A Study to Evaluate the Effects of Multiple Doses of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Cytokinetics
ClinicalTrials.gov Identifier:
NCT01378676
First received: June 20, 2011
Last updated: September 16, 2013
Last verified: March 2012

June 20, 2011
September 16, 2013
June 2011
March 2012   (final data collection date for primary outcome measure)
Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 21 days ] [ Designated as safety issue: Yes ]
Safety and tolerability of CK-2017357 after multiple oral doses to steady state in patients with ALS
Same as current
Complete list of historical versions of study NCT01378676 on ClinicalTrials.gov Archive Site
  • ALSFRS-R [ Time Frame: 21 days ] [ Designated as safety issue: No ]
  • Measurement of muscle fatigue [ Time Frame: 21 days ] [ Designated as safety issue: No ]
  • Measurement of pulmonary function [ Time Frame: 21 days ] [ Designated as safety issue: No ]
  • Patient global assessment [ Time Frame: 21 days ] [ Designated as safety issue: No ]
  • Investigator global assessment [ Time Frame: 21 days ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
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)

The study will generate data on safety and tolerability 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.

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 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.

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Amyotrophic Lateral Sclerosis
  • Drug: Placebo
    Three matching placebo tablets once daily for 14 days (Part A) or three matching placebo tablets once daily for 14 days taken concurrently with 50 mg riluzole (Part B)
  • Drug: CK-2017357
    One 125 mg CK-2017357 tablet and two matching placebo tablets once daily for 14 days (Part A) or one 125 mg CK-2017357 tablet and two matching placebo tablets once daily for 14 days taken concurrently with 50 mg riluzole (Part B)
    Other Name: tirasemtiv
  • Drug: CK-2017357
    Two 125 mg CK-2017357 tablets and one matching placebo tablet once daily for 14 days (Part A) or two 125 mg CK-2017357 tablets and one matching placebo tablet once daily for 14 days taken concurrently with 50 mg riluzole (Part B)
  • Drug: CK-2017357
    Three 125 mg CK-2017357 tablets once daily for 14 days (Part A) or three 125 mg CK-2017357 tablets once daily for 14 days taken concurrently with 50 mg riluzole (Part B)
  • Placebo Comparator: Matching Placebo
    Intervention: Drug: Placebo
  • Experimental: Active Drug Low Dose
    Intervention: Drug: CK-2017357
  • Experimental: Active Drug Mid Dose
    Intervention: Drug: CK-2017357
  • Experimental: Active Drug High Dose
    Intervention: Drug: CK-2017357
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
50
March 2012
March 2012   (final data collection date for primary outcome measure)

Key Inclusion Criteria:

  1. Able to comprehend and willing to sign an Informed Consent Form (ICF)
  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. Upright Slow Vital Capacity (SVC) >50% of predicted for age, height, and sex
  6. Able to swallow tablets with water
  7. Willing and able to remain off riluzole for 4 weeks (Part A only)
  8. Currently taking and tolerating a stable dose of 50 mg BID riluzole (Part B only)
  9. Willing and able to reduce daily dose of riluzole to 50 mg for 4 weeks (Part B only)
  10. Willing and able to refrain from caffeine-containing products during study participation
  11. Willing and able to remain off warfarin and theophylline-containing medications during study participation
  12. Has a caregiver who is capable of observing and reporting patient status, and also assisting in the proper use of nocturnal oximetry equipment
  13. Able to perform pulmonary function tests

Key Exclusion Criteria:

  1. Life expectancy <3 months
  2. 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
  3. Any prior treatment with CK-2017357
  4. Use of non-invasive positive pressure ventilation (NIPPV) for any part of the day or night

Other protocol-defined inclusion/exclusion criteria may apply.

Both
18 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01378676
CY 4024
No
Cytokinetics
Cytokinetics
Not Provided
Study Chair: Jeremy Shefner, MD, PhD State University of New York - Upstate Medical University
Cytokinetics
March 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP