Clinical trial: Phase 2, Randomized, Double Blind, Placebo Controlled Multicenter Study of Autologous MSC-NTF Cells in Patients With ALS (NCT02017912)

Eligibility criteria:
Inclusion Criteria

          1. Males and females ages 18 to 75 years old, inclusive.

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

          3. Disease onset, as defined by first reported occurrence of symptomatic weakness,
             spasticity, or bulbar symptoms, of more than 12 months and less than or equal to 24
             months.

          4. Current disease symptoms must include limb weakness.

          5. ALSFRS-R ≥30 at the Screening Visit.

          6. Upright slow vital capacity (SVC) measure ≥65% of predicted for gender, height, and
             age at the Screening Visit.

          7. Subjects must be taking a stable dose of riluzole for at least 30 days prior to
             enrolment or not be on riluzole, and not have been on it for at least 30 days prior to
             enrolment (riluzole-naïve subjects are permitted in the study).

          8. Capable of providing informed consent and willing and able to follow study procedures,
             including willingness to undergo lumbar puncture.

          9. Geographic accessibility to the study site and willingness and ability to comply with
             follow-up.

         10. Women of child-bearing potential must agree not to become pregnant for the duration of
             the study. Women must be willing to consistently use two forms of contraceptive
             therapy throughout the course of the trial, and undergo a pregnancy test one week
             before bone marrow aspiration. Men must be willing to consistently use two forms of
             contraceptive if their partners are of child-bearing age.

         11. Citizen or permanent resident of the United States.

        Exclusion Criteria:

          1. Prior stem cell therapy of any kind.

          2. Inability to lie flat for the duration of intrathecal cell transplantation and/or bone
             marrow biopsy, or inability to tolerate study procedures for any other reason.

          3. History of autoimmune disease (excluding thyroid disease) myelodysplastic or
             myeloproliferative disorder, leukemia or lymphoma, whole body irradiation, hip
             fracture, or severe scoliosis.

          4. Any unstable clinically significant medical condition other than ALS (e.g., within six
             months of baseline, had myocardial infarction, angina pectoris, and/or congestive
             heart failure), treatment with anticoagulants that, in the opinion of the
             investigator, would compromise the safety of patients.

          5. Any history of malignancy including any malignancy affecting the central nervous
             system and melanoma, within the previous 5 years, with the exception of localized skin
             cancers (with no evidence of metastasis, significant invasion, or re-occurrence within
             three years of baseline).

          6. Serum AST or ALT value >3.0 times the upper normal limit.

          7. Serum creatinine value >2.0 times the upper normal limit.

          8. Positive test for Hepatitis B, Hepatitis C, HIV.

          9. Current use of immunosuppressant medication or use of such medication within 4 weeks
             of Screening visit (Visit 1).

         10. Any history of acquired or inherited immune deficiency syndrome.

         11. Exposure to any other experimental agent (off-label use or investigational) or
             participation in a clinical trial within 30 days prior to Screening Visit (Visit 1).

         12. Use of non-invasive ventilation (NIV), diaphragm pacing system or invasive ventilation
             (tracheostomy).

         13. Any history of either substance abuse within the past year, or unstable psychiatric
             disease according to PI judgment.

         14. Placement or usage of feeding tube.

         15. Pregnant women or women currently breastfeeding.
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.
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? Entailment