Clinical trial: A Study to Evaluate Efficacy, Safety and Tolerability of CK-2127107 in Patients With Amyotrophic Lateral Sclerosis (ALS) (NCT03160898)

Eligibility criteria:
Inclusion Criteria:

          -  Diagnosis of familial or sporadic ALS ≤ 24 months prior to screening

          -  Upright Slow Vital Capacity (SVC) ≥ 60% 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
             after receiving the first dose of study drug until 10 weeks after the last dose 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 or abstain from heterosexual intercourse
             from Screening until 10 weeks after last dose of study drug

          -  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.

          -  Patients on edaravone must have completed at least 2 cycles of dosing with edaravone
             at the time of screening or have not taken edaravone for at least 30 days prior to
             screening and not planning to start edaravone 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
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 18 Years old.
Subject must be at most 80 Years

Patient description (medical language): A 47-year-old man comes to the clinic for the follow up of his neuromuscular disease. He experienced gradual, progressive weakness of the left upper extremity over the last year.  Over the last few months, he has also noticed weakness in the right upper extremity.  BP is 120/75, PR is 80 and temperature is 37 C. Reflexes are brisk in the upper extremities, and the plantar responses are extensor.  Mild gait ataxia is present. The patient is under treatment of Riluzole 50 mg BID with the diagnosis of ALS.

Patient description (patient's own words): I've been suffering from a neuromuscular disease for a while now, and I went to my doctor's office. I'm now a 47-year-old man and over the past year I experienced a progressive and gradual weakness of my left upper extremity, and over the past month, I also noticed a weakness over my right upper extremity. My heart rate was 120/75, and my  PR was 80 with 37°C for temperature. My reflexes are not good in my upper extremities, and I have trouble with my balance. I'm also under Exservan 50 mg for my sclerosis.

Does the patient match the trial criteria? 1