A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia

NCT06681766 | Terminated Phase 1 DMC FDA Drug

• 1 other identifier: CLIN-1601-103
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief 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).

Conditions

Friedreich Ataxia

Outcome Measures

Primary Outcomes (67)

• Number of AEs, TEAEs, related TEAEs, Grade 3/4 TEAEs, and SAEs

  Number

  Up to 72 days (including screening)

• Number of subjects with ISRs

  Number

  Up to 37 days

• Change from baseline in electrocardiogram (ECG) parameter - heart rate (HR)

  Number

  Baseline, Day 7

• Change from baseline in electrocardiogram (ECG) parameter - PR interval

  Number

  Baseline, Day 7

• Change from baseline in electrocardiogram (ECG) parameter - QRS complex

  Number

  Baseline, Day 7

• Change from baseline in electrocardiogram (ECG) parameter - QT interval

  Number

  Baseline, Day 7

• Change from baseline in electrocardiogram (ECG) parameter - QTcF (Corrected QT Interval)

  Number

  Baseline, Day 7

• Change from baseline in echocardiogram (ECHO) parameter - ejection fraction (EF)

  Number

  Baseline, Day 7

• Change from baseline in echocardiogram (ECHO) parameter - left ventricular end-diastolic volume (LV EDV)

  Number

  Baseline, Day 7

• Change from baseline in echocardiogram (ECHO) parameter - left ventricular end-systolic volume (LV ESV)

  Number

  Baseline, Day 7

• Change from baseline in echocardiogram (ECHO) parameter - relative wall thickness (RWT)

  Number

  Baseline, Day 7

• Change from baseline in echocardiogram (ECHO) parameter - left ventricular mass (LVM)

  Number

  Baseline, Day 7

• Change from baseline in echocardiogram (ECHO) parameter - left ventricular posterior wall thickness

  Number

  Baseline, Day 7

• Change from baseline in echocardiogram (ECHO) parameter - septal wall thickness

  Number

  Baseline, Day 7

• Change from baseline in echocardiogram (ECHO) parameter - mitral valve inflow Doppler

  Number

  Baseline, Day 7

• Change from baseline in echocardiogram (ECHO) parameter - tissue Doppler

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - sodium

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - potassium

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - glucose

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - blood urea nitrogen

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - creatinine

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - calcium, chloride

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - phosphorus

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - total protein

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - total CO2

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - albumin

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - AST

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - ALT

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - GGT

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - ALP

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - total bilirubin

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - uric acid

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - HbA1c

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - hemoglobin

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - hematocrit

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - RBC count

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - RDW

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - MCV

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - MCH

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - platelet count

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - WBC count

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - ANC

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - Eosinophils

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - Monocytes

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - Basophils

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - Lymphocytes

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - Bands

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - Neutrophils

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - Cholesterol

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - HDL cholesterol

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - LDL cholesterol

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - Triglycerides

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - very low-density lipoprotein cholesterol

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - pH

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - protein

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - blood

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - ketones

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - bilirubin

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - urobilinogen

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - nitrites

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - leukocyte esterase

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - color

  Number

  Baseline, Day 7

• Change from baseline in clinical laboratory assessment - specific gravity

  Number

  Baseline, Day 7

• Change from baseline in pulse rate

  Number

  Baseline, Day 7

• Number of subjects with abnormal physical examinations

  Number

  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

  Up to 72 days (including screening)

• Number of subjects who discontinue treatment and/or study

  Number

  Up to 37 days

Secondary Outcomes (5)

• AUC0-last

  Days 1, 7

• Cmax

  Days 1, 7

• Tmax

  Days 1, 7

• Ctrough

  Days 1, 7

• Change from baseline in FXN concentrations normalized to total protein observed in buccal cells collected from cheek swabs

  Baseline, Day 7

Study Arms (2)

Nomlabofusp

EXPERIMENTAL

Subcutaneous injection of 0.8 mg/kg, with a maximum dose of 50 mg, once daily for 7 days

Drug: Nomlabofusp

Placebo

PLACEBO COMPARATOR

Subcutaneous injection once daily for 7 days

Drug: Placebo

Interventions

Nomlabofusp DRUG

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.

Also known as: CTI-1601

Nomlabofusp

Placebo DRUG

The placebo is a sterile, preservative-free, clear liquid for subcutaneous injection.

Also known as: CTI-1601 Placebo, Nomlabofusp Placebo

Placebo

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Larimar Therapeutics, Inc.: lead

Study Sites (1)

Uncommon Cures

Chevy Chase, Maryland, 20815, United States

Location

Related Publications (10)

• 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.

  PMID: 22752493 BACKGROUND

• Deutsch 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.

  PMID: 20675166 BACKGROUND

• Fahey 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.

  PMID: 17056635 BACKGROUND

• Goodkin 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.

  PMID: 3178453 BACKGROUND

• Indelicato 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.

  PMID: 32746884 BACKGROUND

• Koeppen 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.

  PMID: 21315377 BACKGROUND

• Lawerman 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.

  PMID: 28815574 BACKGROUND

• Lazaropoulos 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.

  PMID: 26339677 BACKGROUND

• Pandolfo M. Friedreich ataxia. Arch Neurol. 2008 Oct;65(10):1296-303. doi: 10.1001/archneur.65.10.1296.

  PMID: 18852343 BACKGROUND

• Rudick 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.

  PMID: 8797541 BACKGROUND

MeSH Terms

Conditions

Friedreich Ataxia

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Larimar Therapeutics, Inc.

  Larimar Therapeutics, Inc.

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 6, 2024
• First Posted: November 8, 2024
• Study Start: December 6, 2024
• Primary Completion: April 28, 2025
• Study Completion: April 28, 2025
• Last Updated: January 28, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)