Amyotrophic Lateral Sclerosis (ALS) Clinical Trial
— VIGOR-ALSOfficial title:
A Phase 3, Open-Label Extension Study of Tirasemtiv for Patients With Amyotrophic Lateral Sclerosis (ALS) Who Completed VITALITY-ALS (CY 4031)
Verified date | May 2021 |
Source | Cytokinetics |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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).
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 |
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 |
Lead Sponsor | Collaborator |
---|---|
Cytokinetics |
United States, Belgium, Canada, France, Germany, Ireland, Italy, Netherlands, Portugal, Spain, United Kingdom,
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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