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A Phase II, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Effects of Multiple Doses of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS) |
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Study Focus:
The study will generate data on safety and tolerability after multiple daily doses of CK-2017357 in patients with ALS. Patients will be randomized into one of four different treatment groups, receiving daily oral doses of either placebo, 125 mg, 250 mg, or 375 mg of CK-2017357 for 14 days. |
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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 Shefner, MD, PhD (SUNY) |
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Clinicaltrials.gov ID:
NCT01378676 |
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NEALS Affiliated?
No |
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Study Summary:
Patients will be randomized to one of four different treatment groups: daily oral doses of placebo, 125 mg, 250 mg, or 375 mg of CK-2017357. Patients will be required to decrease their riluzole dose to 50 mg once a day (QD) for 7 days prior to randomization. After this 7 day period, patients will take riluzole at 50 mg QD concurrently with their morning dose of blinded study drug for 14 days. |
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Participant Duration:
21 days
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# of Subjects:
50 |
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Enrollment Start Date:
06/01/2011 |
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Enrollment End Date:
12/01/-0001 |
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Posting Last Modified Date:
03/01/2012 |
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Date Study Added to alscortium.org:
11/01/2011 |
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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):
50% |
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Time since Symptom Onset:
Not applicable |
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Time since Diagnosis:
Not applicable |
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Can participants use Riluzole?
Riluzole use required by protocol |
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Coordinating Center
Contact Information |
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| Jean Masonek |
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| jmasonek@cytokinetics.com |
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| Cytokinetics |
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280 East Grand Avenue
California 94080
UNITED STATES |
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Other Eligibility Criteria:
Key Inclusion Criteria:
Able to comprehend and willing to sign an Informed Consent Form (ICF)
Males or females 18 years of age or older
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)
Maximum voluntary grip strength in at least one hand between 10 & 40 pounds (females) and 10 & 60 pounds (males)
Upright Slow Vital Capacity (SVC) >50% of predicted for age, height, and sex
Able to swallow tablets with water
Willing and able to refrain from caffeine-containing products during study participation
Willing and able to remain off warfarin and theophylline-containing medications during study participation
Has a caregiver who is capable of observing and reporting patient status, and also assisting in the proper use of nocturnal oximetry equipment
Able to perform pulmonary function tests
Key Exclusion Criteria:
Life expectancy <3 months
Participation in any trial in which receipt of investigational study drug occurred within 30 days or 5 half-lives of the prior agent, whichever is greater, prior to dosing
Any prior treatment with CK-2017357
Use of non-invasive positive pressure ventilation (NIPPV) for any part of the day or night
Other protocol-defined inclusion/exclusion criteria may apply.
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Site Contact Information (Please click to show/hide) |
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| California |
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California Pacific Medical Center
San Francisco, California 94115
UNITED STATES |
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| Florida |
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Mayo Florida
Jacksonville, Florida 32224
UNITED STATES |
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| Kansas |
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University of Kansas
Kansas City, Kansas 66045
UNITED STATES |
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| Maryland |
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Johns Hopkins Hospital
Baltimore, Maryland 21287
UNITED STATES |
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| New York |
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Columbia University Medical Center
New York, New York 10032
UNITED STATES |
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SUNY Upstate Medical Center
Syracuse, New York 13210
UNITED STATES | |
| North Carolina |
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Carolinas Neuromuscular ALS-MND Center
Charlotte, North Carolina 28207
UNITED STATES |
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| Pennsylvania |
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Penn State Hershey Medical Center
Hershey, Pennsylvania 17033
UNITED STATES |
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Drexel University College of Medicine
Philadelphia, Pennsylvania 19107
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. |
