Clinical trial: A Safety Study of sNN0029 Administration Via Intracerebroventricular Route to Patients With ALS (NCT01999803)

Eligibility criteria:
Inclusion Criteria:

          -  Clinical diagnosis of ALS classified as definite, or probable with or without
             additional laboratory evidence, according to the revised World Federation of Neurology
             (WFN) El Escorial criteria.

          -  If patients are being treated with riluzole, they must have been on a stable dose for
             at least the past 30 days prior to screening.

          -  The patient is, in the opinion of the investigator, medically fit to undergo the
             surgery required for stereotactic implantation of the catheter and infusion pump.

        Exclusion Criteria:

          1. Impaired respiratory function judged to pose a risk to the patient during anaesthesia
             for the device implantation.

          2. Hypertension defined as blood pressure >160 mmHg systolic or >90 mmHg diastolic.

          3. Values for coagulation parameters including platelet count, normalised prothrombin
             complex (PK-INR), activated partial thromboplastin time (APTT) outside normal ranges.

          4. Ophthalmological examination (fundus photography, visual acuity and perimetry) with
             any clinically significant findings that imply safety concerns for this study.

          5. Diagnosis of diabetes mellitus.

          6. History of structural brain disease other than ALS, including tumours and hyperplasia.

          7. An MRI of the brain and cervical spine, and an Magnetic Resonance Angiography (MRA) of
             the brain with findings of tumours or potential sources of pathological bleedings, or
             abnormality that may interfere with the assessments of safety or efficacy or that
             would, in the judgment of the investigator, represent a surgical risk to the patient.
             If an MRI and/or MRA has been performed within 1 month prior to screening, the results
             from that examination can be used.

          8. Any disorder that precludes a surgical procedure (e.g., signs of sepsis or
             inadequately treated infection), alters wound healing (e.g., including bleeding
             disorders), or renders chronic i.c.v. delivery or device implants medically
             unsuitable.

          9. Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that
             cannot be not managed optimally due to:

             i. anatomical factors at or near the implant site (e.g., vascular abnormalities,
             neoplasms, or other abnormalities), ii. underlying disorders of the coagulation
             cascade, platelet function, or platelet count (e.g., haemophilia, Von Willebrand's
             disease, liver disease, or other medical conditions) iii. administration of any
             antiplatelet or anticoagulant medication in the preoperative period

         10. A personal history of thromboembolic disease. A family history of thromboembolic
             disease will prompt a laboratory assessment to exclude hereditary liability before the
             patient is declared eligible.

         11. Presence of additional risk factors for thromboembolism such as obesity (BMI > 35) or
             use of oestrogens including combined contraceptive pills.

         12. Presence of an implanted shunt for the drainage of CSF or an implanted Central Nervous
             System (CNS) catheter.

         13. Clinically significant abnormalities in haematology or clinical chemistry parameters
             as assessed by the investigator.

         14. Serological evidence of Hepatitis B virus (HBV), Hepatitis C virus (HCV) or Human
             immunodeficiency virus (HIV)

         15. Ongoing medical condition that according to the investigator would interfere with the
             conduct and assessments in the study. Examples are medical disability (e.g., severe
             degenerative arthritis, compromised nutritional state, peripheral neuropathy) that
             would interfere with the assessment of safety and efficacy of investigational product
             or device performance, or would compromise the ability of the patient to undergo study
             procedures (e.g., MRI), or to give informed consent.

         16. Participation in another clinical trial with an investigational drug or device within
             3 months prior to screening visit.

         17. For women only: pregnant, breast feeding and/or for fecund women unwillingness to use
             adequate contraception during the trial such as:

               -  Established use of oral, injected or implanted hormonal methods of contraception
                  that do NOT contain oestrogens.

               -  Placement of an intrauterine device.

               -  Barrier methods of contraception: Condom or occlusive cap (diaphragm or
                  cervical/vault caps) with spermicidal foam/gel/film/cream/suppository.
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