Clinical trial: Immunosuppression in Amyotrophic Lateral Sclerosis (ALS) (NCT01884571)

Eligibility criteria:
Inclusion Criteria for participants with symptom onset within the past 24 months:

          -  Male or female patients 18-65 years of age.

          -  ALS diagnosed as possible, laboratory-supported probable, probable, or definite as
             defined by revised El Escorial Criteria.

          -  Symptom onset ≤ 24 months from screening visit.

          -  A score of ≥38 on the Revised ALS Functional Rating Scale.

          -  Slow vital capacity (SVC) measure >80% of predicted for gender, height and age at
             screening.

          -  Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of
             riluzole for at least 30 days, prior to the screening visit (riluzole-naïve subjects
             are permitted in the study).

          -  Negative tuberculosis (TB) test within 3 months of Screening Visit.

          -  Subjects medically able to undergo lumbar puncture (LP) as determined by the
             investigator (i.e., no bleeding disorder, allergy to local anesthetics, or a skin
             infection at or near the LP site).

          -  Capable of providing informed consent and following study procedures.

          -  Women must not be able to become pregnant (e.g. post menopausal, surgically sterile,
             or using adequate birth control methods) for the duration of the study.

          -  Women of childbearing potential must have a negative pregnancy test at screening and
             be non-lactating.

          -  Geographic accessibility to the study site.

        Inclusion Criteria for participants with symptom onset greater than 24 months before
        screening:

          -  Male or female patients age 18 or older.

          -  ALS diagnosed as possible, laboratory-supported probable, probable, or definite as
             defined by revised El Escorial Criteria.

          -  Symptom onset >24 months from screening visit.

          -  Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of
             riluzole for at least 30 days, prior to the screening visit (riluzole-naïve subjects
             are permitted in the study).

          -  Negative tuberculosis (TB) test within 3 months of Screening Visit.

          -  Subjects medically able to undergo lumbar puncture (LP) as determined by the
             investigator (i.e., no bleeding disorder, allergy to local anesthetics, or a skin
             infection at or near the LP site).

          -  Capable of providing informed consent and following study procedures.

          -  Geographic accessibility to the study site.

          -  Women must not be able to become pregnant (e.g. post menopausal, surgically sterile,
             or using adequate birth control methods) for the duration of the study.

          -  Women of childbearing potential must have a negative pregnancy test at screening and
             be non-lactating.

        Exclusion Criteria

          -  Prior use of basiliximab, solumedrol, prednisone, tacrolimus or mycophenolate mofetil
             within 30 days of the Screening Visit.

          -  Known allergy or sensitivity to basiliximab, solumedrol, prednisone, tacrolimus or
             mycophenolate mofetil or a formulation of one of these drugs.

          -  Treatment with an immunosuppressant medication within 30 days of the Screening Visit.

          -  Active peptic ulcer disease.

          -  Any medical disorder that would make immunosuppression contraindicated including, but
             not limited to, human immunodeficiency virus (HIV), tuberculosis, or evidence of
             active cytomegalovirus (CMV) or infection.

          -  Subjects who have a diaphragm pacing system (DPS).

          -  Women who are pregnant, breastfeeding, or planning to become pregnant in the next 12
             months.

          -  Active drug or alcohol use or dependence that, in the opinion of the site
             investigator, would interfere with adherence to study requirements.

          -  Use of invasive or non-invasive mechanical ventilation (including Continuous Positive
             Airway Pressure (CPAP) or Bi-level Positive Airway Pressure (BiPAP)) for any part of
             the day or night prior to the Screening Visit (participants with symptom onset within
             past 24 months only).

          -  Exposure to any other agent currently under investigation for the treatment of
             patients with ALS (off-label use or investigational) within 30 days of the Screening
             Visit.

          -  Inability to safely complete study activities based on the discretion of the site
             investigator.
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? 1