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A Study of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS) |
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Study Focus:
The primary objective of this study is to demonstrate a pharmacodynamic effect of CK 2017357 on measures of skeletal muscle function or fatigability in patients with ALS. |
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Disease:
Amyotrophic Lateral Sclerosis (ALS), Sporadic ALS, Familial ALS |
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Study Category:
Drug Trial |
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Study Status:
Closed |
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Phase:
Phase II |
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Type:
Interventional Trial
with active agents/drugs & a placebo |
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Funding Source:
Cytokinetics |
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Study Chair(s)/Principal Investigator(s):
Jeremy M Shefner, MD, PhD (State University of New York - Upstate Medical University) |
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Clinicaltrials.gov ID:
NCT01089010 |
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NEALS Affiliated?
Yes
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Study Summary:
This study is a Phase II, double-blind, randomized, placebo-controlled, three-way crossover study of CK-2017357 in patients with ALS. 36 to 72 patients will be randomized to one of six different treatment sequences. Each treatment sequence consists of three dosing periods; in each dosing period¸ patients receive a single oral dose of placebo, 250 mg of CK-2017357, or 500 mg of CK-2017357. All six treatment sequences will enroll approximately the same number of patients. A washout period of at least 6 days (to a maximum of 10 days) will be employed between the doses for each patient. This study is designed to assess the effect of CK-2017357 on maximal voluntary muscle strength, on the development of fatigue at maximal and sub-maximal voluntary muscle contraction, and on selected pulmonary function parameters. The plasma concentration of CK-2017357 will be measured at selected time points after each of two single doses of CK-2017357 in men and women. The plasma concentration versus time data obtained in this study may be used to develop a population PK model and estimate inter-subject variability of PK parameters in this target patient population, in particular between male and female study patients.
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# of Subjects:
72 |
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Enrollment Start Date:
03/01/2010 |
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Enrollment End Date:
12/01/-0001 |
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Posting Last Modified Date:
11/01/2011 |
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Date Study Added to alscortium.org:
11/04/2011 |
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| Eligibility Criteria |
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Gender:
Male & Female |
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Minimum Age:
18 |
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Min Vital Capacity (% predicted normal):
40% |
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Time since Symptom Onset:
Not applicable |
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Time since Diagnosis:
Not applicable |
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Coordinating Center
Contact Information |
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| State University of New York - Upstate Medical University |
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Syracuse, New York 13210
UNITED STATES |
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Other Eligibility Criteria:
Inclusion Criteria:
•Able to comprehend and willing to sign an Informed Consent Form (ICF)
•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) (Brooks, et al., 2000)
•Males or Females 18 years of age or older
•Body mass index (BMI) of 18.0 to 30.0 kg/m2, inclusive
•Maximum voluntary grip strength between 10 & 40 pounds (females) and 10 & 60 pounds (males)
•Able to maintain grip contraction for 15 seconds
•Upright Slow Vital Capacity (SVC) ≥40% of predicted for age, height, and sex
•Able to perform pulmonary function tests
•Pre-study clinical laboratory findings (including troponin I (TnI) and CPK) within normal range, or if outside of the normal range, deemed not clinically significant by the Investigator
•For female patients only: The patient is post-menopausal (≥ 1 year) or sterilized, or if she is of childbearing potential, she is not breastfeeding, her pregnancy test is negative, she has no intention to become pregnant during the course of the study, and she is using contraceptive drugs or devices. For male patients only: Male patients 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 reproductive potential 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 for 10 weeks after the end of the study.
Exclusion Criteria:
•Significant hepatic/renal insufficiency as defined by Upper Limit of Normal (ULN) laboratory findings
•Life expectancy <3 months
•Participation in any trial in which receipt of investigational study drug occurred within 30 days prior to dosing
•Any prior treatment with CK-2017357
•In the opinion of the Investigator, the patient is not suitable to participate in the study
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Site Contact Information (Please click to show/hide) |
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| Arizona |
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Phoenix Neurological Associates, Ltd.
Phoenix, Arizona 85018
UNITED STATES |
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| California |
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University Neurology Associates
Fresno, California 93701
UNITED STATES |
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California Pacific Medical Center
San Francisco, California 94115
UNITED STATES | |
| Florida |
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Mayo Clinic Florida
Jacksonville, Florida 32224
UNITED STATES |
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| Kentucky |
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University of Kentucky
Lexington, Kentucky 40536
UNITED STATES |
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| Maryland |
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Johns Hopkins Hospital
Baltimore, Maryland 21287
UNITED STATES |
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| Massachusetts |
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Massachusetts General Hospital
Boston, Massachusetts 02114
UNITED STATES |
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| Missouri |
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Washington University
St Louis, Missouri 63110
UNITED STATES |
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| New York |
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SUNY Upstate Medical Center
Syracuse, New York 13210
UNITED STATES |
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| North Carolina |
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Duke University
Durham, North Carolina 27705
UNITED STATES |
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| Oregon |
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Providence ALS Center
Portland, Oregon 97213
UNITED STATES |
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| Pennsylvania |
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Drexel University College of Medicine, Dept of Neurology
Philadelphia, Pennsylvania 19102
UNITED STATES |
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Penn State
University Park, Pennsylvania 17033
UNITED STATES | |
| Texas |
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The University of Texas Health Science Center at San Antonio
San Antonio, Texas 78229
UNITED STATES |
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| Vermont |
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University of Vermont
Burlington, Vermont 05401
UNITED STATES |
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Familial ALS- ALS cases are inherited from 2 or more family members, affects 5-10% of ALS cases. Sporadic ALS- disease occurs at random with no clearly associated risk factors; most common form of ALS, affects approximately 90-95% ALS cases. Interventional trial- study where exposure, for example a drug, is assigned; used to determine the effectiveness of a treatment or intervention. With drugs and a placebo- One group gets the active treatment, the other gets the placebo. Everything else is held the same between the two groups, so that any difference in their outcome can be attributed to the active treatment. Observational study- study in which patients are observed and where no treatment is given; researcher has no control over the experiment because they are done in a more natural setting than an interventional trial. Trial Phase- objectives are different for each phase of a trial. - Phase I, drug is given to a small number of people to see if it is safe.
- Phase II begins to look at effectiveness of the drug.
- Phase III is considered the definitive test of whether a drug is effective, held at multiple research sites and much larger than the previous phases.
Vital Capacity- the maximum amount of air that can be expelled from the lungs after a maximum drawing of air into the lungs, measured by a spirometer and then scored as a percentage. |
