Acthar in Treatment of Refractory Dermatomyositis and Polymyositis
The purpose of this research study is to evaluate the effectiveness of the study drug, ACTH Gel in people diagnosed with dermatomyositis a disease that causes muscle weakness and is associated with a rash (DM) or polymyositis (PM) a disease that causes muscle weakness without a rash. The study doctors want to evaluate whether ACTH Gel will improve the symptoms of this disease. This drug is approved by the Food and Drug Administration (FDA) for dermatomyositis (DM) and polymyositis (PM). ACTH gel has been an FDA-approved treatment for myositis since 1952, and in 2010 the FDA retained PM and DM as diseases approved for ACTH gel use.
Drug: Adrenocorticotropic Hormone Gel
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Open Label Proof of Concept Study to Evaluate Efficacy and Safety of Adrenocorticotropic Hormone Gel in Refractory Dermatomyositis or Polymyositis|
- Specific Aim 1: To determine the efficacy of H.P. Acthar Gel in refractory adult PM and DM patients defined by the IMACS preliminary definition of improvement (DOI). [ Time Frame: Primary end point: IMACS preliminary definition of improvement (DOI) ] [ Designated as safety issue: No ]3 of any of the 6 core set measures (CSM) improved by ≥ 20%, with no more than 2 CSM worsening by ≥25% (worsening measure cannot include the MMT). The DOI should be met at least once on any of the 6 follow up visits. Subjects not meeting DOI during the trial are treatment failures.
- To assess the safety, tolerability, and steroid-sparing effect of H.P. Acthar Gel in refractory adult PM and DM patients. [ Time Frame: Steroid sparing effect and safety and tolerability at 24 weeks compared to baseline ] [ Designated as safety issue: No ]
- Mean/Median change in glucocorticoid dose (equivalent prednisone dose) at 24 weeks compared to baseline.
- Safety & tolerability: Safety and tolerability is measured by frequency and type of adverse events and serious adverse events. This will be measured by detailed questionnaires, patient report and study withdrawal due to study drug side effects or tolerability problems.
|Study Start Date:||September 2013|
|Estimated Study Completion Date:||July 2016|
|Estimated Primary Completion Date:||November 2015 (Final data collection date for primary outcome measure)|
Experimental: Acthar Gel
Acthar Gel (Adrenocorticotropic Hormone Gel)in refractory PM and DM patients using an open label design for 6 months. We will enroll 10 active and refractory PM/DM patients over a 15 month period, followed by 6 months of additional follow-up for each subject. Study subjects will self-administer subcutaneously H.P. Acthar Gel 80 units (1 ml) twice a week for a period of six months.
Drug: Adrenocorticotropic Hormone Gel
H.P. Acthar Gel 80 units will be self-administered subcutaneously twice weekly by the subject for a period of 6 months.
Other Name: H.P. Acthar Gel
Despite its FDA approval there is very limited data on its clinical effectiveness in PM and DM. There was a recent study published in the peer-review journal Drug Design, Development and Therapy on a retrospective case series evaluating Acthar in the treatment of PM and DM. Acthar was administered to five patients who had previously failed multiple steroid and immunosuppressant treatment regimens. The patients received injections of Acthar over the course of 12 weeks or more. Improvement in PM and DM symptoms related to disease exacerbations was seen in all five patients. Symptom improvements included increased muscle strength, resolution of disease-related skin manifestations and improvements in the ability to perform tasks associated with daily living. All of these patients tolerated the treatment well with no significant side effects reported. The paper, "Treating refractory dermatomyositis or polymyositis with adrenocorticotropic hormone gel: a retrospective case series," was authored by Dr. Todd Levine, M.D., Co-Director of the Neurophysiology Department at Banner Good Samaritan Medical Center, Assistant Professor at the University of Arizona in Neurology, and Member of Phoenix Neurological Associates.
H.P. Acthar® Gel, or Acthar, is a prescription medication containing the hormone adrenocorticotropin (hormone produced and secreted by the anterior pituitary gland), also known as ACTH. H.P. Acthar Gel is a highly purified preparation of adrenocorticotropic hormone (ACTH) in a gel that is designed to provide extended release of the ACTH following injection. Acthar was originally approved by the FDA in 1952. It is approved for use in 19 different conditions including dermatomyositis and polymyositis.
Acthar is designed to provide a prolonged release of the medication after it is injected. Acthar is not a steroid; it works by helping your body produce its own natural steroid hormones, such as cortisol, corticosterone, and aldosterone. Acthar is an injection that is given intramuscularly (into the muscle). Subjects enrolled in the study will be asked to self administer Acthar two times per week. Subjects will be provided training by the principal investigator on how to perform the self injections.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01906372
|United States, New York|
|North Shore LIJ Medical Center|
|Great Neck, New York, United States, 11021|
|United States, Pennsylvania|
|University of Pittsburgh|
|Pittsburgh, Pennsylvania, United States, 15261|
|Principal Investigator:||Rohit Aggarwal, MD||University of Pittsburgh, Division of Rheumatology|