NCT02936635

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).

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
280

participants targeted

Target at P50-P75 for phase_3

Timeline
Completed

Started Oct 2016

Geographic Reach
11 countries

67 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 12, 2016

Completed
5 days until next milestone

Study Start

First participant enrolled

October 17, 2016

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 18, 2016

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 26, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 26, 2018

Completed
2.5 years until next milestone

Results Posted

Study results publicly available

May 12, 2021

Completed
Last Updated

June 15, 2021

Status Verified

May 1, 2021

Enrollment Period

2 years

First QC Date

October 12, 2016

Results QC Date

April 20, 2021

Last Update Submit

May 27, 2021

Conditions

Keywords

Amyotrophic Lateral SclerosisALStirasemtiv

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (4)

  • Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 24 in CY 4033

    baseline and 24 weeks

  • Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 48 in CY 4033

    baseline and 48 weeks

  • Change From CY 4031 Baseline in ALS Functional Rating Scale - Revised (ALSFRS-R) Total Score at Week 24

    baseline and 24 weeks

  • Change From CY 4031 Baseline in ALSFRS-R Total Score at Week 48

    baseline and 48 weeks

Study Arms (2)

Delayed Start Treatment

EXPERIMENTAL

The Delayed Start Treatment group consisted of patients who received placebo in CY 4031 and tirasemtiv in CY 4033.

Drug: tirasemtiv

Early Start Treatment

EXPERIMENTAL

The Early Start Treatment group consisted of patients who received tirasemtiv in both CY 4031 and CY 4033.

Drug: tirasemtiv

Interventions

Oral

Also known as: CK-2017357
Delayed Start TreatmentEarly Start Treatment

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (67)

St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics

Phoenix, Arizona, 85013, United States

Location

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

University of California, Irvine

Orange, California, 92868, United States

Location

UC Davis Medical Center

Sacramento, California, 95817, United States

Location

Forbes Norris MDA/ALS Research Center

San Francisco, California, 94115, United States

Location

Stanford Hospital and Clinics

Stanford, California, 94305, United States

Location

University of Colorado Hospital Anschutz Outpatient Pavilion

Aurora, Colorado, 80045, United States

Location

Hospital for Special Care

New Britain, Connecticut, 06053, United States

Location

George Washington University Medical Center

Washington D.C., District of Columbia, 20037, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

University of Miami, Miller School of Medicine

Miami, Florida, 33136, United States

Location

Carol & Frank Morsani Center for Advanced Health Care - University of South Florida

Tampa, Florida, 33512, United States

Location

The Emory Clinic

Atlanta, Georgia, 30322, United States

Location

Indiana University

Indianapolis, Indiana, 46202, United States

Location

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21287, United States

Location

University of Massachusetts Memorial Medical Center/University of Massachusetts Medical School

Worcester, Massachusetts, 01655, United States

Location

University of Michigan Health System

Ann Arbor, Michigan, 48109, United States

Location

Henry Ford Health System

Detroit, Michigan, 48202, United States

Location

Hennepin County Medical Center

Minneapolis, Minnesota, 55415, United States

Location

St. Louis University, Department of Neurology & Psychiatry

St Louis, Missouri, 63104, United States

Location

Barnes-Jewish Hospital

St Louis, Missouri, 63110, United States

Location

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, 03756, United States

Location

Hospital for Special Surgery

New York, New York, 10021, United States

Location

Neurological Institute

New York, New York, 10032, United States

Location

SUNY Upstate Medical University

Syracuse, New York, 13210, United States

Location

Neurosciences Institute, Neurology - Charlotte

Charlotte, North Carolina, 28207, United States

Location

Duke Neurological Disorders Clinic

Durham, North Carolina, 27705, United States

Location

Department of Neurology, Wake Forest School of Medicine

Winston-Salem, North Carolina, 27157, United States

Location

The Ohio State University Wexner Medical Center

Columbus, Ohio, 43210, United States

Location

Providence Brain and Spine Inst. ALS Center

Portland, Oregon, 97213, United States

Location

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Temple University School of Medicine

