Clinical trial: A Study to Evaluate the Effects of Multiple Doses of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS) (NCT01378676)

Eligibility criteria:
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.
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.


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? Entailment