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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04519567
Recruitment Status : Recruiting
First Posted : August 19, 2020
Last Update Posted : August 19, 2020
Sponsor:
Collaborators:
Veristat, Inc.
Metrum Research Group, LLC
Information provided by (Responsible Party):
Larimar Therapeutics, Inc.

Brief Summary:
To evaluate the safety and tolerability of multiple ascending doses of CTI-1601 in participants with Friedreich's ataxia

Condition or disease Intervention/treatment Phase
Friedreich Ataxia Biological: CTI-1601 Biological: Placebo Phase 1

Detailed Description:

Multiple Ascending Dose (MAD), Double-Blind, Placebo Controlled Study.

To evaluate the safety and tolerability of multiple ascending doses of CTI-1601 in subjects with Friedreich's ataxia.

Secondary Objectives:

  1. To evaluate the pharmacokinetics (PK) of CTI-1601 following, multiple, increasing, doses of subcutaneously (SC) administered CTI-1601.
  2. To evaluate the pharmacodynamics (PD) of CTI-1601 following, multiple, increasing, doses of SC administered CTI-1601.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1 Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneous CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
Actual Study Start Date : July 31, 2020
Estimated Primary Completion Date : June 30, 2021
Estimated Study Completion Date : June 30, 2021


Arm Intervention/treatment
Experimental: CTI-1601 Biological: CTI-1601
CTI-1601 is a recombinant fusion protein and is intended to deliver human frataxin, the protein deficient in Friedreich's ataxia

Placebo Comparator: Placebo Biological: Placebo
Placebo Comparator




Primary Outcome Measures :
  1. Number of Participants with Treatment Emergent Adverse Events [ Time Frame: Through study completion, an average of 75 days ]
    Overall summary of Participants with Treatment Emergent Adverse Events

  2. Number of Participants with Treatment Emergent Adverse Events by System Organ Classification and Preferred Term [ Time Frame: Through study completion, an average of 75 days ]
    Overall summary of Participants with Treatment Emergent Adverse Events by System Organ Classification (MedDRA version 23.0)


Secondary Outcome Measures :
  1. Pharmacokinetics - Maximum observed plasma concentration after multiple doses [ Time Frame: At baseline and up to 15 days ]
    Summary assessment of changes in the maximum observed plasma concentration after multiple doses

  2. Pharmacokinetics - Minimum or "trough" plasma concentration after multiple doses [ Time Frame: At baseline and up to 15 days ]
    Summary assessment of minimum or "trough" observed plasma concentration after multiple doses just prior to the administration of a subsequent dose

  3. Pharmacokinetics - Area under the concentration time curve (AUC) from time 0 through the last measurable time point [ Time Frame: At baseline and up to 15 days ]
    Summary assessment of changes in the AUC from time 0 to the last measurable time point and during the dosing interval

  4. Pharmacokinetics - Terminal half-life estimation [ Time Frame: At baseline and up to 15 days ]
    Summary assessment of changes in the terminal half-life estimation

  5. Changes from Baseline in Frataxin Levels in Buccal Cell [ Time Frame: At baseline and up to 43 days ]
    Summary assessment of changes in frataxin levels in buccal cells

  6. Changes from Baseline in Levels of Protein Markers in Buccal Cell [ Time Frame: At baseline and up to 43 days ]
    Summary assessment of changes in levels of protein markers in buccal cells

  7. Changes from Baseline in Gene Expression in Buccal Cells [ Time Frame: At baseline and up to 43 days ]
    Summary assessment of changes in gene expression in buccal cells

  8. Changes from Baseline in Frataxin Levels in Platelets [ Time Frame: At baseline and up to 13 days ]
    Summary assessment of changes in frataxin levels in platelets

  9. Changes from Baseline in Gene Expression in Whole Blood [ Time Frame: At baseline and up to 16 days ]
    Summary assessment of changes in gene expression in whole blood

  10. Changes from Baseline in Frataxin Levels in Skin Punch Cells [ Time Frame: At baseline and up to 13 days ]
    Summary assessment of changes in frataxin levels in skin punch cells

  11. Changes from Baseline in Levels of Defined Protein Markers in Blood [ Time Frame: At baseline and up to 16 days ]
    Summary assessment of changes in levels of defined protein markers in blood

  12. Changes from Baseline in Levels of Specialized Lipids in Blood [ Time Frame: At baseline and up to 16 days ]
    Summary assessment of changes in levels of specialized lipids in blood



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Subject has genetically confirmed Friedreich's ataxia diagnosis manifested by homozygous GAA repeat expansions, with repeat sizing (if available) included on diagnostic report.
  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 assistance if, in the opinion of the principal 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:

  1. Subjects who had a serious adverse event (SAE), an adverse event (AE) that is Grade 3 or higher according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0 (or higher), or an AE considered clinically significant during participation in CLIN-1601-101 (NCT04176991).
  2. Subjects who are confirmed as compound heterozygous (GAA repeat expansion on only one allele) for Friedreich's ataxia.
  3. Subject use of investigational drug (other than CTI-1601) or device within 90 days prior to screening.
  4. Subject requires use of amiodarone.
  5. Subject used erythropoietin, etravirine, or gamma interferon within 3 months prior to screening.
  6. 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.
  7. 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.
  8. Male subject who has a QT interval corrected for heart rate using Fridericia's formula (QTcF) > 450 milliseconds or female subject who has a QTcF > 470 milliseconds on an ECG.
  9. Subject has a screening echocardiogram left ventricular ejection fraction < 45 percent.
  10. 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04519567


Contacts
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Contact: Rupal Patel 212-981-2715 rpatel@clinilabs.com

Locations
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United States, New Jersey
Clinilabs Drug Development Corporation Recruiting
Eatontown, New Jersey, United States, 07724
Contact: Rupal Patel    212-981-2715    rpatel@clinilabs.com   
Sponsors and Collaborators
Larimar Therapeutics, Inc.
Veristat, Inc.
Metrum Research Group, LLC
Investigators
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Principal Investigator: Magdy Shenouda, M.D. Clinilabs, Inc.
Additional Information:
Publications:
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Responsible Party: Larimar Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04519567    
Other Study ID Numbers: CLIN-1601-102
First Posted: August 19, 2020    Key Record Dates
Last Update Posted: August 19, 2020
Last Verified: August 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Ataxia
Cerebellar Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases