Immunosuppression in Amyotrophic Lateral Sclerosis (ALS)
Condition: Amyotrophic Lateral Sclerosis (ALS)
MeSH terms: Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis
Intervention: Basiliximab; Methylprednisolone; Prednisone; Tacrolimus; Mycophenolate mofetil
Intervention description: 20 mg, IV (in the vein) on day 1 and 4.; 125 mg, IV (in the vein) on day 1.; 60 mg PO (by mouth) on days 2-7, 40 mg PO days 8-14, 20 mg PO days 15-21, and 10mg PO days 22-28.; 1-5 mg PO, twice a day (BID) days 2-180.; 500 mg PO, BID days 2-7, 500 mg PO each morning and 1000 mg each night, days 8-14, 1000 mg PO BID days 15-180.
Intervention MeSH terms: Mycophenolic Acid; Prednisone; Methylprednisolone; Methylprednisolone Hemisuccinate; Tacrolimus; Basiliximab
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 31.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Immunosuppression; ALS; Neuromuscular Disorders

Summary:

      This is a multicenter, 15-month study evaluating the effect of immunosuppression treatment on
      the rate of change on the ALS Functional Rating Scale (Revised) (ALSFRS-R) score in up to 33
      subjects with Amyotrophic Lateral Sclerosis (ALS).
    

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.