Friedreich AtaxiaPhase 1NCT06681766
A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia
A Phase 1 study testing Nomlabofusp in people with Friedreich Ataxia, run by Larimar Therapeutics, Inc. at 1 site in United States.
Trial summary
The goal of this clinical trial is to evaluate the safety and tolerability of nomlabofusp (CTI-1601) in adolescents and children with Friedreich's ataxia (FRDA). Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Friedreich Ataxia
- What is being tested?
- Nomlabofusp
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Dec 2024to 27 Apr 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Subject has genetically confirmed diagnosis of FRDA manifested by homozygous GAA repeat expansions, with repeat sizing (if available) included on the diagnosis report.
- Male or female subjects ≥ 2 to < 18 years of age at screening.
- Subjects must weigh ≥ 10.0 kg.
- Subject must be able to traverse a distance of 25 feet with or without some assistive device (e.g., cane, walker, crutches, self-propelled wheelchair) and meet the following requirements:
- Be able to sit upright with thighs together and arms crossed without requiring support on more than 2 sides;
- Be able to transfer from bed to chair independently or with assistance if, in the opinion of the investigator, the degree of physical disability does not result in undue risk to the subject while participating in the study; and
- Perform basic age-appropriate daily care, such as feeding themselves and personal hygiene, with minimal assistance.
Who cannot join
- Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only 1 allele) for FRDA.
- Subject has any condition, disease, or situation, including a cardiac condition or disease, that in the opinion of the investigator could confound the results of the study or put the subject at undue risk, making participation inadvisable.
- Subjects currently receiving or having received omaveloxolone within 30 days prior to Screening.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Nomlabofusp in Adolescents and Children With Friedreich's Ataxia
The goal of this clinical trial is to evaluate the safety and tolerability of nomlabofusp (CTI-1601) in adolescents and children with Friedreich's ataxia (FRDA).
Read the detailed description
This is a randomized, double-blind, placebo-controlled study evaluating a weight-based dose of nomlabofusp versus placebo in adolescents and children with FRDA. The study will consist of at least two cohorts with at least 12 to 15 participants in each cohort. Participants will be dosed once daily (QD) for 7 days.
- Conditions
- Friedreich Ataxia
- Being tested
- Nomlabofusp, Placebo
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NomlabofuspDrug
Also called CTI-1601
Nomlabofusp is a recombinant fusion protein provided in a sterile, preservative-free buffered solution for subcutaneous injection intended to deliver human frataxin, the protein deficient in Friedreich's ataxia.
PlaceboDrug
Also called CTI-1601 Placebo, Nomlabofusp Placebo
The placebo is a sterile, preservative-free, clear liquid for subcutaneous injection.
| Study group | Role | What they receive |
|---|---|---|
| Nomlabofusp | Experimental | Subcutaneous injection of 0.8 mg/kg, with a maximum dose of 50 mg, once daily for 7 days Receives: Drug: Nomlabofusp |
| Placebo | Placebo comparator | Subcutaneous injection once daily for 7 days Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of AEs, TEAEs, related TEAEs, Grade 3/4 TEAEs, and SAEs
Number
Measured: Up to 72 days (including screening)
Number of subjects with ISRs
Number
Measured: Up to 37 days
Change from baseline in electrocardiogram (ECG) parameter - heart rate (HR)
Number
Measured: Baseline, Day 7
Change from baseline in electrocardiogram (ECG) parameter - PR interval
Number
Measured: Baseline, Day 7
Change from baseline in electrocardiogram (ECG) parameter - QRS complex
Number
Measured: Baseline, Day 7
Change from baseline in electrocardiogram (ECG) parameter - QT interval
Number
Measured: Baseline, Day 7
Change from baseline in electrocardiogram (ECG) parameter - QTcF (Corrected QT Interval)
Number
Measured: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - ejection fraction (EF)
Number
Measured: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - left ventricular end-diastolic volume (LV EDV)
Number
Measured: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - left ventricular end-systolic volume (LV ESV)
Number
Measured: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - relative wall thickness (RWT)
Number
Measured: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - left ventricular mass (LVM)
Number
Measured: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - left ventricular posterior wall thickness
Number
Measured: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - septal wall thickness
Number
Measured: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - mitral valve inflow Doppler
Number
Measured: Baseline, Day 7
Change from baseline in echocardiogram (ECHO) parameter - tissue Doppler
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - sodium
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - potassium
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - glucose
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - blood urea nitrogen
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - creatinine
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - calcium, chloride
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - phosphorus
