Amyotrophic Lateral Sclerosis (ALS)Phase 3VIGOR-ALSNCT02936635
A Study for Patients Who Completed VITALITY-ALS (CY 4031)
A Phase 3 study testing tirasemtiv in people with Amyotrophic Lateral Sclerosis (ALS), run by Cytokinetics at 67 sites in 11 countries.
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- tirasemtiv
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 280 participants
- Where
- 67 sitesin 11 countries
- Study dates
- 17 Oct 2016to 26 Oct 2018
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
Show all 10Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
67 sites in 11 countries.
67 sites in 11 countries
United States41 sites
University of Michigan Health System
Ann Arbor, Michigan, 48109
The Emory Clinic
Atlanta, Georgia, 30322
University of Colorado Hospital Anschutz Outpatient Pavilion
Aurora, Colorado, 80045
Johns Hopkins University
Baltimore, Maryland, 21287
Neurosciences Institute, Neurology - Charlotte
Charlotte, North Carolina, 28207
University of Virginia Health System
Charlottesville, Virginia, 22908
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210
Texas Neurology, PA
Dallas, Texas, 75214
Henry Ford Health System
Detroit, Michigan, 48202
Duke Neurological Disorders Clinic
Durham, North Carolina, 27705
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
Indiana University
Indianapolis, Indiana, 46202
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
Mayo Clinic
Jacksonville, Florida, 32224
University of Kansas Medical Center
Kansas City, Kansas, 66160
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, 03756
Cedars-Sinai Medical Center
Los Angeles, California, 90048
University of Miami, Miller School of Medicine
Miami, Florida, 33136
Froedtert Memorial Lutheran Hospital
Milwaukee, Wisconsin, 53226
Hennepin County Medical Center
Minneapolis, Minnesota, 55415
West Virginia University Hospitals
Morgantown, West Virginia, 26506
Vanderbilt University Medical Center - Clinical Research Center
Nashville, Tennessee, 37232
Hospital for Special Care
New Britain, Connecticut, 06053
Hospital for Special Surgery
New York, New York, 10021
Neurological Institute
New York, New York, 10032
University of California, Irvine
Orange, California, 92868
Temple University School of Medicine
Philadelphia, Pennsylvania, 19140
St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics
Phoenix, Arizona, 85013
Providence Brain and Spine Inst. ALS Center
Portland, Oregon, 97213
UC Davis Medical Center
Sacramento, California, 95817
UTHSCSA - First Outpatient Research Unit
San Antonio, Texas, 78229
Forbes Norris MDA/ALS Research Center
San Francisco, California, 94115
University of Washington Medical Center
Seattle, Washington, 98195
St. Louis University, Department of Neurology & Psychiatry
St Louis, Missouri, 63104
Barnes-Jewish Hospital
St Louis, Missouri, 63110
Stanford Hospital and Clinics
Stanford, California, 94305
SUNY Upstate Medical University
Syracuse, New York, 13210
Carol & Frank Morsani Center for Advanced Health Care - University of South Florida
Tampa, Florida, 33512
George Washington University Medical Center
Washington D.C., District of Columbia, 20037
Department of Neurology, Wake Forest School of Medicine
Winston-Salem, North Carolina, 27157
University of Massachusetts Memorial Medical Center/University of Massachusetts Medical School
Worcester, Massachusetts, 01655
Canada10 sites
University of Calgary
Calgary, Alberta, T2N 1N4
University of Alberta
Edmonton, Alberta, T6G 1Z1
Stan Cassidy Centre for Rehabilitation
Fredericton, New Brunswick, E3B 0C7
QE II Health Sciences, Nova Scotia Health Authority
Halifax, Nova Scotia, B3H 3A7
McMaster University Medical Centre
Hamilton, Ontario, L8N 3Z5
London Health Sciences Centre
London, Ontario, N6A 5A5
Hopital Notre-Dame/CHUM
Montreal, Quebec, H2L 4M1
Montreal Neurological Institute and Hospital
Montreal, Quebec, H3A 2B4
CHU de Quebec - Univerite' Laval
Québec, Quebec, G1J 1Z4
Sunnybrook Health Sciences Center
Toronto, Ontario, M4N 3M5
France4 sites
Hopital Dupuytren, service de neurologie
Limoges, 87042
Hopital Gui de chauliac
Montpellier, 34295
CHU de Nice - Hopital Pasteur 2
Nice, 06001
Hopital Bretonneau
Tours, 37044
Germany3 sites
Charite Campus Virchow-Klinikum, Department of Neurology
Berlin, 13353
Hannover Medical School, Department of Neurology
Hanover, Lower Saxony, 30625
University of Ulm, Department of Neurology
Ulm, Baden-Wurttemberg, 89081
Italy3 sites
IRCCS Istituto Auxologico Italiano - U.O. Neurologia
Milan, Lombardy, 20149
Centro Clinico NEMO - Fondazione Serena Onlus, ASST Grande Ospedale Metropolitano Niguarda, Ospedale Niguarda
Milan, Lombardy, 20162
Dipartimento di Neuroscienze "Rita Levi Moltalcini" A.O.U. Citta della Salute e della Scienza di Torino P.O. "Molinette"
Turin, Piedmont, 10126
Portugal1 site
Hospital Santa Maria - Centro Hospitalar Lisboa Norte
Lisbon, 1649-035
United Kingdom1 site
King's College Hospital NHS Foundation Trust
London, SE5 9RS
About this study
Official title: A Phase 3, Open-Label Extension Study of Tirasemtiv for Patients With Amyotrophic Lateral Sclerosis (ALS) Who Completed VITALITY-ALS (CY 4031)
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).
