A Study of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01089010
• Date of registration: 16/03/2010
• Prospective Registration: No
• Primary sponsor: Cytokinetics
• Public title: A Study of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
• Scientific title: A Phase II, Double-Blind, Randomized, Placebo-Controlled, Three-Way Crossover, Pharmacokinetic and Pharmacodynamic Study of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
• Date of first enrolment: March 2010
• Target sample size: 67
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01089010
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

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

Key inclusion & exclusion criteria

Inclusion Criteria

For enrollment, patients were required to satisfy all of the following criteria at
baseline:

1. Able to comprehend and willing to sign an Informed Consent Form (ICF)

1. 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) (Brooks, Miller
et al. 2000)

2. Males or females 18 years of age or older

3. Body Mass Index (BMI) of 18.0 to 30.0 kg/m2, inclusive

4. Maximum voluntary grip strength in at least one hand between 10 and 40 pounds
(females) or 10 and 60 pounds (males)

5. Able to swallow capsules with water

6. Upright Slow Vital Capacity (SVC) > 40% of predicted for age, height, and sex [See
Appendix 16.6.1]

7. Able to perform pulmonary function tests

8. Pre-study clinical laboratory findings (including troponin I [TnI] and creatine
phosphokinase [CPK]) within normal range, or, if outside of the normal range, deemed
not clinically significant by the Investigator

9. For female patients only: The patient is post-menopausal (= 1 year) or sterilized, or
if she is of childbearing potential, she is not breastfeeding, her pregnancy test is
negative, she has no intention to become pregnant during the course of the study, and
she is using contraceptive drugs or devices for the duration of the study and for 10
weeks after the end of the study.

For male patients only: Male patients agree for the duration of the study and 10 weeks
after the end of the study to use a condom during sexual intercourse with female partners
who are of reproductive potential and to have female partners use an additional effective
means of contraception (e.g., diaphragm plus spermicide or oral contraceptives) or the male
patient must agree to abstain from sexual intercourse for 10 weeks after the end of the
study.

Exclusion Criteria

Patients satisfying any of the following criteria at baseline were excluded from
enrollment:

1. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 times the upper
limit of normal (ULN)

2. Life expectancy < 3 months

3. Participation in any trial in which receipt of investigational study drug occurred
within 30 days prior to dosing

4. Any prior treatment with CK-2017357

5. In the opinion of the Investigator, the patient is not suitable to participate in the
study



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

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: 500 mg CK-2017357

Drug: Placebo

Drug: 250 mg CK-2017357

Primary Outcome(s)

Slow Vital Capacity (SVC) [Time Frame: 2 days]

Repeated Sub-Maximum Grip Strength Fatigability [Time Frame: 2 days]

Sniff Inspiratory Pressure (SNIP) [Time Frame: 2 days]

Maximum grip strength [Time Frame: 2 days]

Maximum Voluntary Ventilation (MVV) [Time Frame: 2 days]

Maximum Voluntary Muscle Contraction (MVC) [Time Frame: 2 days]

Shoulder extension fatigue [Time Frame: 2 days]

ALSFRS-R [Time Frame: 2 days]

Maximum grip strength fatigability [Time Frame: 2 days]

Secondary Outcome(s)

Characterize the relationship, if any, between the plasma concentration of CK-2017357 and slow vital capacity [Time Frame: 2 days]

Characterize the relationship, if any, between the plasma concentration of CK-2017357 and maximum grip strength fatigability [Time Frame: 2 days]

Characterize the relationship, if any, between the plasma concentration of CK-2017357 and shoulder extension fatigue [Time Frame: 2 days]

Characterize the relationship, if any, between the plasma concentration of CK-2017357 and maximum voluntary muscle contraction [Time Frame: 2 days]

Characterize the relationship, if any, between the plasma concentration of CK-2017357 and ALSFRS-R. [Time Frame: 2 days]

Characterize the relationship, if any, between the plasma concentration of CK-2017357 and maximum grip strength [Time Frame: 2 days]

Number of patients with adverse events [Time Frame: 4 weeks]

Effect of CK-2017357 on investigator determined global functional assessment [Time Frame: 2 days]

Characterize the relationship, if any, between the plasma concentration of CK-2017357 and repeated sub-maximum grip strength fatigability [Time Frame: 2 days]

Characterize the relationship, if any, between the plasma concentration of CK-2017357 and sniff inspiratory pressure [Time Frame: 2 days]

Characterize the relationship, if any, between the plasma concentration of CK-2017357 and maximum voluntary ventilation [Time Frame: 2 days]

Effect of CK-2017357 on patient determined global functional assessment [Time Frame: 2 days]

Secondary ID(s)

CY 4021

Source(s) of Monetary Support

Please refer to primary and secondary sponsors