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NBIB-0000223

Phase 1

Friedreich's Ataxia | Small Molecules | Rare Disease |Neurocrine Biosciences, Inc.|Trials Updated: Nov 12, 2026

NBIB-0000223 development status

Highest phase Phase 1
Registered trials 1 across 1 sponsor since Nov 2026

NBIB-0000223 target and mechanism

ModalitySmall Molecules

Also known as NBIB-223

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment24

FDA Designations

No designations recorded

NBIB-0000223 clinical trials

NBIB-0000223 · 1 trial · 1 indication

Phase 1 1
NCT07934303Study of NBIB-223 Gene Therapy in Friedreich's Ataxia (FACET)Friedreich's Ataxia
NOT YET_RECRUITING24 Analytics
PHASE1NOT YET_RECRUITING
Study of NBIB-223 Gene Therapy in Friedreich's Ataxia (FACET)
Friedreich's Ataxia
NCT0793430324
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Study Endpoints

Primary Endpoints

Number of Participants with Treatment-emergent Adverse Events
Day 1 through 48-Week Post-Treatment Follow-Up

Secondary Endpoints

Change from Baseline in Frataxin Protein Expression
Baseline, up to 48-Week Post-Treatment Follow-Up
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part A: NBIB-223EXPERIMENTALDose escalation in adult participants.
Part B: NBIB-223EXPERIMENTALDose expansion in pediatric participants.
Part C: NBIB-223EXPERIMENTALDose expansion in adult and/or pediatric participants with FA-related cardiomyopathy.

Interventions

NameTypeDescription
NBIB-0000223DRUGIntravenous (IV) infusion
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Eligibility Criteria

Age Range8 Years to 40 Years
SexALL
Healthy VolunteersNo
Study Sites5

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 repe...

Countries:United States
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Recent Changes (Last 90 Days)

LOWNov 12, 2026NCT07934303NEW_TRIAL: changed
LOWNov 12, 2026NCT07934303NEW_TRIAL: changed

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