Clinical trial: Multiple Doses of AT-1501-A201 in Adults With ALS (NCT04322149)

Eligibility criteria:
Inclusion Criteria:

          1. ALS diagnosed as possible, laboratory supported probable, probable, or definite as
             defined by revised El Escorial criteria.

          2. ALSFRS-R Aggregate score of 37 or greater

          3. No more than 24 months from diagnosis.

          4. Able and willing to give informed consent, follow trial procedures, and make multiple
             clinical site visits.

        Exclusion Criteria:

          1. Any other central or peripheral nervous system disease that may interfere with the
             evaluation of ALS or its progression.

          2. Presence of a tracheostomy, or use of permanent assistive ventilation (ventilatory
             support for 23 hours per day or more).

          3. History of malignancy within the previous 5 years, except for localized non-melanoma
             skin cancers.

          4. Abnormal function of the immune system resulting from:

               -  Clinical conditions affecting the immune system (e.g. HIV infection,
                  agammaglobulinemia),

               -  Systemic administration of corticosteroids (PO/IV/IM) at a dose equivalent to 20
                  mg/day of prednisone for more than 14 consecutive days within 90 days prior to
                  screening,

               -  Administration of anti-neoplastic and/or immunomodulating agents (e.g. TNF α
                  antagonists or anti-B cell antibodies) or radiotherapy within 1 year prior to
                  screening.

          5. Recipient of Stem Cell or Gene Therapy.

          6. Positive test for Hepatitis B surface antigen, Hepatitis C antibody, or HIV.

          7. History of deep venous thrombosis or pulmonary embolism.

          8. History of active substance abuse within the past 2 years;

          9. History of stroke, poorly controlled or significant cardiovascular disease, diabetes.
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