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - total protein
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - total CO2
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - albumin
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - AST
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - ALT
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - GGT
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - ALP
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - total bilirubin
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - uric acid
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - HbA1c
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - hemoglobin
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - hematocrit
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - RBC count
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - RDW
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - MCV
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - MCH
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - platelet count
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - WBC count
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - ANC
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - Eosinophils
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - Monocytes
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - Basophils
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - Lymphocytes
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - Bands
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - Neutrophils
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - Cholesterol
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - HDL cholesterol
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - LDL cholesterol
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - Triglycerides
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - very low-density lipoprotein cholesterol
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - pH
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - protein
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - blood
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - ketones
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - bilirubin
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - urobilinogen
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - nitrites
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - leukocyte esterase
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - color
Number
Measured: Baseline, Day 7
Change from baseline in clinical laboratory assessment - specific gravity
Number
Measured: Baseline, Day 7
Change from baseline in pulse rate
Number
Measured: Baseline, Day 7
Number of subjects with abnormal physical examinations
Number
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)
Number
Measured: Up to 72 days (including screening)
Number of subjects who discontinue treatment and/or study
Number
Measured: Up to 37 days
Secondary outcomes (5)
AUC0-last
Area under the concentration-time-curve to the last quantifiable timepoint
Measured: Days 1, 7
Cmax
Maximum observed concentration
Measured: Days 1, 7
Tmax
Time of maximum observed concentration
Measured: Days 1, 7
Ctrough
Predose concentration
Measured: Days 1, 7
Change from baseline in FXN concentrations normalized to total protein observed in buccal cells collected from cheek swabs
Number
Measured: Baseline, Day 7
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Nov 2024
- Study start6 Dec 2024
- Primary completion27 Apr 2025
- Study completion27 Apr 2025
Why it stopped: 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.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (10)Papers the sponsor has linked to this study.
- Other trials for Friedreich AtaxiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Larimar Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Larimar Therapeutics, Inc., Study chair, Larimar Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06681766
- Sponsor's protocol ID
- CLIN-1601-103
- Status last verified
- Jul 2026
- First submitted
- 6 Nov 2024
- Last updated
- 10 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Delatycki MB, Corben LA. Clinical features of Friedreich ataxia. J Child Neurol. 2012 Sep;27(9):1133-7. doi: 10.1177/0883073812448230. Epub 2012 Jun 29.
PubMed 22752493Deutsch EC, Santani AB, Perlman SL, Farmer JM, Stolle CA, Marusich MF, Lynch DR. A rapid, noninvasive immunoassay for frataxin: utility in assessment of Friedreich ataxia. Mol Genet Metab. 2010 Oct-Nov;101(2-3):238-45. doi: 10.1016/j.ymgme.2010.07.001. Epub 2010 Jul 8.
PubMed 20675166Fahey MC, Corben L, Collins V, Churchyard AJ, Delatycki MB. How is disease progress in Friedreich's ataxia best measured? A study of four rating scales. J Neurol Neurosurg Psychiatry. 2007 Apr;78(4):411-3. doi: 10.1136/jnnp.2006.096008. Epub 2006 Oct 20.
PubMed 17056635Goodkin DE, Hertsgaard D, Seminary J. Upper extremity function in multiple sclerosis: improving assessment sensitivity with box-and-block and nine-hole peg tests. Arch Phys Med Rehabil. 1988 Oct;69(10):850-4.
PubMed 3178453Indelicato E, Nachbauer W, Eigentler A, Amprosi M, Matteucci Gothe R, Giunti P, Mariotti C, Arpa J, Durr A, Klopstock T, Schols L, Giordano I, Burk K, Pandolfo M, Didszdun C, Schulz JB, Boesch S; EFACTS (European Friedreich's Ataxia Consortium for Translational Studies). Onset features and time to diagnosis in Friedreich's Ataxia. Orphanet J Rare Dis. 2020 Aug 3;15(1):198. doi: 10.1186/s13023-020-01475-9.
