Clinical trial: Safety and Efficacy of TUDCA as add-on Treatment in Patients Affected by ALS (NCT03800524)

Eligibility criteria:
Inclusion Criteria:

          -  Probable laboratory-supported, probable, or definite ALS, as defined by El Escorial
             Revised ALS diagnostic criteria 34

          -  Disease duration ≤ 18 months

          -  No swallowing difficulty (4 at ALSFRS-R swallowing subscore)

          -  Able to perform reproducible pulmonary function tests

          -  Forced vital capacity ≥70% of normal

          -  Stable on riluzole treatment for 3 months in the lead-in period

          -  Able to perform reproducible pulmonary function tests

          -  Signed informed consent

        Exclusion Criteria:

          -  Treatment with edaravone

          -  Other causes of neuromuscular weakness

          -  Presence of other neurodegenerative diseases

          -  Significant cognitive impairment, clinical dementia or psychiatric illness

          -  Severe cardiac or pulmonary disease

          -  Other diseases precluding functional assessments

          -  Other life-threatening diseases

          -  At the time of screening, any use of non-invasive ventilation (e.g. continuous
             positive airway pressure, non-invasive bi-level positive airway pressure or
             non-invasive volume ventilation) for any portion of the day, or mechanical ventilation
             via tracheostomy, or on any form of oxygen supplementation

          -  Gastrointestinal disorder that is likely to impair absorption of study drug from the
             gastrointestinal tract

          -  Has taken any investigational study drug within 30 days or five half-lives of the
             prior agent, whichever is longer, prior to dosing

          -  Any clinically significant laboratory abnormality

          -  Other concurrent investigational medications

          -  Active peptic ulcer

          -  Previous surgery or infections of small intestine

          -  Patients unable to easily swallow the treatment pills at time of enrolment

          -  Occurrence of frequent biliary colic, biliary infections, severe pancreatic
             abnormalities

          -  Subjects who weigh 88 lbs (40 kg) or less at screening

          -  Aspartate aminotransferase or alanine aminotransferase concentrations more than 3
             times the upper limit of normal

          -  Creatinine clearance 50 ml/min or less

          -  Previous exposure to bile acids

          -  Any clinically significant neurological, haematological, autoimmune, endocrine,
             cardiovascular, neoplastic, renal, gastrointestinal, or other disorder that, in the
             Investigator's opinion, could interfere with the subject's participation in the study,
             place the subject at increased risk, or confound interpretation of study results

          -  Consideration by the investigator, for any reason, that the subject is an unsuitable
             candidate to receive TUDCA or that the subject is unable or unlikely to comply with
             the dosing schedule or study evaluations

          -  The patient is sexually active and is not willing to use highly effective
             contraception during the study and up to 90 days after the day of last dose
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 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