Clinical trial: GM604 Phase 2A Randomized Double-blind Placebo Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS) (NCT01854294)

Eligibility criteria:
Inclusion Criteria:

          1. Patients with ALS: Familial and Sporadic ALS, with symptom onset < or equal to 24
             months.

          2. At least 18 years of age

          3. Subjects meet the El Escorial criteria of definite criteria for a diagnosis of ALS.

          4. Subjects can be on a stable dose of riluzole for at least a month or not taking or
             initiating riluzole for the duration of the trial.

          5. Not on any experimental medication for the last 1 month or five times the half-life of
             experimental medication.

          6. At screening, must have a Forced Vital Capacity (FVC) ≥ 65% of predicted capacity for
             age, height and gender.

          7. Have fully completed informed consent form

          8. Ability to comply with study procedures

          9. Women of child-bearing age must be on birth control. Pregnancy test should be done in
             women in child bearing age.

         10. Medically safe to have lumbar puncture to collect CSF

        Exclusion Criteria:

          1. History of liver disease, severe renal failure, diabetes, coronary heart disease,
             cancer

          2. Clinically significant EKG abnormality at screening

          3. Any comorbid condition which would make completion of the trial unlikely

          4. FVC < 65%

          5. Presence of a bleeding disorder

          6. Allergy to local anesthetics

          7. Problem with CSF pressure

          8. Topical or other skin infection at the lumbar puncture site

          9. BMI > 32 kg/m2

         10. Medical or surgical conditions in which a lumbar puncture is contraindicated

         11. Use of any anti-platelet or anticoagulant drugs, such as plavix, aggrenox, ticlid,
             warfarin or coumadin

             -
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