Clinical trial: Therapeutic Nanocatalysis to Slow Disease Progression of Amyotrophic Lateral Sclerosis (ALS) (NCT04098406)

Eligibility criteria:
Inclusion Criteria:

          1. Able to understand and give written informed consent.

          2. Male or female patients aged 30 years or greater (inclusive) and less than 80 years of
             age at the time of Screening.

          3. Patients whose conditions are defined as possible or probable or definite ALS per the
             diagnostic criteria by Awaji-Shima criteria as determined by a neurologist sub-
             specialising in ALS (e.g., the Principal Investigator by study site).

          4. For patients taking riluzole, stable dosing of riluzole over the prior 30-days from
             Screening.

          5. At the time of Screening either disease duration less than or equal to 24-months from
             symptom onset, or disease duration less than or equal to 12-months from diagnosis.

          6. Forced vital capacity (FVC) 60% of predicted value as adjusted for gender, height, and
             age at the Screening Visit.

          7. Patient who has established care with a neurologist at one of the specialized ALS
             clinics involved in the study and will maintain this clinical care throughout the
             study. If a patient is referred from a third party (neurologist or a State based ALS
             organization) they must be willing to transfer care to the neurologist participating
             in the study.

        Following completion of the 36-week randomized placebo controlled treatment period,
        interested participants must meet the following inclusion criteria to enroll in the
        open-label extension:

          1. Participants must have completed the randomized placebo controlled Treatment Period
             without compliance issues

          2. Able to understand and give written informed consent to participant in the open-label
             extension.

          3. If referred from a third party (neurologist or a State based ALS organization),
             participant agrees to maintain transfer of care to a neurologist participating in the
             study.

        Exclusion Criteria:

          1. Patients will be excluded from the study if they meet any of the following criteria:

          2. At Screening patients who utilize, or in the Investigator's judgment will be
             imminently dependent upon:

               1. Non-invasive ventilation > 22 hours per day, or

               2. Tracheostomy Note: If the patient requires non-invasive ventilation
                  postrandomisation, they will be allowed to continue in the study.

          3. Patients with Familial ALS (e.g., 2 or more family members with ALS or motor neuron
             disease)

          4. Patients with a history of carpal tunnel syndrome, polyneuropathy, or in the
             investigators judgement diseases that could induce polyneuropathy and interfere with
             electromyography (EMG) recordings.

          5. Patients with too severe atrophy of the Abductor Digiti Minimi (ADM), Abductor
             Pollicis Brevis (APB), Biceps Brachii (BB), or Tibialis Anterior (TA) muscles in the
             least clinically affected hand and leg, respectively, to allow for reliable EMG
             recordings.

          6. Patient with a history of significant other major medical conditions based on the
             Investigator's judgment.

          7. Based on the investigator's judgment, patients who may have difficulty complying with
             the protocol and/or any study procedures.

          8. Patient with clinically significant abnormalities in haematology, blood chemistry,
             ECG, or physical examination not resolved by the Baseline visit which according to
             Investigator can interfere with study participation.

          9. Patients with clinically significant hepatic or renal dysfunction or clinical
             laboratory findings that would limit the interpretability of change in liver or kidney
             function, or those with low platelet counts (< 150 x 109 per liter) or eosinophilia
             (absolute eosinophil count of ≥ 500 eosinophils per microliter) at Screening.

         10. Patient participating in any other investigational drug trial or using investigational
             drug (within 12 weeks prior to screening and thereafter).

         11. Females who are pregnant or nursing or who plan to get pregnant during the course of
             this clinical trial or within 6 months of the end of this trial.

         12. Females of child-bearing potential, or men, who are unwilling or unable to use
             accepted methods of birth control.

         13. Active inflammatory condition or autoimmune disorder.

         14. Positive screen for drugs of abuse.

         15. History of gold allergy.

         16. Patient is considered a suicide risk in the opinion of the Investigator, has
             previously made a suicide attempt, or is currently demonstrating active suicidal
             ideation. Subjects with intermittent passive suicidal ideation are not necessarily
             excluded based on the assessment of the Investigator.

        Following completion of the 36-week randomized placebo controlled treatment period,
        interested participants will be excluded from participating in the open-label extension
        phase if they meet any of the following criteria:

          1. Lack of treatment compliance during the randomized placebo controlled Treatment
             Period.

          2. Positive pregnancy test at the Week 36 visit, or, females who plan to get pregnant
             during the course of this extension or within 6 months of the end of this extension.

          3. Based on the investigator's judgment, patients who may have difficulty complying with
             the protocol and/or any study procedures.

          4. Patient with clinically significant abnormalities in hematology, blood chemistry, ECG,
             or physical examination identified during the W36 visit which according to
             Investigator may interfere with continued participation.

          5. Patients with clinically significant hepatic or renal dysfunction or clinical
             laboratory.

             findings that would limit the interpretability of change in liver or kidney function,
             or those with low platelet counts (< 150 x 109 per liter) or eosinophilia (absolute
             eosinophil count of ≥ 500 eosinophils per microliter) at the Week 36 visit.

          6. Patient is considered a suicide risk in the opinion of the Investigator, has
             previously made a suicide attempt, or is currently demonstrating active suicidal
             ideation. Subjects with intermittent passive suicidal ideation are not necessarily
             excluded based on the assessment of the Investigator.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 30 Years old.
Subject must be at most 80 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? 1