Clinical trial: Study to Investigate the Safety of the Transplantation (by Injection) of Human Glial Restricted Progenitor Cells (hGRPs; Q-Cells®) Into Subjects With Amyotrophic Lateral Sclerosis (ALS) (NCT02478450)

Eligibility criteria:
Inclusion Criteria:

          1. Subject has the ability to understand the purpose and risks of the study and provide a
             signed and dated informed consent and authorization to collect and use protected
             health information (PHI) in accordance with national and local subject privacy
             regulations.

          2. Subject lives within reasonable driving distance of study center (approximately 3
             hours).

          3. Subject has a caregiver willing/able to assist in the transportation and care required
             by study participation.

          4. Subject is 18 - 80 years of age (inclusive) on the first day of the Screening Period.

          5. Subject is diagnosed with sporadic or familial ALS within the past 48 months.

          6. Subject meets the laboratory-supported probable, clinically probable, or definite
             criteria for diagnosing ALS according to the World Federation of Neurology El Escorial
             criteria.

          7. Subject has an upright FVC ≥65% of predicted value for age, height, and gender at
             Screening.

          8. Subject has not taken riluzole for at least 30 days prior to the first day of the
             Screening Period, or has been on a stable dose of riluzole for at least 30 days prior
             to the first day of the Screening Period (Riluzole-naïve subjects are permitted in the
             study).

          9. Subject is medically able to undergo the study procedures and physically able to
             adhere to the visit schedule at the time of study entry.

         10. Women of childbearing capacity must have a negative pregnancy test during the
             Screening Period and at the Pre-Operative Visit.

         11. Subject must agree to practice effective birth control during study participation.

        Exclusion Criteria:

          1. Subject in whom causes of neuromuscular weakness other than ALS have not been
             practically excluded.

          2. Subject with a diagnosis of significant cognitive impairment, clinical dementia, or
             major psychiatric illness including psychosis, bipolar disease, major depression, as
             determined by the DSM-V.

          3. Subject with a diagnosis of other neurodegenerative disease (e.g., Parkinson's
             disease, Alzheimer's disease).

          4. Subject with a diagnosis of any medical condition that impairs nerve or muscle
             function (e.g., notable peripheral neuropathy, metabolic muscle disease).

          5. Subject with a clinically significant history of unstable cardiac, pulmonary, renal,
             hepatic, endocrine, hematologic, or active malignancy or infectious disease or other
             medically significant illness, which, in the opinion of the Investigator, would
             preclude study participation.

          6. Subject with a history of spine surgery or anatomic variation incompatible with route
             of administration (as determined by neurosurgeon).

          7. Subject with severe cervical or lumbar stenosis, cord compression, or cervical or
             lumbar myelopathy.

          8. Subject with abnormal flow voids on the surface of the spinal cord suggestive of
             arteriovenous malformation (AVM).

          9. Subject demonstrating any evidence of CNS malignancy or CNS lesions as defined by
             imaging studies of the CNS (MRI of brain and spinal cord).

         10. Subject having uncontrolled hypertension (Systolic BP>180mmHg and/or Diastolic BP
             >110mmHg) or having a history of thrombotic events or poorly controlled medical
             conditions that, in the opinion of the Investigator and/or surgeon, increase risk of
             surgery.

         11. Subject who cannot undergo MRI examination because of the presence of a pacemaker, an
             implanted defibrillator or certain other implanted electronic or metallic devices, or
             who have been or might have been exposed to metal fragments, or any reason the subject
             cannot undergo an MRI routinely for the duration of the trial.

         12. Subject with clinically significant abnormal clinical laboratory values, as determined
             by the Investigator during the Screening Period.

         13. Subject who is immune compromised or who has a condition contraindicated to treatment
             with immunosuppression agents (e.g., tuberculosis, latent infection).

         14. Subject with an aspartate aminotransferase (AST) or alanine aminotransferase (ALT)
             value >3.0 times the upper limit of normal or creatinine >1.5 times the upper limit of
             normal and/or eGFR <50cc/min during the Screening Period.

         15. Subject with a history of alcohol or drug abuse or dependence within 1 year of the
             first day of the Screening Period, per DSM-V criteria.

         16. Subject unlikely to comply with study requirements, as determined by Investigator.

         17. Subject who has been exposed to any other experimental agent (off-label use or
             investigational) within 30 days of the first day of the Screening Period. Biologic
             agents may need additional time for washout and will be evaluated by the Sponsor on a
             case-by-case basis.

         18. Subject who has previously been administered stem cells.

         19. Subject with pre-existing anti-human leukocyte antigen (HLA) class I or class II
             antibodies directed against the Q-Cells®, as determined by panel reactive antibody
             (PRA) assay during the Screening Period.

         20. Subject with an allergy to Q-Cells® or any of its constituents (e.g., chicken eggs),
             or an allergy to any of the co-administered immunosuppressants or any of their
             excipients.

         21. Subject with any medical condition or using concomitant medication that would
             contraindicate the use of tacrolimus, mycophenolate mofetil, or prednisone as
             determined by Investigator.

         22. Subject with evidence of deep vein thrombosis (DVT) by venous ultrasound or any
             previous evidence of DVT.

         23. Subject who, in the opinion of the Investigator, has taken or is taking concomitant
             medications, supplements, or other agents that may interfere with the safety
             evaluation of Q-Cells® or may affect the course of the subject's ALS progression.
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? Entailment