Dermatomyositis and Polymyositis Registry (ADAPT)

Acthar Dermatomyositis and Polymyositis Treatment

By creating a registry, physicians will have the opportunity to understand the clinical outcomes of Myositis patients treated with Acthar. Despite the availability of clinical exams, muscle biopsies, and other testing, it is surmised that there may be a more important classification of myositis that physicians are not diagnosing which could possibly lead to improper treatment due to inaccurate diagnosis. There may be several types of immune and inflammatory myositis (IIM) that do not fit well into the typical sub classifications of myositis.

Study Overview

• Status: Unknown
• Conditions: Dermatomyositis; Polymyositis
• Intervention / Treatment: Drug: Acthar

Detailed Description

Retrospective and prospective data will be collected from physicians who have prescribed Acthar to myositis patients to determine what specific characteristics each patient has based on biopsy analysis, laboratory results, and clinical exams. Through biopsy analysis, subcategories of IIM will be determined and could illustrate which of these IIMs may be more responsive to Acthar therapy.

• Study Type: Observational
• Enrollment (Anticipated): 100

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85018
      • PNA Center for Neurological Research
  • California
    • San Diego, California, United States, 92108
      • Ara Dikranian MD
  • Missouri
    • Columbia, Missouri, United States, 65201
      • Neurology INC
  • New York
    • Brooklyn, New York, United States, 11215
      • New York Methodist Hospital
  • Vermont
    • Burlington, Vermont, United States, 05401
      • University of Vermont

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 85 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

All sites chosen are well-established universities, academic centers or private practices that specialize in neuromuscular diseases. These are physicians who have several myositis patients and who are prescribing or would prescribe Acthar to myositis patients

Description

Inclusion Criteria:

1. Age 18-85
2. Male or Female
3. Clinical or pathologic diagnosis of polymyositis or dermatomyositis
4. Capable of providing informed consent and complying with treatment regimen

Exclusion Criteria:

1. History of scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex
2. Recent surgery, history of or the presence of a peptic ulcer, congestive heart failure, uncontrolled hypertension, or sensitivity to proteins of porcine origin
3. Any other co-morbid condition which would make completion of the trial unlikely
4. If female, pregnant or breast-feeding; or, if of childbearing age, an unwillingness to use appropriate birth control

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Determining if Acthar treatment improves disease progression	To create and maintain a registry linking clinical information, dosing and clinical response in patients with refractory myositis and to determine if Acthar treatment improves disease progression	2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Subgroups may predict response to Acthar therapy	To determine if there are different subgroups that can be defined myopathologically that may predict response to Acthar therapy.	2 years

Collaborators and Investigators

• Sponsor: Phoenix Neurological Associates, LTD
• Investigators: Principal Investigator: Petros Efthimiou, MD, New York Presbyterian Brooklyn Methodist Hospital; Principal Investigator: Ara Dikranian, MD, Ara Dikranian MD; Principal Investigator: Justine Malone, MD, Neurology INC; Principal Investigator: Rup Tandan, MD, University of Vermont

Study record dates

Study Major Dates

• Study Start: April 1, 2013
• Primary Completion (ANTICIPATED): December 1, 2015

Study Registration Dates

• First Submitted: July 6, 2012
• First Submitted That Met QC Criteria: July 6, 2012
• First Posted (ESTIMATE): July 10, 2012

Study Record Updates

• Last Update Posted (ESTIMATE): November 6, 2015
• Last Update Submitted That Met QC Criteria: November 5, 2015
• Last Verified: November 1, 2015

More Information

Terms related to this study

• Keywords: Myositis; Acthar treatment
• Additional Relevant MeSH Terms: Nervous System Diseases; Skin Diseases; Musculoskeletal Diseases; Connective Tissue Diseases; Muscular Diseases; Neuromuscular Diseases; Myositis; Dermatomyositis; Polymyositis
• Other Study ID Numbers: ADAPT