Immunosuppression in Amyotrophic Lateral Sclerosis (ALS)

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.