Study of NBIB-223 Gene Therapy in Friedreich's Ataxia (FACET)

November 6, 2026 updated by: Neurocrine Biosciences

Phase 1/2, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Efficacy of a Single Intravenous Infusion of NBIB-0000223 in Patients With Friedreich's Ataxia

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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

24

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90095
        • Neurocrine Clinical Site
    • Florida
      • Gainesville, Florida, United States, 32608
        • Neurocrine Clinical Site
      • Tampa, Florida, United States, 33612
        • Neurocrine Clinical Site
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Neurocrine Clinical Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19406
        • Neurocrine Clinical Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

8 years to 40 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

    • Documented prior genetic diagnosis of Friedreich's Ataxia with a pathogenic GAA trinucleotide repeat expansion in intron 1 of both frataxin alleles, based on analysis by a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory.
    • Clinical evidence of symptomatic neurologic disease consistent with Friedreich's Ataxia.
    • Have an Modified Friedreich Ataxia Rating Scale (mFARS) overall score ≥15 and <80, and an Modified Friedreich Ataxia Rating Scale subscale E: Upright Stability (mFARS-E) gait score of ≥1.

  • Participants who are currently taking omaveloxolone must have initiated treatment at least 1 year prior to Screening and have been on a stable dose for at least 12 weeks prior to Screening, with no anticipated dose adjustments during the 48-week period following NBIB-223 infusion.

  • Participants who are not currently taking omaveloxolone must agree not to start taking omaveloxolone during the 48-week period following NBIB-223 infusion.

Key Exclusion Criteria:

  • Pregnant, plan to become pregnant during the first 12 months of the study, or lactating.
  • Have protocol-specified laboratory abnormalities at screening.
  • Previous exposure to adeno-associated virus (AAV)-9 as determined by the presence of serum total anti-AAV9 antibodies (TAb).
  • Contraindication to cerebrospinal fluid collection via lumbar puncture, magnetic resonance imaging with contrast, or endomyocardial biopsy.
  • Have had a myocardial infarction, cerebrovascular accident, thromboembolic event, or transient ischemic attacks, or have evidence of myocardial ischemia and/or clinically significant cardiac valve disease, obstructive cardiac disease, or arrhythmias.
  • Have an ongoing medical condition that, in the opinion of the investigator, would compromise the participant's safety or successful participation in the study or interpretation of study results.
  • Have uncontrolled diabetes (hemoglobin [Hb] A1c >8.0%)
  • Have an active fungal, bacterial, or viral infection.
  • Have scheduled or planning to schedule surgical treatment for scoliosis, foot deformity, or other indications during the first 18 months of the study that will interfere with the participant's ability to complete study assessments.
  • Any history of participation in a cellular or gene therapy clinical study, or any history of having been treated with a cellular or gene therapy.
  • Have participated in any other interventional clinical study within 30 days prior to Study Day 1 or receipt of any investigational product within the 30 days of enrollment or 5 half-lives of the investigational product, whichever is longer.

Additional inclusion/exclusion criteria may apply, per protocol.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Part A: NBIB-223

Dose escalation in adult participants.
Drug: NBIB-0000223

Intravenous (IV) infusion

Other Names:
  • NBIB-223
Experimental: Part B: NBIB-223

Dose expansion in pediatric participants.
Drug: NBIB-0000223

Intravenous (IV) infusion

Other Names:
  • NBIB-223
Experimental: Part C: NBIB-223

Dose expansion in adult and/or pediatric participants with FA-related cardiomyopathy.
Drug: NBIB-0000223

Intravenous (IV) infusion

Other Names:
  • NBIB-223

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of Participants with Treatment-emergent Adverse Events
Time Frame: Day 1 through 48-Week Post-Treatment Follow-Up
Day 1 through 48-Week Post-Treatment Follow-Up

Secondary Outcome Measures

Outcome Measure
Time Frame
Change from Baseline in Frataxin Protein Expression
Time Frame: Baseline, up to 48-Week Post-Treatment Follow-Up
Baseline, up to 48-Week Post-Treatment Follow-Up

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Neurocrine Biosciences

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

November 1, 2026

Primary Completion (Estimated)

November 1, 2028

Study Completion (Estimated)

November 1, 2032

Study Registration Dates

First Submitted

November 6, 2026

First Submitted That Met QC Criteria

November 6, 2026

First Posted (Actual)

November 12, 2026

Study Record Updates

Last Update Posted (Actual)

November 12, 2026

Last Update Submitted That Met QC Criteria

November 6, 2026

Last Verified

November 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NBIB-0000223-FA1789

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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