Dose Titration Study to Test Safety and Effects of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01486849
• Date of registration: 23/11/2011
• Prospective Registration: No
• Primary sponsor: Cytokinetics
• Public title: Dose Titration Study to Test Safety and Effects of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
• Scientific title: 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 (ALS)
• Date of first enrolment: November 2011
• Target sample size: 27
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01486849
• 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

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. Able to swallow tablets with water

6. Currently taking and tolerating a stable dose of 50 mg BID riluzole

7. Willing and able to reduce daily dose of riluzole to 50mg QD for 5 weeks

8. Not currently taking or willing and able to remain off theophylline-containing
medications during study participation

9. Patient has a caregiver who is capable of observing and reporting patient status

10. Upright Slow Vital Capacity (SVC) >50% of predicted for age, height, and sex

11. Able to perform pulmonary function tests

Exclusion Criteria:

1. Life expectancy <3 months

2. Receipt of investigational study drug 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. 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.



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

Drug: Riluzole 50 MG

Drug: Placebo

Primary Outcome(s)

Number of participants with adverse events [Time Frame: approximately 29 days]

Secondary Outcome(s)

Change from baseline in scores on tests of Timed Up and Go [Time Frame: 22 days]

Evaluate the pharmacokinetics of riluzole in patients receiving CK-2017357 [Time Frame: Day 1, Day 15, and Day 22]

Change from baseline in score on the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) [Time Frame: 22 days]

Change from baseline in Investigator Global Assessment [Time Frame: 22 days]

Change from baseline in scores on tests of Sniff Nasal Inspiratory Pressure (SNIP) [Time Frame: 22 days]

Change from baseline in Patient Global Assessment [Time Frame: 22 days]

Change from baseline in scores on tests of Maximum Voluntary Ventilation (MVV) [Time Frame: 21 days]

Change from baseline in scores on tests of maximum handgrip strength and handgrip fatigue [Time Frame: 22 days]

Change from baseline in scores on tests of muscle strength [Time Frame: 22 days]

Change from baseline in scores on tests of Slow Vital Capacity (SVC) [Time Frame: 22 days]

Evaluate the pharmacokinetics of CK-2017357 [Time Frame: Day 1, Day 15, and Day 22]

Secondary ID(s)

CY 4025

Source(s) of Monetary Support

Please refer to primary and secondary sponsors