An Exploratory Study on the Efficacy and Safety of Abatacept in the Treatment of Refractory Dermatomyositis
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04946669|
Recruitment Status : Recruiting
First Posted : July 1, 2021
Last Update Posted : July 1, 2021
- Study Details
- Tabular View
- No Results Posted
- How to Read a Study Record
|Condition or disease||Intervention/treatment||Phase|
|Dermatomyositis Abatacept||Drug: Abatacept||Early Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Exploratory Study on the Efficacy and Safety of Abatacept in the Treatment of Refractory Dermatomyositis|
|Actual Study Start Date :||February 1, 2021|
|Estimated Primary Completion Date :||July 31, 2022|
|Estimated Study Completion Date :||July 31, 2022|
Experimental: Refractory Dermatomyositis
Patients who have been receiving glucocorticoids in combination with at least one immunosuppressive therapy for at least 3 months and who have failed therapy or are intolerant to therapy
Abatacept is a CTLA-4 fusion protein that inhibits T cell activation by competitively binding to CD80/CD86 and blocking the second signal. To date, Abatacept has been approved by the FDA for the treatment of three types of rheumatoid immune diseases (refractory rheumatoid arthritis, juvenile idiopathic arthritis, and active psoriatic arthritis). The international consensus of experts recommends abacepil as a second-line regimen for the treatment of refractory dermatomyositis based on the evidence of case reports. However, to date, there is no relevant report or clinical study registration information of Abatacept in the treatment of dermatomyositis.
- IMACS score improved [ Time Frame: 12 weeks after treat with Abatacept ]
Improvement of 3 core parameters of disease activity in the core assessment index of IMACS over 20 percent with no more than 2 parameters deteriorating over 25 percent.
And parameters of IMACS include Physician Global Assessment, Patient Global Assessment, Muscle strength assessment with MMT-8, Sports Ability Assessment Questionnaire, laboratory evaluation including creatine kinase, aldolase, lactic dehydrogenase and so on, Evaluation of myositis disease activity using MDAAT and appraisal of life quality.
- Dose of glucocorticoids used [ Time Frame: 12 weeks after treat with Abatacept ]Dose of glucocorticoids used
- Occurrence of adverse events [ Time Frame: 12 weeks after treat with Abatacept ]Occurrence of adverse events
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Dermatomyositis confirmed in accordance with Bohan-Peter criteria for inflammatory myopathy, or clinically free myopathic dermatomyositis according to the revised Sontheimer criteria, aged 18 years or older, regardless of gender.
- Patients with refractory dermatomyositis, specifically defined as those who have received glucocorticoid combined with at least one immunosuppressive therapy for at least 3 months and have failed treatment or intolerance to treatment.Treatment failure was defined as improvement of 3 core parameters in the core assessment measures of iMACS <20%, or more than 2 parameters deterioration >25%.Treatment intolerance is defined as a patient experiencing side effects that require discontinuation of the drug or an underlying condition that prevents further use of the drug.Before enrollment, the dosage of glucocorticoids was <1mg/kg/day, prior use of at least one immunosuppressant (including methotrexate, azathioprine, cyclosporine, tacrolimus, mycophenolic ester and cyclophosphamide, etc.) at a stable dose >3 months.
- If the patient has previously used biological agents, etc., the washout period shall be completed.
- Patients or their guardians fully understand the content of this study, are willing to participate in the study, and sign the informed consent.
- Other rheumatoid immune diseases: including but not limited to rheumatoid arthritis, systemic lupus erythematosus, systemic sclerosis, Sjogren's syndrome, primary biliary cirrhosis, etc.
- combined with other myopathy causing myopathy and myasthenia;These include neurological diseases (such as muscular dystrophy, myasthia gravis, amyotrophic lateral sclerosis, Guillain-Barre syndrome, etc.), tumors, drug effects (such as statins, etc.), infections, genetic diseases, endocrine diseases, electrolyte disorders, rhabdomyolysis, etc.
- Patients with severe heart, liver, kidney and other important organs and blood and endocrine system lesions:Including but not limited to decompensated cardiac insufficiency, refractory hypertension and abnormal ecg, cereal third transaminase or aspertate aminotransferase more than 2 times higher than normal reference value online, renal tubular acidosis, renal interstitial lesions, renal insufficiency, serious leucopenia, severe anemia, severe thrombocytopenia and other serious diseases, such as tumor, etc.).
- Active infection, glucocorticoid and immunosuppressive therapy may aggravate infection;Hepatitis B virus surface antigen and hepatitis C antibody were positive.Active TB patients who have been treated for active TB within the previous 3 years, or who have been screened for latent TB, and who are positive for PPD combined with T-SPOT, or positive for sputum bacteria.
- Pregnant and lactating women, women of reproductive age who cannot guarantee contraception.
- Patients with allergic constitution, who have been allergic to various drugs in the past.
- Mental disorders, or other patients unable to cooperate with treatment.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04946669
|The First Affiliated Hospital with Nanjing Medical University||Recruiting|
|Nanjing, Jiangsu, China, 210029|
|Contact: Wenfeng Tan, PhD, MD 086 18061202878 email@example.com|
|Contact: Lingxiao Xu, PhD, MD 086 18020130778 firstname.lastname@example.org|
|Responsible Party:||Wenfeng Tan, Chief Physician, The First Affiliated Hospital with Nanjing Medical University|
|Other Study ID Numbers:||
|First Posted:||July 1, 2021 Key Record Dates|
|Last Update Posted:||July 1, 2021|
|Last Verified:||June 2021|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
Nervous System Diseases
Connective Tissue Diseases
Immune Checkpoint Inhibitors
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs