Clinical trial: A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis (NCT01906658)

Eligibility criteria:
Inclusion Criteria:

          -  Able to provide informed consent.

          -  Diagnosis of clinically definite ALS, clinically probable-laboratory supported ALS,
             clinically probable ALS, or clinically possible ALS based on the revised El Escorial
             criteria.

          -  Patients with ALS ≤ 3 years since symptom onset. Symptom onset is defined as date of
             first muscle weakness or dysarthria.

          -  Upright slow vital capacity (SVC)≥ 60% of predicted.

          -  If taking riluzole and/or Nuedexta®, stable regimen is required for ≥ 30 days prior to
             screening.

          -  Medically (either independently or with caregiver assistance) able to comply with
             study procedures, including subcutaneous (SC) injections of study medication and
             adherence to concomitant medication restrictions.

        Exclusion Criteria:

          -  Any medical condition known to have an association with motor neuron dysfunction which
             might confound or obscure the diagnosis of ALS.

          -  Tracheostomy, diaphragm pacing, or ongoing need for assisted ventilation of any type
             (e.g., bilevel positive airway pressure) for treatment of ALS-related respiratory
             dysfunction (vital capacity of < 60% predicted, nocturnal desaturation, and/or
             nocturnal hypoventilation). Patients on assisted ventilation for other reasons require
             approval from the Medical Monitor. (Supplemental oxygen is acceptable).

          -  Recorded diagnosis or evidence of major psychiatric disorder.

          -  Clinically evident cognitive and/or behavioral impairment that in the opinion of the
             Investigator would impair the ability of the patient to comply with the study
             procedures.

          -  Therapies and/or Medications:

               1. History of prior sensitivity to Acthar or other porcine protein products.

               2. Chronic systemic corticosteroid use, defined as > 20 mg of prednisone or
                  equivalent systemic corticosteroid taken for more than 4 consecutive weeks within
                  6 months prior to randomization. Topical, inhaled, or intra-articular
                  corticosteroids are allowed.

               3. Planned treatment with live or live attenuated vaccines once enrolled in the
                  study.

          -  Participation in another therapeutic (drug or device) investigational study within 30
             days prior to screening.

          -  Type 1 or type 2 diabetes mellitus, or patients currently taking hypoglycemic
             medication.

          -  Contraindication per Acthar Prescribing Information, Appendix D Section 4:
             scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent
             surgery, history of or the presence of peptic ulcer, congestive heart failure,
             uncontrolled hypertension, primary adrenocortical insufficiency, or adrenal cortical
             hyperfunction.

               1. For the purposes of this study, osteoporosis is defined as a history of a lumbar
                  spine and/or femoral neck T-score ≤ -2.5 on bone densitometry (DXA), OR
                  osteoporosis requiring pharmacologic therapy, OR a history of non-traumatic low
                  impact hip or vertebral fracture, OR patient reported history of osteoporosis.

               2. For the purposes of this study, history of peptic ulcer is defined as ≤ 6 months
                  prior to screening.

               3. For the purposes of this study, uncontrolled hypertension is defined as mean
                  systolic blood pressure ≥ 140 mmHg and diastolic blood pressure ≥ 90 mmHg on ≥ 3
                  seated readings taken at least 5 minutes apart during the screening period.

               4. For the purposes of this study, congestive heart failure is defined as New York
                  Heart Association Functional Class III-IV.
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