Clinical trial: Open-label Study to Evaluate Safety, Tolerability and PK of BHV-0223 in ALS (NCT03520517)

Eligibility criteria:
Inclusion Criteria:

          1. Subjects with diagnosed ALS by the revised El Escorial diagnostic criteria, including
             laboratory supported probable, probable, or definite ALS;

          2. Subjects who have never taken riluzole tablets, OR Subjects who previously took
             riluzole tablets but discontinued at least 1 month prior to the screening visit.
             Subjects must not have had ALT or AST > 5 x ULN while taking riluzole tablets, or any
             other clinically significant tolerability issues (e.g., hypersensitivity reactions) in
             the judgement of the investigator;

          3. Subjects determined by the investigator to be medically stable;

          4. Subjects determined by the investigator to be willing and physically able to complete
             the study as designed, with or without caregiver assistance.

        Exclusion Criteria:

          1. Target Disease Exceptions

          2. Medical History Exceptions

               1. Subject is known to have a current diagnosis of acute or chronic viral hepatitis;

               2. Subject is known to have any other acute or chronic liver disease that is
                  clinically significant in the investigator's judgment;

               3. Subject has a history of a clinically significant medical condition that would
                  interfere with the subject's ability to comply with study instructions, would
                  place the subject at increased risk, or might confound the interpretation of the
                  study results in the investigator's judgment;

               4. Any other sound medical, psychiatric and/or social reason in the investigator's
                  judgment;

          3. Physical and Laboratory Test Findings

               1. Positive urine pregnancy test in WOCBP at screening;

               2. Subject has evidence of organ dysfunction or any clinically significant deviation
                  from normal in physical examination, vital signs, or other determinations beyond
                  what is consistent with the target population, in the investigator's judgment;

               3. Subject has liver function testing abnormalities (ALT, AST , or total bilirubin)
                  that are > 1 x ULN;

          4. Other Exclusions a. Subjects who are unable to be compliant with the visit schedule or
             protocol procedures.
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.


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