A Study to Evaluate the Effects of Multiple Doses of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
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ClinicalTrials.gov Identifier: NCT01378676 |
Recruitment Status :
Completed
First Posted : June 22, 2011
Last Update Posted : May 7, 2019
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Amyotrophic Lateral Sclerosis | Drug: Placebo (Part A) Drug: CK-2017357 (Part A) Drug: Riluzole 50 MG (Part B) Drug: Placebo (Part B) Drug: CK-2017357 (Part B) | Phase 2 |
In Part A, approximately 24 patients will be randomized to one of four different treatment groups. After a 7-day washout of riluzole, patients in each treatment group will receive daily oral doses of placebo, 125 mg, 250 mg, or 375 mg of CK-2017357 for 14 days. Patients will take daily doses of CK-2017357 or placebo (Day 1 through Day 14) and will return to the study site on Day 2, Day 8 and Day 15. All patients will return for a follow-up visit 7 days (± 2 days) after their last dose.
In Part B, approximately additional 24 patients will be randomized to one of four different treatment groups as in Part A. Patients in Part B 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.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 49 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | 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) |
Study Start Date : | June 2011 |
Actual Primary Completion Date : | March 2012 |
Actual Study Completion Date : | March 2012 |

Arm | Intervention/treatment |
---|---|
Placebo Comparator: Matching Placebo |
Drug: Placebo (Part A)
Placebo tablets once daily for 14 days (Part A) Drug: Riluzole 50 MG (Part B) One 50 mg tablet once daily for 14 days (Part B) Drug: Placebo (Part B) Placebo tablets once daily for 14 days (Part B) |
Experimental: Active Drug Low Dose (CK-2017357 125 mg) |
Drug: Placebo (Part A)
Placebo tablets once daily for 14 days (Part A) Drug: CK-2017357 (Part A) One 125 mg CK-2017357 tablet once daily for 14 days (Part A)
Other Name: tirasemtiv Drug: Riluzole 50 MG (Part B) One 50 mg tablet once daily for 14 days (Part B) Drug: Placebo (Part B) Placebo tablets once daily for 14 days (Part B) Drug: CK-2017357 (Part B) One 125 mg tablet once daily for 14 days (Part B)
Other Name: tirasemtiv |
Experimental: Active Drug Mid Dose (CK-2017357 250 mg) |
Drug: Placebo (Part A)
Placebo tablets once daily for 14 days (Part A) Drug: CK-2017357 (Part A) Two 125 mg CK-2017357 tablets once daily for 14 days (Part A)
Other Name: tirasemtiv Drug: Riluzole 50 MG (Part B) One 50 mg tablet once daily for 14 days (Part B) Drug: Placebo (Part B) Placebo tablets once daily for 14 days (Part B) Drug: CK-2017357 (Part B) Two 125 mg tablets once daily for 14 days (Part B)
Other Name: tirasemtiv |
Experimental: Active Drug High Dose (CK-2017357 375 mg) |
Drug: CK-2017357 (Part A)
Three 125 mg CK-2017357 tablets once daily for 14 days (Part A)
Other Name: tirasemtiv Drug: Riluzole 50 MG (Part B) One 50 mg tablet once daily for 14 days (Part B) Drug: CK-2017357 (Part B) Three 125 mg tablets once daily for 14 days (Part B)
Other Name: tirasemtiv |
- Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 21 days ]Safety and tolerability of CK-2017357 after multiple oral doses to steady state in patients with ALS
- Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) [ Time Frame: 21 days ]An instrument for evaluating the functional status of patients with ALS. Minimum score is 0 and maximum score is 40. The higher the score the more function is retained.
- Measurement of Grip Strength and Handgrip Fatigue [ Time Frame: 21 days ]Measured using the DynEx Electronic Hand Dynamometer. Patients asked to squeeze the device with the maximum possible force to establish the maximum voluntary contraction. Handgrip fatigue is then measured. Patient is asked to squeeze the device until they can no longer stay above 60% of target or 120 seconds.
- Measurement of muscle strength [ Time Frame: 21 days ]Muscle strength is measured using Hand Held Dynamometry. A series of assessments are done on different muscle groups.
- Measurement of Slow Vital Capacity (SVC) [ Time Frame: 21 days ]SVC will be measured using the ndd EasyOne Spirometer System at Screening, Day -7, Day 1, Day 2, Day 8, Day 15 and at the Follow-Up Visit
- Measurement of Sniff Nasal Inspiratory Pressure (SNIP) [ Time Frame: 21 days ]SNIP will be measured using the Micro Medical Respiratory Pressure Meter (MicroRPM) at Screening, Day -7, Day 1, Day 2, Day 8, Day 15 and at the Follow-Up Visit
- Measurement of Maximum Voluntary Ventilation (MVV) [ Time Frame: 21 days ]MVV will be measured using the EasyOne Spirometer System at Screening, Day -7, Day 1, Day 2, Day 8, Day 15 and at the Follow-Up Visit
- Patient global assessment [ Time Frame: 15 days ]Patients will be asked to assess whether they feel the same, better or worse as compared to how they felt at pre-dose on Day 1
- Investigator global assessment [ Time Frame: 15 days ]Investigator will assess whether the patient appears the same, better or worse as compared to the patient's status at pre-dose on Day 1.

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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 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
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01378676
United States, California | |
California Pacific Medical Center | |
San Francisco, California, United States, 94115 | |
United States, Florida | |
Mayo Florida | |
Jacksonville, Florida, United States, 32224 | |
United States, Kansas | |
University of Kansas | |
Kansas City, Kansas, United States, 06053 | |
United States, Maryland | |
Johns Hopkins Hospital | |
Baltimore, Maryland, United States, 21287 | |
United States, New York | |
Columbia University Medical Center | |
New York, New York, United States, 10032 | |
SUNY Upstate Medical Center | |
Syracuse, New York, United States, 13210 | |
United States, North Carolina | |
Carolinas Neuromuscular ALS-MND Center | |
Charlotte, North Carolina, United States, 28207 | |
United States, Pennsylvania | |
Penn State Hershey Medical Center | |
Hershey, Pennsylvania, United States, 17033 | |
Drexel University College of Medicine | |
Philadelphia, Pennsylvania, United States, 19107 |
Study Chair: | Jeremy Shefner, MD, PhD | State University of New York - Upstate Medical University |
Responsible Party: | Cytokinetics |
ClinicalTrials.gov Identifier: | NCT01378676 |
Other Study ID Numbers: |
CY 4024 |
First Posted: | June 22, 2011 Key Record Dates |
Last Update Posted: | May 7, 2019 |
Last Verified: | May 2019 |
Motor Neuron Disease Amyotrophic Lateral Sclerosis Sclerosis Pathologic Processes Neurodegenerative Diseases Nervous System Diseases Neuromuscular Diseases Spinal Cord Diseases Central Nervous System Diseases TDP-43 Proteinopathies Proteostasis Deficiencies |
Metabolic Diseases Riluzole Anticonvulsants Excitatory Amino Acid Antagonists Excitatory Amino Acid Agents Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action Physiological Effects of Drugs Neuroprotective Agents Protective Agents |