Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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 : May 12, 2021
Sponsor:
Information provided by (Responsible Party):
Cytokinetics

Tracking Information
First Submitted Date  ICMJE October 12, 2016
First Posted Date  ICMJE October 18, 2016
Results First Submitted Date  ICMJE April 20, 2021
Results First Posted Date  ICMJE May 12, 2021
Last Update Posted Date May 12, 2021
Actual Study Start Date  ICMJE October 17, 2016
Actual Primary Completion Date October 26, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 20, 2021)
The Incidence of Adverse Events [ Time Frame: From the first dose of tirasemtiv through 28 days after the last dose ]
The incidence of adverse events was used as the measure for the long-term safety and tolerability of tirasemtiv.
Original Primary Outcome Measures  ICMJE
 (submitted: October 14, 2016)
Incidence of adverse events (AEs) in patient population [ Time Frame: Until end of study, up to 36 months ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 20, 2021)
  • 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 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]).
  • 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]).
  • 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.
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: October 14, 2016)
  • Time to first use of assisted ventilation or death [ Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months ]
  • Time to the first occurrence of respiratory insufficiency (defined as tracheostomy or the use of non-invasive ventilation (NIV) for ≥22 hours per day for ≥10 consecutive days) or death [ Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months ]
  • Time to death [ Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months ]
  • Decline in percent predicted Slow Vital Capacity (SVC) from baseline [ Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months ]
  • Decline in ALS Functional Rating Scale - Revised (ALSFRS-R) score from baseline [ Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months ]
  • Slope of the change from baseline in percent predicted SVC [ Time Frame: First 24 weeks and first 48 weeks of either CY 4031 or CY 4033 ]
  • Slope of the change from baseline in ALSFRS-R [ Time Frame: First 24 weeks and first 48 weeks of either CY 4031 or CY 4033 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study for Patients Who Completed VITALITY-ALS (CY 4031)
Official Title  ICMJE A Phase 3, Open-Label Extension Study of Tirasemtiv for Patients With Amyotrophic Lateral Sclerosis (ALS) Who Completed VITALITY-ALS (CY 4031)
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).
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Amyotrophic Lateral Sclerosis (ALS)
Intervention  ICMJE Drug: tirasemtiv
Oral
Other Name: CK-2017357
Study Arms  ICMJE
  • Experimental: Delayed Start Treatment
    The Delayed Start Treatment group consisted of patients who received placebo in CY 4031 and tirasemtiv in CY 4033.
    Intervention: Drug: tirasemtiv
  • Experimental: Early Start Treatment
    The Early Start Treatment group consisted of patients who received tirasemtiv in both CY 4031 and CY 4033.
    Intervention: Drug: tirasemtiv
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 6, 2017)
280
Original Estimated Enrollment  ICMJE
 (submitted: October 14, 2016)
350
Actual Study Completion Date  ICMJE October 26, 2018
Actual Primary Completion Date October 26, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   Canada,   France,   Germany,   Ireland,   Italy,   Netherlands,   Portugal,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02936635
Other Study ID Numbers  ICMJE CY 4033
2016-002629-13 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Cytokinetics
Study Sponsor  ICMJE Cytokinetics
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: MD, Cytokinetics Cytokinetics, Inc.
PRS Account Cytokinetics
Verification Date April 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP