Clinical trial: Single-Ascending-Dose Safety/Tolerability of NP001 in Amyotrophic Lateral Sclerosis (ALS) (NCT01091142)

Eligibility criteria:
Inclusion Criteria:

          -  Males and females, age 21 years - 75 years

          -  Subjects must be in generally sound health as appropriate for their ALS diagnosis.

          -  Subjects must have a clinical diagnosis of laboratory-supported probable or definite
             ALS, according to modified El Escorial criteria.

          -  Subjects receiving riluzole must be on a stable dose for at least 30 days prior to
             enrollment.

          -  Women of childbearing age must be non-lactating and surgically sterile or using an
             effective method of birth control and have a negative pregnancy test prior to dosing
             with study medication.

          -  Subjects must understand the study and be willing to adhere to protocol requirements
             as evidenced by provision of written informed consent.

          -  Subject must be willing and able to give signed informed consent that has been
             approved by the Institutional Review Board (IRB).

          -  Subjects must be willing to have an intravenous infusion.

          -  Subjects must have suitable veins for IV access as determined by examination.

        Exclusion Criteria:

          -  Subjects should not require nor are expected to require life sustaining interventions
             for the next six months or longer. (e.g. invasive ventilation).

          -  Subjects must not have:

               -  presence of a tracheotomy or invasive ventilation. Nocturnal non-invasive
                  ventilation system (e.g. C-PAP) is allowed.

               -  a diagnosis of neurologic disease known to mimic the muscle atrophy or weakness
                  seen in ALS including MS, muscular dystrophy, spinal stenosis, peripheral
                  neuropathy, inherited neuropathies or neuromuscular diseases, foramen magnum or
                  brainstem tumor, or toxic conditions.

               -  an active pulmonary disease under treatment including uncontrolled asthma,
                  chronic obstructive pulmonary disease (pneumonia, bronchitis, etc.), pulmonary
                  fibrosis, pulmonary infection in the last 2 months, or history of aspiration that
                  may expose the subject to increased risk by participating in this trial as
                  determined by the Investigator.

               -  a history of unstable medical illness in the 3 months prior to screening
                  including any emergent hospitalizations.

               -  renal disease based on screening estimated creatinine clearance (eCcr) < 50
                  mL/minute (Cockcroft Gault estimate using ideal body weight) where:

               -  evidence of elevated alanine aminotransferase greater than 3 times the upper
                  limit of normal.

               -  evidence of anemia, thrombocytopenia, or neutropenia (screening hematocrit <33%,
                  platelet count < lower limit of normal for the site laboratory, or neutrophil
                  count less than 1,500/mm3).

               -  any condition that requires periodic red blood cell transfusions, erythropoietin
                  or any blood dyscrasias undergoing active treatment in the past year.

               -  clinical laboratory parameters that are clinically significant in the opinion of
                  the Investigator.

               -  systolic blood pressure in excess of 160 mmHg nor less than 100 mmHG or a
                  diastolic blood pressure above 98 mmHg.

               -  a history of G6PD deficiency (Glucose-6-phosphate dehydrogenase deficiency)
                  determined by subject report.

               -  a current history of hepatitis or HIV determined by subject report.

          -  Subjects must not be using systemic immunosuppressants including steroids and
             chemotherapeutic agents. Inhaled steroids, eye drops and local topical use are
             permitted with concurrence of Medical Monitor.

          -  Subjects must not have a hematologic disorder such as autoimmune anemia, or hemolytic
             anemia of any type including paroxysmal nocturnal hemoglobinuria or myoglobinuria.

          -  Subjects must not have a history of unexplained jaundice determined by subject report.

          -  Subjects must not have received IV Immunoglobulin (IG) within 30 days of the planned
             initial dose of study drug.

          -  Subjects must not be participating in another drug study or have participated in a
             drug study within the last 30 days prior to enrollment. Observational trials with no
             intervention are acceptable provided permission for the other study Sponsor is
             obtained in writing.

          -  Subjects must not have any other condition which in the Investigator's opinion would
             put the subject at risk by participating in this study.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 21 Years old.
Subject must be at most 75 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