Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia

NCT04176991 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: CLIN-1601-101
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 1

Brief Summary

To evaluate the safety and tolerability of single ascending doses of CTI-1601 in participants with Friedreich's ataxia

Conditions

Friedreich Ataxia

Outcome Measures

Primary Outcomes (2)

• Number of Participants with Treatment-Emergent Adverse Events

  Overall summary of the Participants with Treatment Emergent Adverse Events

  Through study completion, an average of 70 days

• Number of Treatment Emergent Adverse Events by System Organ Classification and Preferred Term

  Overall summary of Participants with Treatment Emergent Adverse Events by System Organ Classification (MedDRA version 22.0)

  Through study completion, an average of 70 days

Secondary Outcomes (11)

• Pharmacokinetics - Area under the concentration-time curve after a single dose

  Up to 48 hours

• Pharmacokinetics - Maximum observed plasma concentration after a single dose

  Up to 48 hours

• Pharmacokinetics - Time to reach maximum plasma concentration after a single dose

  Up to 48 hours

• Pharmacokinetics - Area under the concentration-time curve from time 0 to infinity

  48 hours

• Pharmacokinetics - Area under the concentration-time curve from time 0 to the last measurable time point

  48 hours

Study Arms (2)

CTI-1601

EXPERIMENTAL

Biological: CTI-1601

Placebo

PLACEBO COMPARATOR

Biological: 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-1601

Placebo BIOLOGICAL

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 genetically confirmed Friedreich's ataxia diagnosis, homozygous GAA repeat expansions, with repeat sizing (if available) included on diagnostic report.
• Subject is male or female, 18 years of age or older at screening.
• Subject must have a mFARS\_neuro score ≥ 20 and be able to traverse a distance of 25 feet with or without some assistive device (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 minimal 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 (c) perform basic daily care, such as feeding themselves and personal hygiene, with minimal assistance.
• Subjects must weigh \> 40 kilograms (kg).

You may not qualify if:

• Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only one allele) for Friedreich's ataxia.
• Subject requires use of amiodarone.
• Subject used erythropoietin, etravirine, or gamma interferon within 3 months prior to screening.
• Subject use of investigational drug (other than CTI-1601) or device within 90 days prior to screening.
• Subject use of daily biotin supplementation that exceeds 30 mcg/day, either as part of a multivitamin or as a standalone supplement, within 7 days prior to study drug administration and/or throughout the entire study.
• Subject has clinically significant arrhythmia on electrocardiogram (ECG), or evidence of predisposition to significant ventricular arrhythmia on ECG, or evidence of active and unstable coronary artery disease.
• Male subject who has an ECG QTcF \> 450 milliseconds or female subject who has an ECG QTcF \> 470 milliseconds.
• Subject has a screening echocardiogram ejection fraction \<45 percent.
• Subject has a history of aspiration, aspiration pneumonia, or recurrent episodes of pneumonia (greater than or equal to 2 episodes of pneumonia) within the last 12 months.
• Subjects with known or suspected chronic use of cannabinoid products.

Sponsors & Collaborators

• Larimar Therapeutics, Inc.: lead
• Veristat, Inc.: collaborator
• Metrum Research Group, LLC: collaborator

Study Sites (1)

Clinilabs Drug Development Corporation

Eatontown, New Jersey, 07724, United States

Location

Related Publications (5)

• 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

• 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

• 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

• Plasterer HL, Deutsch EC, Belmonte M, Egan E, Lynch DR, Rusche JR. Development of frataxin gene expression measures for the evaluation of experimental treatments in Friedreich's ataxia. PLoS One. 2013 May 17;8(5):e63958. doi: 10.1371/journal.pone.0063958. Print 2013.

  PMID: 23691127 BACKGROUND

Related Links

• Friedreich's Ataxia Research Alliance

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 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 14, 2019
• First Posted: November 26, 2019
• Study Start: December 11, 2019
• Primary Completion: October 31, 2020
• Study Completion: October 31, 2020
• Last Updated: November 12, 2020

Record last verified: 2020-11

Locations

US (1)