An Exploratory Study on the Efficacy and Safety of Abatacept in the Treatment of Refractory Dermatomyositis

NCT04946669 | Unknown Early Phase 1

• 1 other identifier: ABA-DM
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

Dermatomyositis is a heterogeneous disease characterized by involvement of the proximal muscles of the extremities. Some patients have treatment failure or intolerance to the above treatment regimens, which is called refractory dermatomyositis. Abatacept has been approved by the FDA for the treatment of three types of rheumatoid immune diseases, and 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.

Conditions

Dermatomyositis; Abatacept

Keywords

Refractory Dermatomyositis; Abatacept; Efficacy; Safety

Outcome Measures

Primary Outcomes (1)

• IMACS score improved

  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.

  12 weeks after treat with Abatacept

Secondary Outcomes (1)

• Dose of glucocorticoids used

  12 weeks after treat with Abatacept

Other Outcomes (1)

• Occurrence of adverse events

  12 weeks after treat with Abatacept

Study Arms (1)

Refractory Dermatomyositis

EXPERIMENTAL

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

Drug: Abatacept

Interventions

Abatacept DRUG

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.

Refractory Dermatomyositis

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• The First Affiliated Hospital with Nanjing Medical University: lead
• National Natural Science Foundation of China: collaborator

Study Sites (1)

The First Affiliated Hospital with Nanjing Medical University

Nanjing, Jiangsu, 210029, China

RECRUITING

MeSH Terms

Conditions

Dermatomyositis

Interventions

Abatacept

Condition Hierarchy (Ancestors)

Polymyositis; Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Intervention Hierarchy (Ancestors)

Immunoconjugates; Antibodies; Immunoglobulins; Serum Globulins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Globulins

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief Physician

Study Record Dates

• First Submitted: May 17, 2021
• First Posted: July 1, 2021
• Study Start: February 1, 2021
• Primary Completion: July 31, 2022
• Study Completion: July 31, 2022
• Last Updated: July 1, 2021

Record last verified: 2021-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)