CompletedPhase 4

Abatacept in Juvenile Dermatomyositis

United States10 participantsStarted 2015-11

Plain-language 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.

Who can participate

Age range7 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Adults with definite or probable JDM and pediatric patients 7 years of age and older with definite or probable JDM.
✓. Body weight of at least 25 kg (or at least 55 lbs) and age ≥ 7 years of age.
✓. Patients must reside within the United States or Canada.
✓. Refractory myositis, as defined by the intolerance to or an inadequate response to corticosteroids plus an adequate regimen of at least one other immunosuppressive agent, including azathioprine, methotrexate, IVIG, mycophenolate mofetil, cyclophosphamide, tacrolimus or cyclosporine A, or a biologic therapy. The definition of intolerance is: side effects that require discontinuation of the medication or an underlying condition that precludes the further use of the medication.
✓. At least moderately active disease as documented by:
✓. Therapy with prednisone or another glucocorticoid is required, unless there is documented intolerance in the medical record or a medical condition that contraindicates further use of prednisone. The prednisone dose must be stable for at least 4 weeks prior to the screening visit.
✓. Background therapy with at least 1 non-corticosteroid immunosuppressive agent is required at a stable dose for at least 6 weeks prior to the screening visit unless there is documentation that the patient is intolerant, which is defined as side effects that require discontinuation of the medication(s) or an underlying condition that precludes the further use of the IS medication.
✓. If an immunosuppressive agent was discontinued prior to the screening visit then there must be a:

Exclusion criteria

✕. Drug-induced myositis (myositis in patients taking medications known to induce myositis-like syndromes, including, but not limited to, statin agents, fibric acid derivatives, and colchicine).
✕. Juvenile polymyositis; inclusion body myositis; cancer-associated myositis, defined as the diagnosis of myositis within 2 years of the diagnosis of cancer except basal or squamous cell skin cancer or carcinoma in situ of the cervix if at least 5 years since excision
✕. Myositis in overlap with another connective tissue disease (CTD) that precludes the accurate assessment of a treatment response (for example, difficulty in assessing muscle strength in a scleroderma patient with associated myositis)
✕. History of receiving a live vaccine 4 weeks prior to initiation of study treatment
✕. Joint disease, severe calcinosis, or other musculoskeletal condition, which precludes the ability to quantitate muscle strength.
✕. Wheelchair bound patients.
✕. Known hypersensitivity to abatacept or prior receipt of abatacept
✕. Concomitant illness that would prevent adequate patient assessment or in the investigators opinion pose an added risk for study participants:

What they're measuring

Number of Patients Meeting the Definition of Improvement (DOI) at Week 24: at Least 3 of 6 Core Set Measures (CSM) Improved by ≥ 20% With no More Than 2 CSM Worsening by ≥ 25% (Not Including the Manual Muscle Testing).

Timeframe: week 0 to week 24

Number of Treatment-emergent Adverse Events

Timeframe: week 0 to week 24

Trial details

NCT IDNCT02594735

SponsorGeorge Washington University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2021-07-16

Results submitted2022-06-29

View on ClinicalTrials.gov More Dermatomyositis trials

Who can participate

Age range7 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Adults with definite or probable JDM and pediatric patients 7 years of age and older with definite or probable JDM.
✓. Body weight of at least 25 kg (or at least 55 lbs) and age ≥ 7 years of age.
✓. Patients must reside within the United States or Canada.
✓. Refractory myositis, as defined by the intolerance to or an inadequate response to corticosteroids plus an adequate regimen of at least one other immunosuppressive agent, including azathioprine, methotrexate, IVIG, mycophenolate mofetil, cyclophosphamide, tacrolimus or cyclosporine A, or a biologic therapy. The definition of intolerance is: side effects that require discontinuation of the medication or an underlying condition that precludes the further use of the medication.
✓. At least moderately active disease as documented by:
✓. Therapy with prednisone or another glucocorticoid is required, unless there is documented intolerance in the medical record or a medical condition that contraindicates further use of prednisone. The prednisone dose must be stable for at least 4 weeks prior to the screening visit.
✓. Background therapy with at least 1 non-corticosteroid immunosuppressive agent is required at a stable dose for at least 6 weeks prior to the screening visit unless there is documentation that the patient is intolerant, which is defined as side effects that require discontinuation of the medication(s) or an underlying condition that precludes the further use of the IS medication.
✓. If an immunosuppressive agent was discontinued prior to the screening visit then there must be a:

Exclusion criteria

✕. Drug-induced myositis (myositis in patients taking medications known to induce myositis-like syndromes, including, but not limited to, statin agents, fibric acid derivatives, and colchicine).
✕. Juvenile polymyositis; inclusion body myositis; cancer-associated myositis, defined as the diagnosis of myositis within 2 years of the diagnosis of cancer except basal or squamous cell skin cancer or carcinoma in situ of the cervix if at least 5 years since excision
✕. Myositis in overlap with another connective tissue disease (CTD) that precludes the accurate assessment of a treatment response (for example, difficulty in assessing muscle strength in a scleroderma patient with associated myositis)
✕. History of receiving a live vaccine 4 weeks prior to initiation of study treatment
✕. Joint disease, severe calcinosis, or other musculoskeletal condition, which precludes the ability to quantitate muscle strength.
✕. Wheelchair bound patients.
✕. Known hypersensitivity to abatacept or prior receipt of abatacept
✕. Concomitant illness that would prevent adequate patient assessment or in the investigators opinion pose an added risk for study participants:

What they're measuring

Timeframe: week 0 to week 24

Number of Treatment-emergent Adverse Events

Timeframe: week 0 to week 24