Member Portal    
ALS Trials
Browse Recruiting Trials
Search for a Trial
Clinical Trials 101
Questions about Clinical Research?
Contact an ALS Trial Liaison

Call or email us with your questions
about clinical research

(877) 458-0631

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

Study Focus:
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).
Amyotrophic Lateral Sclerosis (ALS), Sporadic ALS, Familial ALS
Study Category:
Drug Trial
Study Status:
Phase II
Interventional Trial
with active agents/drugs & a placebo
Funding Source:
Study Chair(s)/Principal Investigator(s):
Jeremy Shefner, MD, PhD (State University of New York - Upstate Medical University) ID:
NEALS Affiliated?
Study Summary:
Patients will be randomized to one of two dosing groups, active CK-2017357 or placebo, in a 3:1 ratio. Prior to study drug dosing, patients will be required to decrease their riluzole dose to 50 mg QD for 7 days; after this 7 day period patients will either receive placebo or start the titration on active CK-2017357 while continuing to take riluzole at 50 mg QD.

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.
Participant Duration:
21 days on treatment, 29 days total
# of Subjects:
Enrollment Start Date:
Posting Last Modified Date:
Date Study Added to
Eligibility Criteria
Male & Female
Minimum Age:
Min Vital Capacity (% predicted normal):
Can participants use Riluzole?
Riluzole use required by protocol
Coordinating Center
Contact Information
Jean Masonek
Other Eligibility Criteria:
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)
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

Exclusion Criteria:

Life expectancy less than 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)

Other protocol-defined inclusion/exclusion criteria may apply.

Site Contact Information

 (Please click to show/hide)
For Patients & Families
Educational Webinars
Online NEALS Resources
Other Resources
Contact a Disease Specialist
Clinical Research Blog

For Researchers
Become a NEALS member
Post an ALS Clinical Trial
NEALS CRO Capabilities
Annual NEALS Meeting
NEALS Samples
NEALS Historical Data
ALS Trials
Browse Recruiting Trials
Search for a Trial
Clinical Trial News
Clinical Trials 101

Get Involved
Donate samples
Donate to our Clinical Research
About Us
Our Mission
Our Structure
Our Research
NEALS Members
Motor Neuron Disease
Connect with us: Member Portal       Donate Now!       Help       Sitemap       Contact Us