A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia
← catalyst calendarSponsored by Larimar Therapeutics, Inc. (industry) · LRMR — their whole pipeline →.
Phase
Phase 1
Status
Terminated
Study type
Interventional
Design
Randomized · Quadruple · Treatment
Enrollment
18actual
Sites
1
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
Why the sponsor stopped it
Review of Cohort 1 data facilitated direct enrollment into open label study and eliminate pause in study drug administration between PK study and open label study.
As filed on the registry record, in the sponsor's own words.
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2024-12-06 | actual | When the study began enrolling |
| Primary completion | 2025-04-27 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2025-04-27 | actual | The whole study's end, after follow-up |
| First posted | 2024-11-08 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-07-10 | 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
Interventions
- Drug: Nomlabofusp — also filed as CTI-1601
- Drug: Placebo — also filed as CTI-1601 Placebo, Nomlabofusp Placebo
Primary outcomes
what the primary-completion date above is the date OFNumber of AEs, TEAEs, related TEAEs, Grade 3/4 TEAEs, and SAEs
measured Up to 72 days (including screening)
Number of subjects with ISRs
measured Up to 37 days
Change from baseline in electrocardiogram (ECG) parameter - heart rate (HR)
measured Baseline, Day 7
Change from baseline in electrocardiogram (ECG) parameter - PR interval
measured Baseline, Day 7
Change from baseline in electrocardiogram (ECG) parameter - QRS complex
measured Baseline, Day 7
Change from baseline in electrocardiogram (ECG) parameter - QT interval
measured Baseline, Day 7
Change from baseline in electrocardiogram (ECG) parameter - QTcF (Corrected QT Interval)
measured Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - ejection fraction (EF)
measured Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - left ventricular end-diastolic volume (LV EDV)
measured Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - left ventricular end-systolic volume (LV ESV)
measured Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - relative wall thickness (RWT)
measured Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - left ventricular mass (LVM)
measured Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - left ventricular posterior wall thickness
measured Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - septal wall thickness
measured Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - mitral valve inflow Doppler
measured Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - tissue Doppler
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - sodium
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - potassium
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - glucose
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - blood urea nitrogen
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - creatinine
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - calcium, chloride
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - phosphorus
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - total protein
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - total CO2
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - albumin
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - AST
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - ALT
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - GGT
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - ALP
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - total bilirubin
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - uric acid
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - HbA1c
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - hemoglobin
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - hematocrit
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - RBC count
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - RDW
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - MCV
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - MCH
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - platelet count
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - WBC count
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - ANC
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - Eosinophils
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - Monocytes
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - Basophils
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - Lymphocytes
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - Bands
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - Neutrophils
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - Cholesterol
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - HDL cholesterol
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - LDL cholesterol
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - Triglycerides
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - very low-density lipoprotein cholesterol
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - pH
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - protein
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - blood
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - ketones
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - bilirubin
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - urobilinogen
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - nitrites
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - leukocyte esterase
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - color
measured Baseline, Day 7
Change from baseline in clinical laboratory assessment - specific gravity
measured Baseline, Day 7
Change from baseline in pulse rate
measured Baseline, Day 7
Number of subjects with abnormal physical examinations
measured Up to 72 days (including screening)
Number of subjects with suicidal ideation, suicidal behavior, and suicidal ideation or behavior based on the Columbia Suicide Severity Rating Scale (C-SSRS)
measured Up to 72 days (including screening)
Number of subjects who discontinue treatment and/or study
measured Up to 37 days
Publications
- PMID 22752493 — cited as background
- PMID 20675166 — cited as background
- PMID 17056635 — cited as background
- PMID 3178453 — cited as background
- PMID 32746884 — cited as background
- PMID 21315377 — cited as background
- PMID 28815574 — 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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