Status:
COMPLETED
A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis
Lead Sponsor:
Mallinckrodt
Conditions:
Amyotrophic Lateral Sclerosis
Eligibility:
All Genders
18-80 years
Phase:
PHASE2
Brief Summary
This 8-week randomized, open-label evaluation will examine the acute safety and tolerability of 4 different dosing regimens of Acthar to inform dose selection for future studies of Acthar in patients ...
Eligibility Criteria
Inclusion
- 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
- 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:
- History of prior sensitivity to Acthar or other porcine protein products.
- 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.
- 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.
- 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.
- For the purposes of this study, history of peptic ulcer is defined as ≤ 6 months prior to screening.
- 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.
- For the purposes of this study, congestive heart failure is defined as New York Heart Association Functional Class III-IV.
Key Trial Info
Start Date :
July 1 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2014
Estimated Enrollment :
43 Patients enrolled
Trial Details
Trial ID
NCT01906658
Start Date
July 1 2013
End Date
December 1 2014
Last Update
January 6 2017
Active Locations (17)
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1
Questcor Investigational Site
Birmingham, Alabama, United States, 35233
2
Questcor Investigational Site
Phoenix, Arizona, United States, 85018
3
Questcor Investigational Site
San Francisco, California, United States, 94115
4
Questcor Investigational Site
Stanford, California, United States, 94305