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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01378676
• Date of registration: 20/06/2011
• Prospective Registration: No
• Primary sponsor: Cytokinetics
• Public title: A Study to Evaluate the Effects of Multiple Doses of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
• Scientific 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)
• Date of first enrolment: June 2011
• Target sample size: 49
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01378676
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Jeremy Shefner, MD, PhD
• Affiliation: State University of New York - Upstate Medical University

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: CK-2017357 (Part A)

Drug: Placebo (Part A)

Drug: Riluzole 50 MG (Part B)

Drug: CK-2017357 (Part B)

Drug: Placebo (Part B)

Primary Outcome(s)

Number of Participants with Adverse Events as a Measure of Safety and Tolerability [Time Frame: 21 days]

Secondary Outcome(s)

Measurement of muscle strength [Time Frame: 21 days]

Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) [Time Frame: 21 days]

Measurement of Grip Strength and Handgrip Fatigue [Time Frame: 21 days]

Patient global assessment [Time Frame: 15 days]

Measurement of Maximum Voluntary Ventilation (MVV) [Time Frame: 21 days]

Measurement of Slow Vital Capacity (SVC) [Time Frame: 21 days]

Investigator global assessment [Time Frame: 15 days]

Measurement of Sniff Nasal Inspiratory Pressure (SNIP) [Time Frame: 21 days]

Secondary ID(s)

CY 4024

Source(s) of Monetary Support

Please refer to primary and secondary sponsors