PubMed 32746884Koeppen AH. Friedreich's ataxia: pathology, pathogenesis, and molecular genetics. J Neurol Sci. 2011 Apr 15;303(1-2):1-12. doi: 10.1016/j.jns.2011.01.010.
PubMed 21315377Lawerman TF, Brandsma R, Burger H, Burgerhof JGM, Sival DA; the Childhood Ataxia and Cerebellar Group of the European Pediatric Neurology Society. Age-related reference values for the pediatric Scale for Assessment and Rating of Ataxia: a multicentre study. Dev Med Child Neurol. 2017 Oct;59(10):1077-1082. doi: 10.1111/dmcn.13507. Epub 2017 Aug 17.
PubMed 28815574Lazaropoulos M, Dong Y, Clark E, Greeley NR, Seyer LA, Brigatti KW, Christie C, Perlman SL, Wilmot GR, Gomez CM, Mathews KD, Yoon G, Zesiewicz T, Hoyle C, Subramony SH, Brocht AF, Farmer JM, Wilson RB, Deutsch EC, Lynch DR. Frataxin levels in peripheral tissue in Friedreich ataxia. Ann Clin Transl Neurol. 2015 Aug;2(8):831-42. doi: 10.1002/acn3.225. Epub 2015 Jul 1.
PubMed 26339677Pandolfo M. Friedreich ataxia. Arch Neurol. 2008 Oct;65(10):1296-303. doi: 10.1001/archneur.65.10.1296.
PubMed 18852343Rudick R, Antel J, Confavreux C, Cutter G, Ellison G, Fischer J, Lublin F, Miller A, Petkau J, Rao S, Reingold S, Syndulko K, Thompson A, Wallenberg J, Weinshenker B, Willoughby E. Clinical outcomes assessment in multiple sclerosis. Ann Neurol. 1996 Sep;40(3):469-79. doi: 10.1002/ana.410400321.
PubMed 8797541
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Nomlabofusp in Adolescents and Children With Friedreich's Ataxia
- ClinicalTrials.gov ID
- NCT06681766
- Acronym / sponsor ID
- CLIN-1601-103
- Conditions
- Friedreich Ataxia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: 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.
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Larimar Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 6 Dec 2024 (actual)
- Primary completion
- 27 Apr 2025 (actual)
- Study completion
- 27 Apr 2025 (actual)
- First posted
- 8 Nov 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical trial is to evaluate the safety and tolerability of nomlabofusp (CTI-1601) in adolescents and children with Friedreich's ataxia (FRDA).
Detailed description
This is a randomized, double-blind, placebo-controlled study evaluating a weight-based dose of nomlabofusp versus placebo in adolescents and children with FRDA. The study will consist of at least two cohorts with at least 12 to 15 participants in each cohort. Participants will be dosed once daily (QD) for 7 days.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Nomlabofusp | Experimental | Drug: NomlabofuspSubcutaneous injection of 0.8 mg/kg, with a maximum dose of 50 mg, once daily for 7 days |
| Placebo | Placebo comparator | Drug: PlaceboSubcutaneous injection once daily for 7 days |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Subject has genetically confirmed diagnosis of FRDA manifested by homozygous GAA repeat expansions, with repeat sizing (if available) included on the diagnosis report.
- Male or female subjects ≥ 2 to < 18 years of age at screening.
- Subjects must weigh ≥ 10.0 kg.
- Subject must be able to traverse a distance of 25 feet with or without some assistive device (e.g., cane, walker, crutches, self-propelled wheelchair) and meet the following requirements:
Key exclusion criteria (3 listed)
- Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only 1 allele) for FRDA.
- Subject has any condition, disease, or situation, including a cardiac condition or disease, that in the opinion of the investigator could confound the results of the study or put the subject at undue risk, making participation inadvisable.
- Subjects currently receiving or having received omaveloxolone within 30 days prior to Screening.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NomlabofuspAlso: CTI-1601Nomlabofusp is a recombinant fusion protein provided in a sterile, preservative-free buffered solution for subcutaneous injection intended to deliver human frataxin, the protein deficient in Friedreich's ataxia. | Drug | Nomlabofusp |
| PlaceboAlso: CTI-1601 Placebo, Nomlabofusp PlaceboThe placebo is a sterile, preservative-free, clear liquid for subcutaneous injection. | Drug | Placebo |
Primary outcome
Number of AEs, TEAEs, related TEAEs, Grade 3/4 TEAEs, and SAEs
Time frame: Up to 72 days (including screening)
Number
Number of subjects with ISRs
Time frame: Up to 37 days
Number
Change from baseline in electrocardiogram (ECG) parameter - heart rate (HR)
Time frame: Baseline, Day 7
Number
Change from baseline in electrocardiogram (ECG) parameter - PR interval
Time frame: Baseline, Day 7
Number
Change from baseline in electrocardiogram (ECG) parameter - QRS complex
Time frame: Baseline, Day 7
Number
Change from baseline in electrocardiogram (ECG) parameter - QT interval
Time frame: Baseline, Day 7
Number
Change from baseline in electrocardiogram (ECG) parameter - QTcF (Corrected QT Interval)
Time frame: Baseline, Day 7
Number
Change from baseline in echocardiogram (ECHO) parameter - ejection fraction (EF)
Time frame: Baseline, Day 7
Number
Change from baseline in echocardiogram (ECHO) parameter - left ventricular end-diastolic volume (LV EDV)
Time frame: Baseline, Day 7
Number
Change from baseline in echocardiogram (ECHO) parameter - left ventricular end-systolic volume (LV ESV)
Time frame: Baseline, Day 7
Number
Change from baseline in echocardiogram (ECHO) parameter - relative wall thickness (RWT)
Time frame: Baseline, Day 7
Number
Change from baseline in echocardiogram (ECHO) parameter - left ventricular mass (LVM)
Time frame: Baseline, Day 7
Number
Change from baseline in echocardiogram (ECHO) parameter - left ventricular posterior wall thickness
Time frame: Baseline, Day 7
Number
Change from baseline in echocardiogram (ECHO) parameter - septal wall thickness
Time frame: Baseline, Day 7
Number
Change from baseline in echocardiogram (ECHO) parameter - mitral valve inflow Doppler
Time frame: Baseline, Day 7
Number
Change from baseline in echocardiogram (ECHO) parameter - tissue Doppler
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - sodium
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - potassium
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - glucose
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - blood urea nitrogen
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - creatinine
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - calcium, chloride
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - phosphorus
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - total protein
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - total CO2
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - albumin
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - AST
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - ALT
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - GGT
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - ALP
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - total bilirubin
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - uric acid
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - HbA1c
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - hemoglobin
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - hematocrit
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - RBC count
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - RDW
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - MCV
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - MCH
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - platelet count
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - WBC count
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - ANC
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - Eosinophils
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - Monocytes
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - Basophils
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - Lymphocytes
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - Bands
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - Neutrophils
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - Cholesterol
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - HDL cholesterol
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - LDL cholesterol
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - Triglycerides
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - very low-density lipoprotein cholesterol
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - pH
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - protein
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - blood
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - ketones
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - bilirubin
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - urobilinogen
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - nitrites
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - leukocyte esterase
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - color
Time frame: Baseline, Day 7
Number
Change from baseline in clinical laboratory assessment - specific gravity
Time frame: Baseline, Day 7
Number
Change from baseline in pulse rate
Time frame: Baseline, Day 7
Number
Number of subjects with abnormal physical examinations
Time frame: Up to 72 days (including screening)
Number
Number of subjects with suicidal ideation, suicidal behavior, and suicidal ideation or behavior based on the Columbia Suicide Severity Rating Scale (C-SSRS)
Time frame: Up to 72 days (including screening)
Number
Number of subjects who discontinue treatment and/or study
Time frame: Up to 37 days
Number
5 secondary outcomes
AUC0-last
Time frame: Days 1, 7
Cmax
Time frame: Days 1, 7
Tmax
Time frame: Days 1, 7
Ctrough
Time frame: Days 1, 7
Change from baseline in FXN concentrations normalized to total protein observed in buccal cells collected from cheek swabs
Time frame: Baseline, Day 7
Results and safety
The study was stopped early (reason given: 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.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2026.
- Background: PubMed 22752493Delatycki MB, Corben LA. Clinical features of Friedreich ataxia. J Child Neurol. 2012 Sep;27(9):1133-7. doi: 10.1177/0883073812448230. Epub 2012 Jun 29.
- Background: PubMed 20675166Deutsch EC, Santani AB, Perlman SL, Farmer JM, Stolle CA, Marusich MF, Lynch DR. A rapid, noninvasive immunoassay for frataxin: utility in assessment of Friedreich ataxia. Mol Genet Metab. 2010 Oct-Nov;101(2-3):238-45. doi: 10.1016/j.ymgme.2010.07.001. Epub 2010 Jul 8.
- Background: PubMed 17056635Fahey MC, Corben L, Collins V, Churchyard AJ, Delatycki MB. How is disease progress in Friedreich's ataxia best measured? A study of four rating scales. J Neurol Neurosurg Psychiatry. 2007 Apr;78(4):411-3. doi: 10.1136/jnnp.2006.096008. Epub 2006 Oct 20.
- Background: PubMed 3178453Goodkin DE, Hertsgaard D, Seminary J. Upper extremity function in multiple sclerosis: improving assessment sensitivity with box-and-block and nine-hole peg tests. Arch Phys Med Rehabil. 1988 Oct;69(10):850-4.
- Background: PubMed 32746884Indelicato E, Nachbauer W, Eigentler A, Amprosi M, Matteucci Gothe R, Giunti P, Mariotti C, Arpa J, Durr A, Klopstock T, Schols L, Giordano I, Burk K, Pandolfo M, Didszdun C, Schulz JB, Boesch S; EFACTS (European Friedreich's Ataxia Consortium for Translational Studies). Onset features and time to diagnosis in Friedreich's Ataxia. Orphanet J Rare Dis. 2020 Aug 3;15(1):198. doi: 10.1186/s13023-020-01475-9.
- Background: PubMed 21315377Koeppen AH. Friedreich's ataxia: pathology, pathogenesis, and molecular genetics. J Neurol Sci. 2011 Apr 15;303(1-2):1-12. doi: 10.1016/j.jns.2011.01.010.
- Background: PubMed 28815574Lawerman TF, Brandsma R, Burger H, Burgerhof JGM, Sival DA; the Childhood Ataxia and Cerebellar Group of the European Pediatric Neurology Society. Age-related reference values for the pediatric Scale for Assessment and Rating of Ataxia: a multicentre study. Dev Med Child Neurol. 2017 Oct;59(10):1077-1082. doi: 10.1111/dmcn.13507. Epub 2017 Aug 17.
- Background: PubMed 26339677Lazaropoulos M, Dong Y, Clark E, Greeley NR, Seyer LA, Brigatti KW, Christie C, Perlman SL, Wilmot GR, Gomez CM, Mathews KD, Yoon G, Zesiewicz T, Hoyle C, Subramony SH, Brocht AF, Farmer JM, Wilson RB, Deutsch EC, Lynch DR. Frataxin levels in peripheral tissue in Friedreich ataxia. Ann Clin Transl Neurol. 2015 Aug;2(8):831-42. doi: 10.1002/acn3.225. Epub 2015 Jul 1.
- Background: PubMed 18852343Pandolfo M. Friedreich ataxia. Arch Neurol. 2008 Oct;65(10):1296-303. doi: 10.1001/archneur.65.10.1296.
- Background: PubMed 8797541Rudick R, Antel J, Confavreux C, Cutter G, Ellison G, Fischer J, Lublin F, Miller A, Petkau J, Rao S, Reingold S, Syndulko K, Thompson A, Wallenberg J, Weinshenker B, Willoughby E. Clinical outcomes assessment in multiple sclerosis. Ann Neurol. 1996 Sep;40(3):469-79. doi: 10.1002/ana.410400321.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Larimar Therapeutics, Inc. study chair
Larimar Therapeutics, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Nomlabofusp in people with Friedreich Ataxia, run by Larimar Therapeutics, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 10 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: 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.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of AEs, TEAEs, related TEAEs, Grade 3/4 TEAEs, and SAEs (time frame: Up to 72 days (including screening)). The study lists 67 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06681766, which also lists 10 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jul 2026.