A Phase 2, Multi-Center, Double-Blind, Randomized, Dose-Ranging, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of CK-2127107 in Patients With ALS

Study Purpose:

The purpose of this study is to assess the effect of CK-2127107 versus placebo on respiratory function and other measures of skeletal muscle function in patients with ALS.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Active, currently recruiting

Phase:

Phase II

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

Clinicaltrials.gov ID (11 digit #):

NCT03160898

Neals Affiliated?

No

Coordinating Center Contact Information


MD Cytokinetics / .(JavaScript must be enabled to view this email address) / 650-624-2929
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Enrolled participants will be dosed with CK-2127107 150, 300, 450 mg or placebo twice daily (300, 600, 900 mg/day or placebo) for a period of 12 weeks.

Study Sponsor:

Cytokinetics

Participant Duration:

12 weeks

Estimated Enrollment:

445

Estimated Study Start Date:

07/24/2017

Estimated Study Completion Date:

07/01/2018

Posting Last Modified Date:

09/01/2017

Date Study Added to alsconsortium.org:

09/01/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    80

    Min Vital Capacity (% predicted normal):

    65

    Time since Symptom Onset:

    Time since Diagnosis:

    <24 months

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:
    Diagnosis of familial or sporadic ALS ≤ 24 months prior to screening
    Upright Slow Vital Capacity (SVC) ≥ 65% of predicted for age, height and sex at screening
    Able to swallow tablets
    A caregiver (if one is needed)
    Able to perform reproducible pulmonary function tests
    Pre-study clinical laboratory findings within the normal range or, if outside the normal range, deemed not clinically significant by the Investigator
    Male patients who have not had a vasectomy and confirmed zero sperm count must agree to either use acceptable methods of contraception or abstain from sex
    Female patients must be post-menopausal or sterilized or must not be breastfeeding, have a negative pregnancy test, have no intention to become pregnant during the study and use acceptable methods of contraception
    Patients must be either on riluzole for at least 30 days prior to screening or have not taken riluzole for at least 30 days prior to screening and not planning to start riluzole during the course of the study.

    Exclusion Criteria:
    At the time of screening, any use of non-invasive ventilation (NIV), e.g. continuous positive airway pressure [CPAP], noninvasive bi-level positive airway pressure [NPPV] or noninvasive volume ventilation [NVV]for any portion of the day, or mechanical ventilation via tracheostomy, or on any form of oxygen supplementation
    Neurological impairment due to a condition other than ALS
    Presence at screening of any medically significant cardiac, pulmonary, GI, musculoskeletal, or psychiatric illness that might interfere with the patient's ability to comply with study procedures or that might confound the interpretation of clinical safety or efficacy data
    Has taken any investigational study drug within 30 days or five half-lives of the prior agent, whichever is longer, prior to dosing
    Known to have received CK-2127107 or tirasemtiv in any previous clinical trial
    Has received or is considering receiving during the course of the study any form of stem cell therapy for the treatment of ALS
    Has received or is considering receiving during the course of the study any form of gene therapy for the treatment of ALS
    Has received or is considering obtaining during the course of the study a diaphragmatic pacing system
    History of substance abuse within the past 2 years
    Use of certain medications

  • Site Contact Information

    Texas Neurology
    Dallas, Texas 75214
    United States