Clinical trial: AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) (NCT03127514)

Eligibility criteria:
Key Inclusion Criteria:

          1. Male or female, aged 18-80 years of age

          2. Sporadic or familial ALS diagnosed as definite as defined by the World Federation of
             Neurology revised El Escorial criteria

          3. Less than or equal to 18 months since ALS symptom onset

          4. Capable of providing informed consent and following trial procedures

          5. Slow Vital Capacity (SVC) >60% of predicted value for gender, height, and age at the
             Screening Visit

          6. Subjects must either not take riluzole or be on a stable dose of riluzole for at least
             30 days prior to the Screening Visit. Riluzole-naïve subjects are permitted in the
             study.

          7. Women of child bearing potential (e.g. not post-menopausal for at least one year or
             surgically sterile) must agree to use adequate birth control for the duration of the
             study and 3 months after last dose of study drug. Women must not be planning to become
             pregnant for the duration of the study and 3 months after last dose of study drug

          8. Men must agree to practice contraception for the duration of the study and 3 months
             after last dose of study drug. Men must not plan to father a child or provide for
             sperm donation for the duration of the study and 3 months after last dose of study
             drug

        Key Exclusion Criteria:

          1. Presence of tracheostomy

          2. Exposure to PB, Taurursodiol or UDCA within 3 months prior to the Screening Visit or
             planning to use these medications during the course of the study

          3. History of known allergy to PB or bile salts

          4. Abnormal liver function defined as AST and/or ALT > 3 times the upper limit of the
             normal

          5. Renal insufficiency as defined by a serum creatinine > 1.5 times the upper limit of
             normal

          6. Poorly controlled arterial hypertension (SBP>160mmHg or DBP>100mmHg) at the Screening
             Visit

          7. Pregnant women or women currently breastfeeding

          8. History of cholecystectomy

          9. Biliary disease which impedes biliary flow including active cholecystitis, primary
             biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, gallbladder polyps,
             gangrene of the gallbladder, abscess of the gallbladder.

         10. History of Class III/IV heart failure (per New York Heart Association - NYHA)

         11. Severe pancreatic or intestinal disorders that may alter the enterohepatic circulation
             and absorption of TUDCA including biliary infections, pancreatitis and ileal resection

         12. The presence of unstable psychiatric disease, cognitive impairment, dementia or
             substance abuse that would impair ability of the subject to provide informed consent,
             according to Site Investigator judgment

         13. Clinically significant unstable medical condition (other than ALS) that would pose a
             risk to the subject if they were to participate in the study

         14. Active participation in an ALS clinical trial evaluating a small molecule within 30
             days of the Screening Visit

         15. Exposure at any time to any biologic under investigation for the treatment of subjects
             with ALS (off-label use or investigational) including cell therapies, gene therapies,
             and monoclonal antibodies.

         16. Implantation of Diaphragm Pacing System (DPS)
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? 1