A Phase 3 interventional study of Nomlabofusp and Placebo in Friedreich Ataxia and Friedreich's Ataxia, sponsored by Larimar Therapeutics, Inc.. Recruiting at 4 sites in United States. Open to participants aged 12 Years to 40 Years. Per ClinicalTrials.gov, last updated 2026-10-09.
Sponsored by Larimar Therapeutics, Inc. · Phase 3, Interventional, and Treatment
Among 70 studies in Friedreich Ataxia with a site in United States — 17 open to participants now, 24 under way, 4 of them phase 3. Friedreich Ataxia · United States.
Among the 70 studies in Friedreich Ataxia with a site in United States, this one is the 3rd largest by planned enrollment of the 48 interventional ones that give a count, is one of 7 that started in 2026, is one of 10 in phase 3, reaches primary completion later than 14 of the 23 other live ones, and was the 68th registered.
110 studies on the registry are indexed under Friedreich Ataxia; 27 are open to participants now.
Browse Friedreich Ataxia studies →17 of the 70 studies in Friedreich Ataxia with a site in United States are open now. Recruiting first, the newest first:
Biogen · Sites in United States
Scott Barbuto · Sites in United States
Lexeo Therapeutics · Sites in United States
Solid Biosciences Inc. · Sites in United States
Lexeo Therapeutics · Sites in United States, Brazil, Canada and 5 more countries
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 18 Friedreich Ataxia studies recruiting now, 9 (50%) set no upper age limit and 9 (50%) stop at 75 or younger; 15 take children under 18; 3 take healthy volunteers.
This study: 12 Years to 40 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
70 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 17 · Phase 2 19 · Phase 3 12
First in 2001; this study’s year is marked. 7 started in 2026.
This study's planned enrollment of 150 is above the median of 30 across 75 interventional studies indexed under Friedreich Ataxia.
Median enrollment 33 across the 48 interventional studies, 65 across the 21 observational. Of the 43 that completed or were terminated, 16 (37%) have results posted.
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38 of 70 have an industry lead sponsor. The 10 most frequent:
Larimar Therapeutics, Inc. is the lead sponsor of 8 studies on the registry; 2 are open to participants now.
Larimar Therapeutics, Inc. has 8 studies filed under Friedreich Ataxia — fewer than ten, so there is no comparison to draw.
6 of the studies in Friedreich Ataxia with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06953583Phase 3BiogenA Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia | 2027-11-16 |
| NCT06447025Larimar Therapeutics, Inc.An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia | 2027-01 |
| NCT07869654Adjuvia Therapeutics, Inc.A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults | 2027-05-27 |
| NCT07870590Graviton Bioscience CorporationGV101 for Friedreich's Ataxia (FA) Treatment | 2027-07 |
| NCT02316314Weill Medical College of Cornell UniversityCharacterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA) | 2027-08 |
Among the 70 studies in Friedreich Ataxia with a site in United States, from their registry updates:
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To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia
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:
Key Inclusion Criteria:
Subjects who meet all of the following criteria are potentially eligible for study participation:
Subject must have the following at Screening and Day -1 per the following upright stability items from Module E of the mFARS:
Key Exclusion Criteria
Subjects are excluded from the study if any of the following exclusion criteria are met:
Subject has a on an as specified below:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Daily subcutaneous injection of nomlabofusp for 72 weeks
Drug: Nomlabofusp
Daily subcutaneous injection of placebo for 72 weeks
Drug: Placebo
Nomlabofusp is a recombinant fusion protein provided in a sterile, preservative-free buffered solution for subcutaneous injection intended to deliver human frataxin, the protein deficient in Friedreich's ataxia.
Also known as: CTI-1601
The placebo is a sterile, preservative-free, clear liquid for subcutaneous injection.
Also known as: CTI-1601 Placebo, Nomlabofusp Placebo
Change from baseline in Upright Stability Score (USS) subscale E of modified Friedreich's Ataxia Rating Scale (mFARS) at Week 72 (Region-specific)
Number - The USS has a score range from 0 to 36. Lower scores mean less impairment and higher scores mean greater neurological impairment.
Time frame: Baseline, Weeks 12, 24, 36, 48, 72
Change from baseline in the total modified Friedreich's Ataxia Rating Scale (mFARS) at Week 72 (Region-specific)
Number - The mFARS has a total score range from 0 to 93. Lower scores mean less impairment and higher scores mean greater neurological impairment.
Time frame: Baseline, Weeks 12, 24, 36, 48, 72
Change from baseline in the Clinical Global Impression-Severity (CGI-S) score at Week 72
Number - The CGI-S is a 7-point scale. Lower scores mean less impairment and higher scores mean greater neurological impairment.
Time frame: Baseline, Weeks 12, 24, 36, 48, 72
Plan to share: No
Start is estimated for Oct 2026; primary completion for Jul 2029, 33 months later.
Start moved once since we started tracking on Sep 25, 2026: Aug 2026 → Oct 2026, on Oct 9, 2026.
Earlier changes are on the record history on ClinicalTrials.gov ↗
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
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Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Nomlabofusp
Larimar Therapeutics, Inc.