Read the 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.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- tirasemtiv
- Keywords
- Amyotrophic Lateral Sclerosis, ALS, tirasemtiv
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
tirasemtivDrug
Also called CK-2017357
Oral
| Study group | Role | What they receive |
|---|---|---|
| Delayed Start Treatment | Experimental | The Delayed Start Treatment group consisted of patients who received placebo in CY 4031 and tirasemtiv in CY 4033. Receives: Drug: tirasemtiv |
| Early Start Treatment | Experimental | The Early Start Treatment group consisted of patients who received tirasemtiv in both CY 4031 and CY 4033. Receives: Drug: tirasemtiv |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From the first dose of tirasemtiv through 28 days after the last dose
Secondary outcomes (4)
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]).
Measured: baseline and 24 weeks
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]).
Measured: baseline and 48 weeks
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.
Measured: baseline and 24 weeks
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.
Measured: baseline and 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2016 (estimated)
- Study start17 Oct 2016
- Primary completion26 Oct 2018
- Study completion26 Oct 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 280 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cytokinetics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- MD, Cytokinetics, Study director, Cytokinetics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02936635
- Sponsor's protocol ID
- CY 4033
- Other IDs
- 2016-002629-13, 2016
- Acronym
- VIGOR-ALS
- Status last verified
- May 2021
- First submitted
- 12 Oct 2016
- Last updated
- 15 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Open-Label Extension Study of Tirasemtiv for Patients With Amyotrophic Lateral Sclerosis (ALS) Who Completed VITALITY-ALS (CY 4031)
- ClinicalTrials.gov ID
- NCT02936635
- Acronym / sponsor ID
- VIGOR-ALS / CY 4033
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Keywords
- Amyotrophic Lateral Sclerosis, ALS, tirasemtiv
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 280 participants (actual)
- Lead sponsor
- CytokineticsIndustry (pharma, biotech, devices)
- Study start
- 17 Oct 2016 (actual)
- Primary completion
- 26 Oct 2018 (actual)
- Study completion
- 26 Oct 2018 (actual)
- First posted
- 18 Oct 2016 (estimated)
- Results
- Posted 12 May 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Delayed Start Treatment | Experimental | Drug: tirasemtivThe Delayed Start Treatment group consisted of patients who received placebo in CY 4031 and tirasemtiv in CY 4033. |
| Early Start Treatment | Experimental | Drug: tirasemtivThe Early Start Treatment group consisted of patients who received tirasemtiv in both CY 4031 and CY 4033. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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:
- Female patients who are not post-menopausal (≥ 1 year) or sterilized, must:
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| tirasemtivAlso: CK-2017357Oral | Drug | Delayed Start Treatment, Early Start Treatment |
Primary outcome
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.
4 secondary outcomes
Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 24 in CY 4033
Time frame: baseline and 24 weeks
Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 48 in CY 4033
Time frame: baseline and 48 weeks
Change From CY 4031 Baseline in ALS Functional Rating Scale - Revised (ALSFRS-R) Total Score at Week 24
Time frame: baseline and 24 weeks
Change From CY 4031 Baseline in ALSFRS-R Total Score at Week 48
Time frame: baseline and 48 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
MD, Cytokinetics study director
Cytokinetics, Inc.
67 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing tirasemtiv in people with Amyotrophic Lateral Sclerosis (ALS), run by Cytokinetics at 67 sites in 11 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 67 sites in United States, Canada, France, Germany, Italy and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 May 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events (time frame: From the first dose of tirasemtiv through 28 days after the last dose). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jun 2021.