A Study to Evaluate the Effects of Multiple Doses of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
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)
1 other identifier
interventional
49
1 country
9
Brief Summary
The study will generate data on safety, tolerability and pharmacokinetics 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Jun 2011
Shorter than P25 for phase_2
9 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 1, 2011
CompletedFirst Submitted
Initial submission to the registry
June 20, 2011
CompletedFirst Posted
Study publicly available on registry
June 22, 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 7, 2019
May 1, 2019
9 months
June 20, 2011
May 2, 2019
Conditions
Outcome Measures
Primary Outcomes (1)
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Safety and tolerability of CK-2017357 after multiple oral doses to steady state in patients with ALS
21 days
Secondary Outcomes (8)
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
21 days
Measurement of Grip Strength and Handgrip Fatigue
21 days
Measurement of muscle strength
21 days
Measurement of Slow Vital Capacity (SVC)
21 days
Measurement of Sniff Nasal Inspiratory Pressure (SNIP)
21 days
- +3 more secondary outcomes
Study Arms (4)
Matching Placebo
PLACEBO COMPARATORActive Drug Low Dose (CK-2017357 125 mg)
EXPERIMENTALActive Drug Mid Dose (CK-2017357 250 mg)
EXPERIMENTALActive Drug High Dose (CK-2017357 375 mg)
EXPERIMENTALInterventions
Placebo tablets once daily for 14 days (Part A)
One 125 mg CK-2017357 tablet once daily for 14 days (Part A)
One 50 mg tablet once daily for 14 days (Part B)
Placebo tablets once daily for 14 days (Part B)
One 125 mg tablet once daily for 14 days (Part B)
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)
- Upright Slow Vital Capacity (SVC) \>50% of predicted for age, height, and sex
- Able to swallow tablets with water
- Willing and able to remain off riluzole for 4 weeks (Part A only)
- Currently taking and tolerating a stable dose of 50 mg BID riluzole (Part B only)
- Willing and able to reduce daily dose of riluzole to 50 mg for 4 weeks (Part B only)
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Cytokineticslead
Study Sites (9)
California Pacific Medical Center
San Francisco, California, 94115, United States
Mayo Florida
Jacksonville, Florida, 32224, United States
University of Kansas
Kansas City, Kansas, 06053, United States
Johns Hopkins Hospital
Baltimore, Maryland, 21287, United States
Columbia University Medical Center
New York, New York, 10032, United States
SUNY Upstate Medical Center
Syracuse, New York, 13210, United States
Carolinas Neuromuscular ALS-MND Center
Charlotte, North Carolina, 28207, United States
Penn State Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
Drexel University College of Medicine
Philadelphia, Pennsylvania, 19107, United States
Related Publications (1)
Shefner JM, Watson ML, Meng L, Wolff AA; Neals/Cytokinetics STUDY Team. A study to evaluate safety and tolerability of repeated doses of tirasemtiv in patients with amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2013 Dec;14(7-8):574-81. doi: 10.3109/21678421.2013.822517. Epub 2013 Aug 19.
PMID: 23952636RESULT
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
June 20, 2011
First Posted
June 22, 2011
Study Start
June 1, 2011
Primary Completion
March 1, 2012
Study Completion
March 1, 2012
Last Updated
May 7, 2019
Record last verified: 2019-05