A Phase 1 interventional study of NBIB-0000223 in Friedreich's Ataxia, sponsored by Neurocrine Biosciences. Not yet recruiting at 5 sites in United States. Open to participants aged 8 Years to 40 Years. Per ClinicalTrials.gov, last updated 2026-11-12.
Sponsored by Neurocrine Biosciences · Phase 1, Interventional, and Treatment
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Among 71 studies in Friedreich Ataxia with a site in United States — 18 open to participants now, 25 under way, 4 of them phase 3. Friedreich Ataxia · United States.
Among the 71 studies in Friedreich Ataxia with a site in United States, this one is the 33rd largest by planned enrollment of the 49 interventional ones that give a count, is one of 8 that started in 2026, is one of 15 in phase 1, reaches primary completion later than 12 of the 24 other live ones, and was the 71st registered.
111 studies on the registry are indexed under Friedreich Ataxia; 28 are open to participants now.
Browse Friedreich Ataxia studies →18 of the 71 studies in Friedreich Ataxia with a site in United States are open now. Recruiting first, the newest first:
Biogen · Sites in United States
Ages 16+ · Women only
Larimar Therapeutics, Inc. · Sites in United States
Ages 12–40
Scott Barbuto · Sites in United States
Lexeo Therapeutics · Sites in United States
Ages 6+
Solid Biosciences Inc. · Sites in United States
Ages 18–40
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: 8 Years to 40 Years · all sexes · no healthy volunteers.
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71 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 18 · Phase 2 19 · Phase 3 12
First in 2001; this study’s year is marked. 8 started in 2026.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 24 is below the median of 30 across 76 interventional studies indexed under Friedreich Ataxia.
Median enrollment 32 across the 49 interventional studies, 65 across the 21 observational. Of the 43 that completed or were terminated, 16 (37%) have results posted.
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39 of 71 have an industry lead sponsor. The 10 most frequent:
Neurocrine Biosciences is the lead sponsor of 87 studies on the registry; 17 are open to participants now.
Of its 35 completed or terminated interventional studies of FDA-regulated products, 23 (66%) have results posted.
Neurocrine Biosciences has 2 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 |
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Among the 71 studies in Friedreich Ataxia with a site in United States, from their registry updates:
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This is a first-in-human study to evaluate the safety, tolerability, and pharmacodynamics of NBIB-0000223 (hereafter referred to as NBIB-223) in participants with Friedreich's Ataxia.
Key Inclusion Criteria:
Has both a clinical and genetic diagnosis of Friedreich's Ataxia, with diagnosis occurring at least 1 year prior to Screening and when the participant was age ≤16 years.
Key Exclusion Criteria:
Additional inclusion/exclusion criteria may apply, per protocol.
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 reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
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.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
TIA (transient ischaemic attack)
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
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.
Dose escalation in adult participants.
Drug: NBIB-0000223
Dose expansion in pediatric participants.
Drug: NBIB-0000223
Dose expansion in adult and/or pediatric participants with FA-related cardiomyopathy.
Drug: NBIB-0000223
Intravenous (IV) infusion
Also known as: NBIB-223
Number of Participants with Treatment-emergent Adverse Events
Time frame: Day 1 through 48-Week Post-Treatment Follow-Up
Change from Baseline in Frataxin Protein Expression
Time frame: Baseline, up to 48-Week Post-Treatment Follow-Up
Plan to share: No
No publications or documents are linked to this record.
Start is estimated for Nov 2026; primary completion for Nov 2028, 24 months later.
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 ↗
This study is not yet recruiting, as verified in Nov 2026. You cannot join it, but the record below documents what was studied.
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