Philadelphia, Pennsylvania, 19140, United States

Location

Vanderbilt University Medical Center - Clinical Research Center

Nashville, Tennessee, 37232, United States

Location

Texas Neurology, PA

Dallas, Texas, 75214, United States

Location

UTHSCSA - First Outpatient Research Unit

San Antonio, Texas, 78229, United States

Location

University of Virginia Health System

Charlottesville, Virginia, 22908, United States

Location

University of Washington Medical Center

Seattle, Washington, 98195, United States

Location

West Virginia University Hospitals

Morgantown, West Virginia, 26506, United States

Location

Froedtert Memorial Lutheran Hospital

Milwaukee, Wisconsin, 53226, United States

Location

UZ Leuven

Leuven, Vlaams Brabant, 3000, Belgium

Location

University of Calgary

Calgary, Alberta, T2N 1N4, Canada

Location

University of Alberta

Edmonton, Alberta, T6G 1Z1, Canada

Location

Stan Cassidy Centre for Rehabilitation

Fredericton, New Brunswick, E3B 0C7, Canada

Location

QE II Health Sciences, Nova Scotia Health Authority

Halifax, Nova Scotia, B3H 3A7, Canada

Location

McMaster University Medical Centre

Hamilton, Ontario, L8N 3Z5, Canada

Location

London Health Sciences Centre

London, Ontario, N6A 5A5, Canada

Location

Sunnybrook Health Sciences Center

Toronto, Ontario, M4N 3M5, Canada

Location

Hopital Notre-Dame/CHUM

Montreal, Quebec, H2L 4M1, Canada

Location

Montreal Neurological Institute and Hospital

Montreal, Quebec, H3A 2B4, Canada

Location

CHU de Quebec - Univerite' Laval

Québec, Quebec, G1J 1Z4, Canada

Location

Hopital Dupuytren, service de neurologie

Limoges, 87042, France

Location

Hopital Gui de chauliac

Montpellier, 34295, France

Location

CHU de Nice - Hopital Pasteur 2

Nice, 06001, France

Location

Hopital Bretonneau

Tours, 37044, France

Location

University of Ulm, Department of Neurology

Ulm, Baden-Wurttemberg, 89081, Germany

Location

Hannover Medical School, Department of Neurology

Hanover, Lower Saxony, 30625, Germany

Location

Charite Campus Virchow-Klinikum, Department of Neurology

Berlin, 13353, Germany

Location

Clinical Research Centre Beaumont Hospital

Dublin, Dublin 9, Ireland

Location

IRCCS Istituto Auxologico Italiano - U.O. Neurologia

Milan, Lombardy, 20149, Italy

Location

Centro Clinico NEMO - Fondazione Serena Onlus, ASST Grande Ospedale Metropolitano Niguarda, Ospedale Niguarda

Milan, Lombardy, 20162, Italy

Location

Dipartimento di Neuroscienze "Rita Levi Moltalcini" A.O.U. Citta della Salute e della Scienza di Torino P.O. "Molinette"

Turin, Piedmont, 10126, Italy

Location

University Medical Center Utrecht

Utrecht, 3584 CX, Netherlands

Location

Hospital Santa Maria - Centro Hospitalar Lisboa Norte

Lisbon, 1649-035, Portugal

Location

Hospital San Rafael

Madrid, 28016, Spain

Location

King's College Hospital NHS Foundation Trust

London, SE5 9RS, United Kingdom

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

CK-2017357

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Results Point of Contact

Title
MD Cytokinetics
Organization
Cytokinetics, Inc.

Study Officials

  • MD, Cytokinetics

    Cytokinetics, Inc.

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 12, 2016

First Posted

October 18, 2016

Study Start

October 17, 2016

Primary Completion

October 26, 2018

Study Completion

October 26, 2018

Last Updated

June 15, 2021

Results First Posted

May 12, 2021

Record last verified: 2021-05

Locations