Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
NCT ID: NCT04176991
Last Updated: 2020-11-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1
28 participants
INTERVENTIONAL
2019-12-11
2020-10-31
Brief Summary
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Detailed Description
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To evaluate the safety and tolerability of single ascending doses of CTI-1601 in subjects with Friedreich's ataxia.
Secondary Objectives:
1. To evaluate the pharmacokinetics (PK) of CTI-1601 following increasing single doses of subcutaneously (SC) administered CTI-1601.
2. To evaluate the pharmacodynamics (PD) of CTI-1601 following increasing single doses of SC administered CTI-1601.
CTI-1601 or Placebo - Dose/Mode of Administration: Single Dose/Subcutaneous
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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CTI-1601
CTI-1601
CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia
Placebo
Placebo
Placebo Comparator
Interventions
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CTI-1601
CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia
Placebo
Placebo Comparator
Eligibility Criteria
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Inclusion Criteria
2. Subject is male or female, 18 years of age or older at screening.
3. 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.
4. Subjects must weigh \> 40 kilograms (kg).
Exclusion Criteria
2. Subject requires use of amiodarone.
3. Subject used erythropoietin, etravirine, or gamma interferon within 3 months prior to screening.
4. Subject use of investigational drug (other than CTI-1601) or device within 90 days prior to screening.
5. 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.
6. 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.
7. Male subject who has an ECG QTcF \> 450 milliseconds or female subject who has an ECG QTcF \> 470 milliseconds.
8. Subject has a screening echocardiogram ejection fraction \<45 percent.
9. 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.
10. Subjects with known or suspected chronic use of cannabinoid products.
18 Years
ALL
No
Sponsors
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Veristat, Inc.
OTHER
Metrum Research Group, LLC
UNKNOWN
Larimar Therapeutics, Inc.
INDUSTRY
Responsible Party
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Locations
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Clinilabs Drug Development Corporation
Eatontown, New Jersey, United States
Countries
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References
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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.
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.
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.
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.
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.
Related Links
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Friedreich's Ataxia Research Alliance
Other Identifiers
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CLIN-1601-101
Identifier Type: -
Identifier Source: org_study_id