Dose Titration Study to Test Safety and Effects of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
A Phase II, Multicenter, Double-Blind, Randomized, Placebo-Controlled Dose Titration Study to Evaluate the Safety, Tolerability and Pharmacodynamic Effects of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
1 other identifier
interventional
27
1 country
11
Brief Summary
A Phase II, double-blind, randomized, placebo-controlled ascending dose titration study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamic effects of multiple ascending doses of CK-2017357 to an individual patient maximum tolerated dose (MTD), using a within-patient twice daily (BID) dose-titration regimen in ALS patients on 50 mg riluzole once daily (QD).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Nov 2011
Shorter than P25 for phase_2
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
November 1, 2011
CompletedFirst Submitted
Initial submission to the registry
November 23, 2011
CompletedFirst Posted
Study publicly available on registry
December 7, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2012
CompletedMay 3, 2019
April 1, 2019
4 months
November 23, 2011
April 30, 2019
Conditions
Outcome Measures
Primary Outcomes (1)
Number of participants with adverse events
approximately 29 days
Secondary Outcomes (11)
Change from baseline in score on the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
22 days
Change from baseline in scores on tests of maximum handgrip strength and handgrip fatigue
22 days
Change from baseline in scores on tests of muscle strength
22 days
Change from baseline in scores on tests of Timed Up and Go
22 days
Change from baseline in scores on tests of Sniff Nasal Inspiratory Pressure (SNIP)
22 days
- +6 more secondary outcomes
Study Arms (2)
Dose Titration of CK-2017357 (Group 1)
EXPERIMENTALDose titration of active drug as add-on therapy to riluzole
Matching Placebo (Group 2)
PLACEBO COMPARATORPlacebo as add-on therapy to riluzole
Interventions
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
Eligibility Criteria
You may qualify if:
- 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)
- Able to swallow tablets with water
- Currently taking and tolerating a stable dose of 50 mg BID riluzole
- Willing and able to reduce daily dose of riluzole to 50mg QD for 5 weeks
- Not currently taking or willing and able to remain off theophylline-containing medications during study participation
- Patient has a caregiver who is capable of observing and reporting patient status
- Upright Slow Vital Capacity (SVC) \>50% of predicted for age, height, and sex
- Able to perform pulmonary function tests
You may not qualify if:
- Life expectancy \<3 months
- Receipt of investigational study drug within 30 days or 5 half-lives of the prior agent, whichever is greater, prior to dosing
- Any prior treatment with CK-2017357
- Any use of non-invasive positive pressure ventilation (NIPPV), such as Continuous Positive Airway Pressure (CPAP) or Bilevel Positive Airway Pressure (BiPAP)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Cytokineticslead
Study Sites (11)
University of California at San Francisco, Fresno Campus, Central California Neurological Institute
Fresno, California, 93701, United States
Coordinated Clinical Research
La Jolla, California, 92037, United States
University of California at Irvine, ALS and Neuromuscular Center
Orange, California, 92868, United States
Hospital for Special Care
New Britain, Connecticut, 06053, United States
Massachusetts General Hospital, Neurology Clinical Trials Unit
Charlestown, Massachusetts, 02129, United States
Washington University
St Louis, Missouri, 63110, United States
Cornell Faculty, Hospital for Special Surgery
New York, New York, 10021, United States
Duke University School of Medicine, Division of Neurology
Durham, North Carolina, 27710, United States
Ohio State University, Department of Neurology
Columbus, Ohio, 43210, United States
Providence ALS Center
Portland, Oregon, 97213, United States
University of Texas Health Science Center, Department of Neurology
San Antonio, Texas, 78229, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Jeremy Shefner, MD, PhD
State University of New York - Upstate Medical University
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 23, 2011
First Posted
December 7, 2011
Study Start
November 1, 2011
Primary Completion
March 1, 2012
Study Completion
March 1, 2012
Last Updated
May 3, 2019
Record last verified: 2019-04