A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia

NCT05579691 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: CLIN-1601-200
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 1

Brief Summary

To evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) administration of CTI-1601 over 28 days in subjects with Friedreich's ataxia (FRDA).

Conditions

Friedreich Ataxia

Outcome Measures

Primary Outcomes (1)

• Number of Participants with Treatment Emergent Adverse Events

  Overall summary of Participants with Treatment Emergent Adverse Events

  Through study completion, an average of 93 days

Secondary Outcomes (6)

• Maximum observed plasma concentration (Cmax) of CTI-1601 after multiple doses

  At baseline and up to 29 days

• Area under the concentration time curve (AUC) of CTI-1601 from time 0 through the last measurable time point

  At baseline and up to 29 days

• Time to maximum observed plasma concentration (tmax) of CTI-1601 after multiple doses

  At baseline and up to 29 days

• Time to last observed plasma concentration (tlast) of CTI-1601 after multiple doses

  At baseline and up to 29 days

• Changes from baseline in frataxin levels in buccal cells

  At baseline and up to 58 days

Study Arms (2)

CTI-160l

EXPERIMENTAL

CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia

Biological: CTI-1601

Placebo

PLACEBO COMPARATOR

Placebo Comparator

Other: Placebo

Interventions

CTI-1601 BIOLOGICAL

CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia

CTI-160l

Placebo OTHER

Placebo Comparator

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subject has a genetically confirmed diagnosis of FRDA manifested by homozygous GAA repeat expansions, with repeat sizing (if available) included on the diagnosis report.
• Subject is biologically male or female, 18 years of age or older at screening.
• Subject must have a mFARS score ≥ 20 and be able to traverse a distance of 25 feet with or without some assistive device (e.g., cane, walker, crutches, self-propelled wheelchair), and (a) be able to sit upright with thighs together and arms crossed without requiring support on more than two sides; (b) be able to transfer from bed to chair independently or with assistance if, in the opinion of the PI, the degree of physical disability does not result in undue risk to the subject while participating in the study; and (c) perform basic daily care, such as feeding themselves and personal hygiene, with minimal assistance.
• Subject must weigh \> 40.0 kg.

You may not qualify if:

• Subject who is confirmed as compound heterozygous (GAA repeat expansion on only one allele) for FRDA.
• Subject used an investigational drug or device within 90 days prior to screening.
• Subject requires use of amiodarone.
• Subject used erythropoietin, etravirine, or gamma interferon 90 days prior to Screening.
• Subject use of biotin supplementation that exceeds 30.0 mcg/day, either as part of a multivitamin or as a standalone supplement, within 7 days prior to the first dose of study drug.
• Subject uses more than 3.0 grams of acetaminophen daily.
• Subject receives medication that requires SC injection in the abdomen or thigh.
• Subject received a vaccination within 14 days of administration of the first dose of study drug or is scheduled to receive a vaccination within 14 days after administration of the last dose of study drug. As an exception, influenza and tetanus vaccines must be administered more than 72 hours prior to the first dose of study drug or 72 hours after the administration of the last dose of study drug.
• Subject has a screening ECHO LVEF \< 45%.
• Male subject has a QTcF \> 450 milliseconds or female subject has a QTcF \> 470 milliseconds on an ECG.
• Subject currently receiving or having received omaveloxolone within 30 days prior to Screening.

Sponsors & Collaborators

• Larimar Therapeutics, Inc.: lead

Study Sites (1)

Clinilabs Drug Development Corporation

Eatontown, New Jersey, 07724, United States

Location

Related Publications (9)

• 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

• 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

• 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

• 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

• 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

• 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

• 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

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

• Magdy Shenouda, M.D.

  Clinilabs, Inc.

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• 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: October 5, 2022
• First Posted: October 14, 2022
• Study Start: September 21, 2022
• Primary Completion: December 4, 2023
• Study Completion: December 4, 2023
• Last Updated: November 29, 2024

Record last verified: 2024-11

Locations

US (1)