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

NCT ID: NCT01378676

Last Updated: 2019-05-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

49 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-06-30

Study Completion Date

2012-03-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Amyotrophic Lateral Sclerosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Matching Placebo

Group Type PLACEBO_COMPARATOR

Placebo (Part A)

Intervention Type DRUG

Placebo tablets once daily for 14 days (Part A)

Riluzole 50 MG (Part B)

Intervention Type DRUG

One 50 mg tablet once daily for 14 days (Part B)

Placebo (Part B)

Intervention Type DRUG

Placebo tablets once daily for 14 days (Part B)

Active Drug Low Dose (CK-2017357 125 mg)

Group Type EXPERIMENTAL

Placebo (Part A)

Intervention Type DRUG

Placebo tablets once daily for 14 days (Part A)

CK-2017357 (Part A)

Intervention Type DRUG

One 125 mg CK-2017357 tablet once daily for 14 days (Part A)

Riluzole 50 MG (Part B)

Intervention Type DRUG

One 50 mg tablet once daily for 14 days (Part B)

Placebo (Part B)

Intervention Type DRUG

Placebo tablets once daily for 14 days (Part B)

CK-2017357 (Part B)

Intervention Type DRUG

One 125 mg tablet once daily for 14 days (Part B)

Active Drug Mid Dose (CK-2017357 250 mg)

Group Type EXPERIMENTAL

Placebo (Part A)

Intervention Type DRUG

Placebo tablets once daily for 14 days (Part A)

CK-2017357 (Part A)

Intervention Type DRUG

Two 125 mg CK-2017357 tablets once daily for 14 days (Part A)

Riluzole 50 MG (Part B)

Intervention Type DRUG

One 50 mg tablet once daily for 14 days (Part B)

Placebo (Part B)

Intervention Type DRUG

Placebo tablets once daily for 14 days (Part B)

CK-2017357 (Part B)

Intervention Type DRUG

Two 125 mg tablets once daily for 14 days (Part B)

Active Drug High Dose (CK-2017357 375 mg)

Group Type EXPERIMENTAL

CK-2017357 (Part A)

Intervention Type DRUG

Three 125 mg CK-2017357 tablets once daily for 14 days (Part A)

Riluzole 50 MG (Part B)

Intervention Type DRUG

One 50 mg tablet once daily for 14 days (Part B)

CK-2017357 (Part B)

Intervention Type DRUG

Three 125 mg tablets once daily for 14 days (Part B)

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Placebo (Part A)

Placebo tablets once daily for 14 days (Part A)

Intervention Type DRUG

CK-2017357 (Part A)

One 125 mg CK-2017357 tablet once daily for 14 days (Part A)

Intervention Type DRUG

CK-2017357 (Part A)

Two 125 mg CK-2017357 tablets once daily for 14 days (Part A)

Intervention Type DRUG

CK-2017357 (Part A)

Three 125 mg CK-2017357 tablets once daily for 14 days (Part A)

Intervention Type DRUG

Riluzole 50 MG (Part B)

One 50 mg tablet once daily for 14 days (Part B)

Intervention Type DRUG

Placebo (Part B)

Placebo tablets once daily for 14 days (Part B)

Intervention Type DRUG

CK-2017357 (Part B)

One 125 mg tablet once daily for 14 days (Part B)

Intervention Type DRUG

CK-2017357 (Part B)

Two 125 mg tablets once daily for 14 days (Part B)

Intervention Type DRUG

CK-2017357 (Part B)

Three 125 mg tablets once daily for 14 days (Part B)

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

tirasemtiv tirasemtiv tirasemtiv tirasemtiv tirasemtiv tirasemtiv

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Cytokinetics

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Jeremy Shefner, MD, PhD

Role: STUDY_CHAIR

State University of New York - Upstate Medical University

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

California Pacific Medical Center

San Francisco, California, United States

Site Status

Mayo Florida

Jacksonville, Florida, United States

Site Status

University of Kansas

Kansas City, Kansas, United States

Site Status

Johns Hopkins Hospital

Baltimore, Maryland, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

SUNY Upstate Medical Center

Syracuse, New York, United States

Site Status

Carolinas Neuromuscular ALS-MND Center

Charlotte, North Carolina, United States

Site Status

Penn State Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

Drexel University College of Medicine

Philadelphia, Pennsylvania, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

References

Explore related publications, articles, or registry entries linked to this study.

Shefner JM, Watson ML, Meng L, Wolff AA; Neals/Cytokinetics STUDY Team. A study to evaluate safety and tolerability of repeated doses of tirasemtiv in patients with amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2013 Dec;14(7-8):574-81. doi: 10.3109/21678421.2013.822517. Epub 2013 Aug 19.

Reference Type RESULT
PMID: 23952636 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

CY 4024

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Determining the Safety of L-serine in ALS
NCT01835782 UNKNOWN PHASE1/PHASE2
HEALEY ALS Platform Trial - Regimen F ABBV-CLS-7262
NCT05740813 COMPLETED PHASE2/PHASE3