Abatacept in Juvenile Dermatomyositis

Status Completed

Clinical Trial Summary

The purpose of this study is to assess the safety and efficacy of subcutaneous abatacept in 10 patients seven years of age and older with refractory JDM.

Clinical Trial Description

JDM is a chronic systemic autoimmune disease with a predominance of muscle and skin inflammation of unknown etiology and varying prognosis. Children with JDM unresponsive to corticosteroids or other immunosuppressive medications face poor clinical and functional outcome and suffer various sequelae of the disease. Abatacept is a fully human soluble recombinant protein consisting of the cytotoxic T cell Lymphocyte Antigen-4 (CTLA4) fused with Fc region of human IgG1 that has been modified to prevent complement fixation and antibody-dependent cellular cytotoxicity. Abatacept binds specifically to the CD80 (B7-1) and CD86 (B7-2) molecules, those expressed on antigen-presenting cells (APCs). Upon engagement of CTLA4 to CD80 or CD86, the resultant inhibition of signal transduction inhibits T cell activation.. The rationale for use of Abatacept in the therapy of JDM includes the expression of CTLA4, CD28, CD86, and CD40 on inflammatory cells of muscle biopsies of patients with DM, as well as CTLA4 and CD28 on muscle cells. A patient's participation in this study will last approximately 24 weeks with possible extension to 48 weeks. At screening, participants will have a physical exam, muscle strength assessment, blood and urine collection, and chest x-ray; they will also be asked to complete several questionnaires. All participants will receive each week subcutaneous injection of Abatacept. Each injection will be given on an outpatient basis. There will be a total of 6 study visits. All participants will visit the outpatient clinic at selected time points for muscle strength testing, a physical exam, disease activity measurements, blood and urine collection, and muscle MRI; they will also be asked to complete several questionnaires. During the study, participants will be monitored closely for improvement or worsening of their disease and for serious drug related side effects. Eligibility Ages Eligible for Study: ≥ 7 years and older Genders Eligible for Study: Both Race/Ethnic Backgrounds Eligibility for Study: No restrictions ;

Study Design

Related Conditions & MeSH terms

Dermatomyositis

Clinical Trial Details

NCT number	NCT02594735
Study type	Interventional
Source	George Washington University
Contact
Status	Completed
Phase	Phase 4
Start date	November 2015
Completion date	October 22, 2021

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT05833711` - Study Evaluating Efficacy and Safety of Froniglutide (PF1801) in Patients With Idiopathic Inflammatory Myopathy	Phase 2
Completed	`NCT01906372` - Acthar in Treatment of Refractory Dermatomyositis and Polymyositis	Phase 2
Completed	`NCT01813617` - Outcome in Patients With Recent Onset Polymyositis and Dermatomyositis	N/A
Completed	`NCT01165008` - Anakinra in Myositis	Phase 2/Phase 3
Completed	`NCT00004357` - Absorption of Corticosteroids in Children With Juvenile Dermatomyositis	Phase 2
Recruiting	`NCT05832034` - Add-on Intravenous Immunoglobulins in Early Myositis	Phase 2
Recruiting	`NCT05979441` - A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Active Idiopathic Inflammatory Myopathy	Phase 3
Not yet recruiting	`NCT05027152` - Muscle Function and Effects of Repetitive Task Training in Patients With Inflammatory Myopaties	N/A
Active, not recruiting	`NCT04723303` - Phase 1 Study of ULSC in Patients With Polymyositis (PM) and Dermatomyositis (DM)	Early Phase 1
Completed	`NCT03267277` - Sodium Thiosulfate for Treatment of Calcinosis Associated With Juvenile and Adult Dermatomyositis	Phase 2/Phase 3
Recruiting	`NCT05437263` - A Study to Investigate the Efficacy and Safety of Brepocitinib in Adults With Dermatomyositis	Phase 3
Active, not recruiting	`NCT04044690` - A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of IgPro20 in Adults With Dermatomyositis (DM)	Phase 3
Recruiting	`NCT05523167` - A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy.	Phase 2/Phase 3
Not yet recruiting	`NCT06004817` - Evaluation of Severity in Juvenile and Adult-onset Dermatomyositis
Recruiting	`NCT03324152` - Effects of High-intensity Interval Training (HIIT) in Recent Onset Polymyositis and Dermatomyositis	N/A
Completed	`NCT02043548` - Tocilizumab in the Treatment of Refractory Polymyositis and Dermatomyositis	Phase 2
Completed	`NCT03414086` - Predictor of Clinical Response to Acthar in Myositis
Completed	`NCT04628936` - Open-label Extension to the Phase 2 Crossover Study (PRESIDIO) Evaluating KZR-616 in Patients With PM and DM.	Phase 2
Recruiting	`NCT03293615` - Exercise Capacity of Patients With Dermatomyosis	N/A
Completed	`NCT06002750` - Evaluation of Tp-e Interval and Tp-e/QT Ratio in Dermatomyositis and Analysis of Their Relationship With Inflammation

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

Abatacept in Juvenile Dermatomyositis — AID

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms