Clinical trial: Targeting Metabolic Flexibility in Amyotrophic Lateral Sclerosis (ALS) (NCT04788745)

Eligibility criteria:
Inclusion Criteria:

          -  Age between 18 and 75 years

          -  Signed informed consent prior to the initiation of any study-specific procedures

          -  Familial or sporadic ALS/MND, defined as clinically possible, probable, or definite as
             per the El Escorial criteria

          -  Relative TRICALS risk score between -6.0 to -2.0 (75% of patients with ALS/MND)

          -  Metabolic index ≥110%, at the screening visit.

          -  The use of riluzole will be permitted during the study. Individuals taking riluzole
             must be on a stable dose for at least 30 days prior to the baseline visit, or stopped
             taking riluzole at least 30 days prior to the baseline visit.

          -  Ability to swallow tablets

          -  Able to lie with torso elevated at a 35° angle for 30 minutes without respiratory
             support

          -  Able to give informed consent (as judged by the investigator) and able to comply with
             all study visits and all study procedures

          -  Females must not be able to become pregnant (e.g. post-menopausal, surgically sterile
             or using highly effective birth control methods) for the duration of the study. Highly
             effective methods of birth control are those with a failure rate of < 1% per year when
             employed consistently and correctly, e.g. Combined (oestrogen and progestogen
             containing) hormonal contraception associated with inhibition of ovulation:

               -  oral

               -  intravaginal

               -  transdermal

               -  Progestogen-only hormonal contraception associated with inhibition of ovulation:

               -  oral

               -  injectable

               -  implantable

               -  intrauterine device (IUD)

               -  intrauterine hormone-releasing system ( IUS)

               -  vasectomised partner

          -  Females of child-bearing potential must have a negative serum pregnancy test at
             screening and baseline and be non-lactating

        Exclusion Criteria:

          -  Unable to provide informed consent

          -  History of, or current diagnosis of diabetes or medical condition that impacts whole
             body energy expenditure (e.g. Hashimoto's, heart disease)

          -  Parkinson's disease or parkinsonism, tremor, restless-leg syndrome

          -  Safety Laboratory Criteria at screening related to significant kidney disease:

               -  Creatinine clearance < 50 mL / min (Cockcroft-Gault) based on Cystatin C

          -  Tracheostomy or non-invasive ventilation (NIV) use > 22 hours per day

          -  Inability to swallow tablets

          -  Contraindication therapy:

               -  Allergy for one of the product's active pharmaceutical ingredients (APIs) or
                  excipients.

               -  Antihypertensive treatment [Trimetazidine may cause hypotension]

          -  Evidence of malignant disease

          -  Significant neuromuscular disease other than ALS/MND

          -  Ongoing disease that may cause neuropathy

          -  Pregnancy or breastfeeding

          -  Females actively seeking to become pregnant who are not using an adequate form of
             contraceptive as detailed in the Inclusion criteria.

          -  Deprivation of freedom by administrative or court order
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