
  • Placebo

Eligibility

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

Inclusion criteria

  • Subjects who meet all of the following criteria are potentially eligible for study participation:
  • Subject must provide genetically confirmed FRDA diagnosis report and is homozygous for GAA repeat expansions documented on the genetic diagnostic report, with repeat sizing (if available).
  • Subject must complete 1 trial at Screening and 1 trial at Day -1 of the T25-FW test, using their customary assistive device (e.g., cane, 2 canes/crutches [Canadian crutches], wheeled walker/rollator or canine assistance) if needed. Each trial must be completed within 3 minutes.
  • Subject must have the following at Screening and Day -1 per the following upright stability items from Module E of the mFARS:
  • Item #E1, Sitting Posture - no more than a maximum score of 2
  • Item #E2A, Stance Feet Apart - no more than a maximum score of 2 (average of 3 attempts)
  • Subject must have an mFARS score ≥ 20 and < 60 at Screening and Day -1.
  • Subject must have a Functional Staging for Ataxia score of 4 or less at Screening.
  • Subject demonstrates sufficient dexterity and visual acuity to prepare and self-administer SC injections of study drug daily (QD) or has an identified caregiver who will be trained and committed to prepare and administer the injections.
  • Subject has a Screening HbA1c ≤ 7.0%.
  • If the 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 initiation of Screening. Subjects taking niacin and resveratrol must have been on a stable dose and frequency for 90 days prior to initiation of Screening and subjects taking omaveloxolone must have been on a stable dose and frequency for 1 years prior to initiation of Screening.
  • Key

Exclusion criteria

  • Subjects are excluded from the study if any of the following exclusion criteria are met:
  • Subject who is confirmed as compound heterozygous (GAA repeat expansion on only 1 allele) for FRDA.
  • Subject previously participated in a clinical trial involving nomlabofusp. Participation is defined as the subject having signed the informed consent for the study and received at least 1 dose of study drug (nomlabofusp or placebo).
  • The subject has any condition, disease, or situation that could confound the results of the study or put the subject at undue risk, making participation inadvisable in the opinion of the PI.
  • Women of childbearing potential who are pregnant (have a positive pregnancy test at Screening or Day -1), lactating, or planning to attempt to become pregnant during this study or within 90 days after the last dose of study drug (this includes male subjects with partners of childbearing potential who are attempting to become pregnant).
  • Subject used any investigational drug or device within 90 days prior to the initiation of Screening.
  • Subject previously received a gene therapy (investigational or approved) at any time in the past.
  • Subject requires use of amiodarone.
  • Subject used erythropoietin, etravirine, or gamma interferon within 90 days prior to the initiation of Screening.
  • Subject's use of biotin supplementation exceeds 30 μg/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 μg/day is permitted if taken at a stable dose and frequency for at least 28 days prior to the initiation of Screening and there is a commitment from the subject to maintain the biotin dose throughout the study (due to interference with assays).
  • Subject receives medication that requires SC injection in the abdomen or thigh.
  • Subject has a Screening ECHO left ventricular ejection fraction < 45%.
  • Subject has a QTcF on an ECG as specified below:
  • For subjects ≥ 12 and < 18 years of age, a male or female subject with a QTcF > 460 ms.
  • For subjects ≥ 18 years of age, a male subject with a QTcF > 450 ms or a female subject has a QTcF > 470 ms.
  • Subject has suicidal ideation as determined by a "yes" to item #2 on the C-SSRS at Screening (within the last 28 days) or at Day -1.

Study description and what participation involves

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

Detailed description from the registry

This is a global, randomized, multi-center, placebo-controlled study to investigate the efficacy and safety of nomlabofusp in subjects with Friedreich's ataxia The main objectives of the study are to evaluate:

  • The efficacy of subcutaneous (SC) administration of nomlabofusp compared to placebo at Week 72; 1) on the Upright Stability Score (USS) Subscale E of the Modified Friedreich's Ataxia Ratings Scale (mFARS), or 2) mFARS total score (Region specific objective)
  • The efficacy of SC administration of nomlabofusp compared to placebo at Week 72 on the Clinical Global Impression of Severity (CGI-S) score
  • The safety and tolerability of SC administration of nomlabofusp
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 are randomly assigned to daily subcutaneous injections of nomlabofusp or placebo for 72 weeks; injections may be self‑administered or given by a trained caregiver. They will attend clinic visits for assessments at baseline and at Weeks 12, 24, 36, 48, and 72, including the Upright Stability Score (mFARS Subscale E), the total modified Friedreich's Ataxia Rating Scale (mFARS), and the Clinical Global Impression–Severity (CGI‑S). At screening and Day −1 participants complete timed walking trials (T25‑FW); the study also includes ongoing safety and tolerability evaluations. The registry record does not list other invasive procedures such as spinal taps or biopsies.

How the study is structured. Participants are randomly assigned to one of two parallel groups: daily subcutaneous nomlabofusp or matching placebo. The study is quadruple-masked (participants, care providers, investigators, and outcome assessors blinded) and runs for 72 weeks of treatment with assessments at baseline and Weeks 12, 24, 36, 48, and 72. This is a Phase 3, multicenter, placebo-controlled trial.

Questions you could ask

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

  • Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
  • 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
Oct 26, 2026 (estimated)
Primary completion
July 2029 (estimated)
Study completion
December 2029 (estimated)
Target enrollment
150 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 (4)

Terms used on this page · explanations by MedicalUpdates.ai

at Screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
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.
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.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
placebo
A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
Women of childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
ECHO
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.
Key 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.
randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
placebo-controlled
A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.

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 website; 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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