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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02936635
Other study ID # CY 4033
Secondary ID 2016-002629-13
Status Completed
Phase Phase 3
First received
Last updated
Start date October 17, 2016
Est. completion date October 26, 2018

Study information

Verified date May 2021
Source Cytokinetics
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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).


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.


Recruitment information / eligibility

Status Completed
Enrollment 280
Est. completion date October 26, 2018
Est. primary completion date October 26, 2018
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
tirasemtiv
Oral

Locations

Country Name City State
Belgium UZ Leuven Leuven Vlaams Brabant
Canada University of Calgary Calgary Alberta
Canada University of Alberta Edmonton Alberta
Canada Stan Cassidy Centre for Rehabilitation Fredericton New Brunswick
Canada QE II Health Sciences, Nova Scotia Health Authority Halifax Nova Scotia
Canada McMaster University Medical Centre Hamilton Ontario
Canada London Health Sciences Centre London Ontario
Canada Hopital Notre-Dame/CHUM Montreal Quebec
Canada Montreal Neurological Institute and Hospital Montreal Quebec
Canada CHU de Quebec - Univerite' Laval Quebec City Quebec
Canada Sunnybrook Health Sciences Center Toronto Ontario
France Hopital Dupuytren, service de neurologie Limoges Cedex
France Hopital Gui de chauliac Montpellier Cedex 5
France CHU de Nice - Hopital Pasteur 2 Nice Cedex 1
France Hopital Bretonneau Tours cedex 9
Germany Charite Campus Virchow-Klinikum, Department of Neurology Berlin
Germany Hannover Medical School, Department of Neurology Hannover Lower Saxony
Germany University of Ulm, Department of Neurology Ulm Baden-Wuerttemberg
Ireland Clinical Research Centre Beaumont Hospital Dublin
Italy Centro Clinico NEMO - Fondazione Serena Onlus, ASST Grande Ospedale Metropolitano Niguarda, Ospedale Niguarda Milan Lombardy
Italy IRCCS Istituto Auxologico Italiano - U.O. Neurologia Milan Lombardy
Italy Dipartimento di Neuroscienze "Rita Levi Moltalcini" A.O.U. Citta della Salute e della Scienza di Torino P.O. "Molinette" Torino Piemonte
Netherlands University Medical Center Utrecht Utrecht
Portugal Hospital Santa Maria - Centro Hospitalar Lisboa Norte Lisboa
Spain Hospital San Rafael Madrid
United Kingdom King's College Hospital NHS Foundation Trust London
United States University of Michigan Health System Ann Arbor Michigan
United States The Emory Clinic Atlanta Georgia
United States University of Colorado Hospital Anschutz Outpatient Pavilion Aurora Colorado
United States Johns Hopkins University Baltimore Maryland
United States Neurosciences Institute, Neurology - Charlotte Charlotte North Carolina
United States University of Virginia Health System Charlottesville Virginia
United States The Ohio State University Wexner Medical Center Columbus Ohio
United States Texas Neurology, PA Dallas Texas
United States Henry Ford Health System Detroit Michigan
United States Duke Neurological Disorders Clinic Durham North Carolina
United States Penn State Milton S. Hershey Medical Center Hershey Pennsylvania
United States Indiana University Indianapolis Indiana
United States University of Iowa Hospitals and Clinics Iowa City Iowa
United States Mayo Clinic Jacksonville Florida
United States University of Kansas Medical Center Kansas City Kansas
United States Dartmouth Hitchcock Medical Center Lebanon New Hampshire
United States Cedars-Sinai Medical Center Los Angeles California
United States University of Miami, Miller School of Medicine Miami Florida
United States Froedtert Memorial Lutheran Hospital Milwaukee Wisconsin
United States Hennepin County Medical Center Minneapolis Minnesota
United States West Virginia University Hospitals Morgantown West Virginia
United States Vanderbilt University Medical Center - Clinical Research Center Nashville Tennessee
United States Hospital for Special Care New Britain Connecticut
United States Hospital for Special Surgery New York New York
United States Neurological Institute New York New York
United States University of California, Irvine Orange California
United States Temple University School of Medicine Philadelphia Pennsylvania
United States St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics Phoenix Arizona
United States Providence Brain and Spine Inst. ALS Center Portland Oregon
United States UC Davis Medical Center Sacramento California
United States Barnes-Jewish Hospital Saint Louis Missouri
United States St. Louis University, Department of Neurology & Psychiatry Saint Louis Missouri
United States UTHSCSA - First Outpatient Research Unit San Antonio Texas
United States Forbes Norris MDA/ALS Research Center San Francisco California
United States University of Washington Medical Center Seattle Washington
United States Stanford Hospital and Clinics Stanford California
United States SUNY Upstate Medical University Syracuse New York
United States Carol & Frank Morsani Center for Advanced Health Care - University of South Florida Tampa Florida
United States George Washington University Medical Center Washington District of Columbia
United States Department of Neurology, Wake Forest School of Medicine Winston-Salem North Carolina
United States University of Massachusetts Memorial Medical Center/University of Massachusetts Medical School Worcester Massachusetts

Sponsors (1)

Lead Sponsor Collaborator
Cytokinetics

Countries where clinical trial is conducted

United States,  Belgium,  Canada,  France,  Germany,  Ireland,  Italy,  Netherlands,  Portugal,  Spain,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of Participants With Adverse Events The number of participants with adverse events was used as the measure for the long-term safety and tolerability of tirasemtiv. From the first dose of tirasemtiv through 28 days after the last dose
Secondary Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 24 in CY 4033 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]). baseline and 24 weeks
Secondary Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 48 in CY 4033 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]). baseline and 48 weeks
Secondary Change From CY 4031 Baseline in ALS Functional Rating Scale - Revised (ALSFRS-R) Total Score at Week 24 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. baseline and 24 weeks
Secondary Change From CY 4031 Baseline in ALSFRS-R Total Score at Week 48 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. baseline and 48 weeks
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