Dose Titration Study to Test Safety and Effects of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
NCT ID: NCT01486849
Last Updated: 2019-05-03
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE2
27 participants
INTERVENTIONAL
2011-11-30
2012-03-31
Brief Summary
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Detailed Description
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Potential patients will be screened to assess their eligibility to enter the study within 21 days prior to Day -7, when they will begin taking riluzole at the decreased dose of 50 mg QD. Patients will be randomized in a 3:1 ratio to CK-2017357 (Group 1) or placebo (Group 2). On Day 1, patients will begin taking a total daily dose of 250 mg (125 mg BID) of CK-2017357 or matching placebo tablets BID for 7 days. Then they will take a total daily dose of 375 mg (125 mg morning \[AM\] and 250 mg evening \[PM\]) of CK-2017357 or matching placebo tablets BID for 7 days, and finally, they will take a total daily dose of 500 mg (250 mg BID) of CK-2017357 or matching placebo tablets BID for 7 days. A final dose of 250 mg of CK-2017357 or placebo will be taken in the morning on Day 22 at the study site.
Dose-escalation of CK-2017357 or placebo may be stopped, or the dose reduced to a lower level, based on tolerability. All patients who return to a lower dose will stay on that dose for the remainder of the study.
Patients will remain on the decreased dose of riluzole until the follow-up visit approximately 7 days after Day 22.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Dose Titration of CK-2017357 (Group 1)
Dose titration of active drug as add-on therapy to riluzole
CK-2017357
Total daily oral dose of 250 mg (125 mg BID) of CK-2017357 for 7 days followed by total daily oral dose of 375 mg (125 mg AM and 250 mg PM) for 7 days followed by total daily oral dose of 500 mg (250 mg BID) of CK-2017357 for 7 days
Riluzole 50 MG
Matching Placebo (Group 2)
Placebo as add-on therapy to riluzole
Placebo
Matching placebo tablets BID for 21 days
Riluzole 50 MG
Interventions
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CK-2017357
Total daily oral dose of 250 mg (125 mg BID) of CK-2017357 for 7 days followed by total daily oral dose of 375 mg (125 mg AM and 250 mg PM) for 7 days followed by total daily oral dose of 500 mg (250 mg BID) of CK-2017357 for 7 days
Placebo
Matching placebo tablets BID for 21 days
Riluzole 50 MG
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Males or females 18 years of age or older
3. 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)
4. Maximum voluntary grip strength in at least one hand between 10 \& 40 pounds (females) and 10 \& 60 pounds (males)
5. Able to swallow tablets with water
6. Currently taking and tolerating a stable dose of 50 mg BID riluzole
7. Willing and able to reduce daily dose of riluzole to 50mg QD for 5 weeks
8. Not currently taking or willing and able to remain off theophylline-containing medications during study participation
9. Patient has a caregiver who is capable of observing and reporting patient status
10. Upright Slow Vital Capacity (SVC) \>50% of predicted for age, height, and sex
11. Able to perform pulmonary function tests
Exclusion Criteria
2. Receipt of investigational study drug within 30 days or 5 half-lives of the prior agent, whichever is greater, prior to dosing
3. Any prior treatment with CK-2017357
4. Any use of non-invasive positive pressure ventilation (NIPPV), such as Continuous Positive Airway Pressure (CPAP) or Bilevel Positive Airway Pressure (BiPAP)
18 Years
ALL
No
Sponsors
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Cytokinetics
INDUSTRY
Responsible Party
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Principal Investigators
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Jeremy Shefner, MD, PhD
Role: STUDY_CHAIR
State University of New York - Upstate Medical University
Locations
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University of California at San Francisco, Fresno Campus, Central California Neurological Institute
Fresno, California, United States
Coordinated Clinical Research
La Jolla, California, United States
University of California at Irvine, ALS and Neuromuscular Center
Orange, California, United States
Hospital for Special Care
New Britain, Connecticut, United States
Massachusetts General Hospital, Neurology Clinical Trials Unit
Charlestown, Massachusetts, United States
Washington University
St Louis, Missouri, United States
Cornell Faculty, Hospital for Special Surgery
New York, New York, United States
Duke University School of Medicine, Division of Neurology
Durham, North Carolina, United States
Ohio State University, Department of Neurology
Columbus, Ohio, United States
Providence ALS Center
Portland, Oregon, United States
University of Texas Health Science Center, Department of Neurology
San Antonio, Texas, United States
Countries
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Other Identifiers
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CY 4025
Identifier Type: -
Identifier Source: org_study_id
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