A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis

Launched by MALLINCKRODT · Jul 19, 2013

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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.