Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year

Completed · Phase 3 · Has a placebo group

Conditions studied: Amyotrophic Lateral Sclerosis

In brief

This study assessed the effect of tirasemtiv versus placebo on respiratory function in patients with ALS.

Key facts

Study ID: NCT02496767
Run by: Cytokinetics
People needed: 744
Starts: 2015-09-03
Expected to finish: 2017-09-27
Last updated by the study team: 2020-09-09

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

A diagnosis of familial or sporadic ALS (defined as meeting the possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the World Federation of Neurology El Escorial criteria) ≤ 24 months prior to screening
Upright SVC ≥ 70 % of predicted for age, height and sex
Able to swallow tablets without crushing, and in the opinion of the Investigator, is expected to continue to be able to do so during the trial
A caregiver if one is needed
Clinical laboratory findings within the normal range or, if outside the normal range, deemed not clinically significant by the Investigator
Male patients must agree for the duration of the study and 10 weeks after the end of the study to use a condom during sexual intercourse with female partners who are of childbearing potential (i.e., following menarche until post-menopausal if not anatomically and physiologically incapable of becoming pregnant) and to have female partners use an additional effective means of contraception (e.g., diaphragm plus spermicide, or oral contraceptives) or the male patient must agree to abstain from sexual intercourse during and for 10 weeks after the end of the study, unless the male patient has had a vasectomy and confirmed sperm count is zero
Female patients must be post-menopausal (≥ 1 year) or sterilized, or, if of childbearing potential, not be breastfeeding, have a negative pregnancy test, have no intention to become pregnant during the course of the study, and use effective contraceptive drugs or devices while requiring male partner to use a condom for the duration of the study and for 10 weeks after the end of the study
Patients must be either on a stable dose of riluzole 50 mg twice daily for at least 30 days prior to screening or have not taken riluzole for at least 30 days prior to screening and are willing not to begin riluzole use until they complete study drug dosing

You may not qualify if…

At the time of screening, any use of non-invasive positive pressure ventilation (NIPPV, e.g. continuous positive airway pressure [CPAP] or bi-level positive airway pressure [BiPAP]) for any portion of the day, or mechanical ventilation via tracheostomy, or on any form of oxygen supplementation
Patients with a diaphragm pacing system (DPS) at study entry or who anticipate DPS placement during the course of the study
BMI of 20.0 kg/m2 or lower
Unwilling or unable to discontinue tizanidine and theophylline-containing medications during study participation
Serum chloride outside the normal reference range
Neurological impairment due to a condition other than ALS, including history of transient ischemic attack within the past year
Presence at screening of any medically significant cardiac, pulmonary, GI, musculoskeletal, or psychiatric illness that might interfere with the patient's ability to comply with study procedures or that might confound the interpretation of clinical safety or efficacy data, including, but not limited to:
Poorly controlled hypertension
NYHA Class II or greater congestive heart failure
Chronic obstructive pulmonary disease or asthma requiring daily use bronchodilator medications
GI disorder that might impair absorption of study drug
History of significant liver disease defined by bilirubin > 2 times the upper limit of normal (ULN) or ALT or AST > 3 times the ULN on repeat testing
Poorly controlled diabetes mellitus
History of vertigo within three months of study entry
History of syncope without an explainable or treated cause
History of untreated intracranial aneurysm or poorly controlled seizure disorder
Amputation of a limb
Cognitive impairment, related to ALS or otherwise, sufficient to impair the patient's ability to give informed consent and to understand and/or comply with study procedures
Cancer with metastatic potential (other than basal cell carcinoma, carcinoma in situ of the cervix, or squamous cell carcinoma of the skin excised with clean margins) diagnosed and treated within the last two years
Any other condition, impairment or social circumstance that, in the opinion of the Investigator, would render the patient not suitable to participate in the study
Patient judged to be actively suicidal or a suicide risk by the Investigator
Has taken any investigational study drug within 30 days or five half-lives of the prior agent, whichever is greater, prior to dosing
Prior participation in any form of stem cell therapy for the treatment of ALS
Previously received tirasemtiv in any previous clinical trial

Where it is running

University of California San Diego — La Jolla, California, United States
Cedars-Sinai Medical Center — Los Angeles, California, United States
University of California, Irvine — Orange, California, United States
University of California Davis Medical Center — Sacramento, California, United States
Forbes Norris MDA/ALS Research Center — San Francisco, California, United States
Stanford Hospital and Clinics — Stanford, California, United States
University of Colorado Hospital Anschutz Outpatient Pavilion — Aurora, Colorado, United States
Hospital for Special Care — New Britain, Connecticut, United States
George Washington University Medical Center — Washington D.C., District of Columbia, United States
Mayo Clinic — Jacksonville, Florida, United States
University of Miami — Miami, Florida, United States
Carol and Frank Morsini Center for Advanced Health Care - University of South Florida — Tampa, Florida, United States
The Emory Clinic — Atlanta, Georgia, United States
Georgia Regents University — Augusta, Georgia, United States
Northwestern University Feinberg School of Medicine — Chicago, Illinois, United States
Indiana University — Indianapolis, Indiana, United States
University of Iowa Hospitals and Clinics — Iowa City, Iowa, United States
University of Kansas Medical Center — Kansas City, Kansas, United States
Johns Hopkins University — Baltimore, Maryland, United States
Massachusetts General Hospital — Boston, Massachusetts, United States
University of Massachusetts Memorial Medical Center — Worcester, Massachusetts, United States
University of Michigan Hospital and Health System — Ann Arbor, Michigan, United States
Henry Ford Health System — Detroit, Michigan, United States
Hennepin County Medical Center — Minneapolis, Minnesota, United States
St. Joseph's Hospital & Medical Center - Barrow Neurology Clinics — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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