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A Study for Patients Who Completed VITALITY-ALS (CY 4031) (VIGOR-ALS)

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02936635
Recruitment Status : Completed
First Posted : October 18, 2016
Results First Posted : May 12, 2021
Last Update Posted : June 15, 2021
Sponsor:
Information provided by (Responsible Party):
Cytokinetics

Study Description
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Brief Summary:
The purpose of this study is to assess the long-term safety and tolerability of tirasemtiv in patients with ALS who had completed the double-blind placebo-controlled study of tirasemtiv in ALS (CY 4031).

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis (ALS) Drug: tirasemtiv Phase 3

Detailed Description:
Enrolled participants will begin dosing of tirasemtiv 125 mg twice daily (250 mg/day) for a period of 4 weeks and will titrate to their tolerated dose, the maximum dose being 250 mg twice daily (500 mg/day). This study will also compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 with those who completed treatment with placebo in CY 4031 during continued treatment of both groups with tirasemtiv during CY 4033, compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 during that study with their clinical course during continued treatment with tirasemtiv during CY 4033, and compare the clinical course of patients who completed treatment with placebo in CY 4031 during that study with their clinical course during treatment with tirasemtiv during CY 4033.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 280 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Open-Label Extension Study of Tirasemtiv for Patients With Amyotrophic Lateral Sclerosis (ALS) Who Completed VITALITY-ALS (CY 4031)
Actual Study Start Date : October 17, 2016
Actual Primary Completion Date : October 26, 2018
Actual Study Completion Date : October 26, 2018

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Delayed Start Treatment
The Delayed Start Treatment group consisted of patients who received placebo in CY 4031 and tirasemtiv in CY 4033.
 Drug: tirasemtiv
Oral
Other Name: CK-2017357
Experimental: Early Start Treatment
The Early Start Treatment group consisted of patients who received tirasemtiv in both CY 4031 and CY 4033.
 Drug: tirasemtiv
Oral
Other Name: CK-2017357


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Adverse Events [ Time Frame: From the first dose of tirasemtiv through 28 days after the last dose ]
    The number of participants with adverse events was used as the measure for the long-term safety and tolerability of tirasemtiv.


Secondary Outcome Measures :
  1. Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 24 in CY 4033 [ Time Frame: baseline and 24 weeks ]
    Slow vital capacity (SVC) was measured using a spirometer (in units of liters). Following 3 to 5 breaths at rest, patients were instructed to take as deep an inspiration as possible followed by a maximum exhalation (blowing out all the air in their lungs). Values obtained were converted to percent predicted values (ie, the test result as a percent of predicted values for patients of similar demographic and baseline characteristics [eg, height, age, sex]).

  2. Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 48 in CY 4033 [ Time Frame: baseline and 48 weeks ]
    Slow vital capacity was measured using a spirometer (in units of liters). Following 3 to 5 breaths at rest, patients were instructed to take as deep an inspiration as possible followed by a maximum exhalation (blowing out all the air in their lungs). Values obtained were converted to percent predicted values (ie, the test result as a percent of predicted values for patients of similar demographic and baseline characteristics [eg, height, age, sex]).

  3. Change From CY 4031 Baseline in ALS Functional Rating Scale - Revised (ALSFRS-R) Total Score at Week 24 [ Time Frame: baseline and 24 weeks ]
    The ALSFRS-R is used to measure the progression and severity of disability in patients with ALS. The ALSFRS-R consists of 12 questions, assessing a patient's capability and independence in functional activities relevant to ALS, categorized in the following 4 domains: gross motor tasks, fine motor tasks, bulbar functions, and respiratory function. Each question is scored from 0 (indicating incapable or dependent) to 4 (normal). The total score ranges from 0 to 48, with higher scores reflecting more normal function and lower scores reflecting more impaired function. Comparing postbaseline to baseline assessments, a negative value indicates a worsening in function.

  4. Change From CY 4031 Baseline in ALSFRS-R Total Score at Week 48 [ Time Frame: baseline and 48 weeks ]
    The ALSFRS-R is used to measure the progression and severity of disability in patients with ALS. The ALSFRS-R consists of 12 questions, assessing a patient's capability and independence in functional activities relevant to ALS, categorized in the following 4 domains: gross motor tasks, fine motor tasks, bulbar functions, and respiratory function. Each question is scored from 0 (indicating incapable or dependent) to 4 (normal). The total score ranges from 0 to 48, with higher scores reflecting more normal function and lower scores reflecting more impaired function. Comparing postbaseline to baseline assessments, a negative value indicates a worsening in function.


Eligibility Criteria
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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:

  • Able to comprehend and willing to sign an Informed Consent Form (ICF). If verbal consent is given, a Legal Designee of the patient must sign the ICF form
  • Completed participation on study drug and the Follow-Up Visit in the CY 4031 study

  • Male patients, who have not had a vasectomy AND confirmed zero sperm count, must agree for the duration of their participation in the study to either:

    • Use a condom during sexual intercourse with female partners who are of childbearing potential AND to have female partners use a highly effective means of contraception OR
    • Abstain from sexual intercourse during participation in the study

  • Female patients who are not post-menopausal (≥ 1 year) or sterilized, must:

    • Not be breastfeeding
    • Have a negative pregnancy test
    • Have no intention to become pregnant during participation in the study AND
    • Practice sexual abstinence, defined as refraining from intercourse during the duration of the study OR if male partners are not vasectomized with a confirmed zero sperm count, require use of a condom AND use of a highly effective contraceptive measure

Exclusion Criteria:

  • Has a diaphragm pacing system (DPS) at study entry or anticipate DPS placement during the course of the study
  • Has taken an investigational study drug (other than tirasemtiv) prior to dosing, within 30 days or five half-lives of the prior agent, whichever is greater
  • Use of tizanidine and theophylline-containing medications during study participation
  • Participation or planning to participate in any form of stem cell therapy for the treatment of ALS or another investigational drug
Contacts and Locations
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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): NCT02936635


Locations
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Sponsors and Collaborators
Cytokinetics
Investigators
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Study Director: MD, Cytokinetics Cytokinetics, Inc.
  Study Documents (Full-Text)

Documents provided by Cytokinetics:
Study Protocol  [PDF] July 13, 2016
Statistical Analysis Plan  [PDF] October 24, 2017

More Information
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Responsible Party: Cytokinetics
ClinicalTrials.gov Identifier: NCT02936635    
Other Study ID Numbers: CY 4033
2016-002629-13 ( EudraCT Number )
First Posted: October 18, 2016    Key Record Dates
Results First Posted: May 12, 2021
Last Update Posted: June 15, 2021
Last Verified: May 2021
Keywords provided by Cytokinetics:
Amyotrophic Lateral Sclerosis
ALS
tirasemtiv
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
 Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases