An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia

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NCT06447025 · readout ≤ 165 d

Sponsored by Larimar Therapeutics, Inc. (industry) · LRMR — their whole pipeline →.

Phase

Phase 2

Status

Recruiting

Study type

Interventional

Design

Na · None · Treatment

Enrollment

85estimated

Sites

Country

United States

Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →

Source: ClinicalTrials.gov, retrieved 2026-08-19

Dates

each axis at the precision it was filed, with the registry's own basis

Date

Filed

Basis

What it is

Start

2024-01-25

actual

When the study began enrolling

Primary completion

Jan 2027

estimated

The date the last participant is measured for the primary outcome — the readout window

Study completion

Jan 2027

estimated

The whole study's end, after follow-up

First posted

2024-06-06

actual

When this record first appeared on the registry

Results posted

—

When the sponsor posted results to the registry

Record updated

2026-05-13

actual

The sponsor's own last edit to this record — every projection above is as current as this date

What it studies

Condition

Friedreich Ataxia

Intervention

Drug: CTI-1601 — also filed as Nomlabofusp

Primary outcomes

what the primary-completion date above is the date OF

Number of subjects with treatment-emergent adverse events (TEAEs) by System Organ Class (SOC), Preferred Term (PT) and Maximum Severity

measured Up to 24 months

Change from baseline in electrocardiogram (ECG) parameters including, but not limited to, HR, RR interval, PR interval, QRS duration, QT interval, and QTcF interval

measured Up to 24 months

Change from baseline in left ventricular ejection fraction (LVEF)

measured Up to 24 months

Change from baseline in left ventricular end-diastolic volume (LVEDV)

measured Up to 24 months

Number of subjects with any suicidal ideation or behavior (Categories 1-10) of the Columbia Suicide Severity Rating Scale (C-SSRS)

measured Up to 24 months

Change from baseline at each collection timepoint in tissue frataxin concentrations normalized to total protein observed in buccal cells collected from cheek swabs and skin cells collected from skin punch biopsies

measured Up to 24 months

Change from baseline in motor function as assessed by 9-hole peg test (9-HPT)

measured Up to 24 months

Change from baseline in motor function as assessed by the timed 25-foot walk test (T25-FW)

measured Up to 24 months

Change from baseline in neurologic function as assessed by the modified Friedreich's Ataxia Rating Scale (mFARS) total score

measured Up to 24 months

Change from baseline in neurologic function as assessed by the upright stability subscale examination of the mFARS

measured Through study completion, up to 24 months

Change in activities of daily living (ADLs) as assessed by the Friedreich's Ataxia Rating Scale Activities of Daily Living (FARS_ADL)

measured Up to 24 months

Change from baseline in total fatigue score and all the subscale scores as assessed by the Fatigue Impact Scale (MFIS)

measured Up to 24 months

Change from baseline in the assessment of disease as assessed by the Functional Staging for Ataxia

measured Up to 24 months

Overall impression of change as assessed by the patient using the Patient Global Impression of Change (PGI-C) Scale

measured Up to 24 months

Overall impression of change assessed by a clinician using the Clinical Global Impression of Change (CGI-C)

measured Up to 24 months

Area under the concentration-time curve for the dosing interval (AUC0-tau)

measured Days 1, 30, 60, 90: pre-dose, 5, 15, 30 minutes after the dose, and 1, 2, 4, 6, 8 hours after the dose; Day 180: pre-dose and 5, 15 minutes after the dose; Days 270, 360, Q3M thereafter: pre-dose; through study completion, up to 24 months

Area under the concentration-time curve from time 0 to the time of last quantifiable concentration (AUC0-t)

Mean maximum observed concentration (Cmax)

Mean time of maximum observed concentration (Tmax)

Concentration reached immediately before the next dose is administered (Ctrough)

Publications

PMID 22752493 — cited as background
PMID 20675166 — cited as background
PMID 17056635 — cited as background
PMID 8148458 — cited as background
PMID 3178453 — cited as background
PMID 21315377 — cited as background
PMID 26339677 — cited as background
PMID 18852343 — cited as background
PMID 8797541 — cited as background

Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.

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