Clinical trial: Effect of MD1003 in Amyotrophic Lateral Sclerosis (NCT03114215)

Eligibility criteria:
Inclusion Criteria:

          -  Age: 25 to 80 years, inclusive

          -  Male or female subjects with probable or confirmed ALS (revised international El
             Escorial criteria, Forbes et al., 2001).

          -  Patients presenting first motor deficits due to ALS for a maximum of three years at
             the first consultation in an ALS centre.

          -  Patients monitored for at least 6 months in an ALS centre or for whom the previous
             monitoring parameters are available (excepted for MIP and SNIP).

          -  Patients who have lost at least 5 points on the ALSFRS-R (ALS functional rating scale)
             during the last 12 months or at least 2 points during the preceding 6 months

          -  Patients who have been treated with riluzole for at least 3 months at a stable dose.
             In case of intolerance to this product or refusal for this treatment, patients who
             have not been treated with riluzole for at least 1 month before inclusion

          -  For patients with spinal form (onset of the disease affecting limbs) or respiratory
             form, slow vital capacity > 60% of predicted value.

          -  For patients with a bulbar form, slow vital capacity > 60% of theoretical value or, if
             spirometry not assessable (severe bulbar disability), patient should not have
             significant abnormality in both nocturnal capnography and nocturnal oximetry (median
             pCO2 (carbon dioxide partial pressure ) < 52 mmHg, SaO2 (arterial oxygen saturation )
             < 90% less than 5% of the time during night) less than 3 months prior inclusion.

          -  Patients who are willing to give written consent (or oral consent in the presence of a
             trusted person if the patient is no longer able to write)

          -  Patients likely to be able to participate in all scheduled evaluation and complete all
             required study procedures (except for spirometry in bulbar patients with severe
             disability).

        Exclusion Criteria:

          -  Patients on non-invasive ventilation for respiratory insufficiency due to ALS for more
             than 10 hours a day

          -  Patients with an ALSFRS-R score at inclusion of < 20 (maximum score without disability
             = 48)

          -  Patients who have lost less than 5 points on the ALSFRS-R during the last year or less
             than 2 points during the preceding 6 months

          -  Patients with a gastrostomy

          -  Patients who have lost more than 15% of their reference weight (defined as weight
             before disease onset)

          -  Patient with dyspnoea at rest or with the least effort (score < 3 on the dyspnoea item
             of the ALSFRS-R)

          -  Patients with dementia

          -  Patient with severe or rapidly progressive form of ALS for whom the investigator
             estimates the life expectancy less than 3 months

          -  Patients with another progressive disease that has not been stabilized at the time of
             inclusion

          -  Patients with cancer, except basal cell carcinoma, for less than 5 years, or who
             require continuous treatment for cancer even if it is older

          -  Pregnant women.

          -  Subject who are not covered by a social security scheme.

          -  Subject under temporary or permanent Judicial Protection.

          -  Contraception: Both male subjects, and female subjects who are not either surgically
             sterile (tubal ligation/obstruction or removal of ovaries or uterus) or
             post-menopausal (no spontaneous menstrual periods for at least one year confirmed by a
             negative hormone panel), must commit to using two highly effective method of birth
             control for the duration of the study and for two months after the treatment
             termination.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 25 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? Entailment