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

ClinicalTrials.gov ID: NCT07934303

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ClinicalTrials.gov public records Last synced Nov 13, 2026, 4:08 PM EST

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Official title

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

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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 identification

NCT ID: NCT07934303
Recruitment status: Not yet recruiting
Study type: Interventional
Phase: Phase 1
Lead sponsor: Neurocrine Biosciences; Industry
Enrollment: 24 participants

Conditions and interventions

Conditions

Friedreich's Ataxia

Interventions

NBIB-0000223 Drug

Drug

Eligibility (public fields only)

Age range: 8 Years to 40 Years
Sex: All
Healthy volunteers: Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: Nov 2026 (Estimated)
Primary completion: Nov 2028 (Estimated)
Completion: Nov 2032 (Estimated)
Last update posted: Nov 12, 2026

2026 – 2032

United States locations

U.S. sites: 5
U.S. states: 4
U.S. cities: 5

Facility	City	State	ZIP	Site status
Neurocrine Clinical Site	Los Angeles	California	90095	—
Neurocrine Clinical Site	Gainesville	Florida	32608	—
Neurocrine Clinical Site	Tampa	Florida	33612	—
Neurocrine Clinical Site	Iowa City	Iowa	52242	—
Neurocrine Clinical Site	Philadelphia	Pennsylvania	19406	—

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT07934303, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted Nov 12, 2026 · Synced Nov 13, 2026