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 Purpose:

The study will generate data on safety, tolerability and pharmacokinetics 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.

Study Status:

Not recruiting

Disease:

Amyotrophic Lateral Sclerosis

Study Type:

Interventional

Type of Intervention:

Drug

Intervention Name:

Placebo (Part A), CK-2017357 (Part A), Riluzole 50 MG (Part B), Placebo (Part B), CK-2017357 (Part B)

Placebo:

Phase:

Phase 2

Study Chair(s)/Principal Investigator(s):

Jeremy Shefner, MD, PhD, State University of New York - Upstate Medical University

Clinicaltrials.gov ID:

NCT01378676

Neals Affiliated?

No

Coordinating Center Contact Information

Cytokinetics

280 East Grand Avenue
California, 94080 United States

Full Study Summary:

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 Sponsor:

Cytokinetics

Participant Duration:

21 days

Estimated Enrollment:

49

Estimated Study Start Date:

05 / 31 / 2011

Estimated Study Completion Date:

03 / 01 / 2012

Posting Last Modified Date:

05 / 07 / 2019

Date Study Added to neals.org:

06 / 22 / 2011

Minimum Age:

18 Years

Maximum Age:

N/A

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.

California Pacific Medical Center

San Francisco, California 94115
United States

Mayo Florida

Jacksonville, Florida 32224
United States

University of Kansas

Kansas City, Kansas 06053
United States

Johns Hopkins Hospital

Baltimore, Maryland 21287
United States

Columbia University Medical Center

New York, New York 10032
United States

SUNY Upstate Medical Center

Syracuse, New York 13210
United States

Carolinas Neuromuscular ALS-MND Center

Charlotte, North Carolina 28207
United States

Penn State Hershey Medical Center

Hershey, Pennsylvania 17033
United States

Drexel University College of Medicine

Philadelphia, Pennsylvania 19107
United States