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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 July 2021
• Main ID: NCT04946669
• Date of registration: 17/05/2021
• Prospective Registration: No
• Primary sponsor: The First Affiliated Hospital with Nanjing Medical University
• Public title: An Exploratory Study on the Efficacy and Safety of Abatacept in the Treatment of Refractory Dermatomyositis
• Scientific title: An Exploratory Study on the Efficacy and Safety of Abatacept in the Treatment of Refractory Dermatomyositis
• Date of first enrolment: February 1, 2021
• Target sample size: 20
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT04946669
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Early Phase 1

Countries of recruitment

China

Contacts

• Name: Wenfeng Tan, PhD, MD

Key inclusion & exclusion criteria

Inclusion Criteria:

1. 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.

2. 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.

3. If the patient has previously used biological agents, etc., the washout period shall
be completed.

4. Patients or their guardians fully understand the content of this study, are willing to
participate in the study, and sign the informed consent.

Exclusion Criteria:

1. Other rheumatoid immune diseases: including but not limited to rheumatoid arthritis,
systemic lupus erythematosus, systemic sclerosis, Sjogren's syndrome, primary biliary
cirrhosis, etc.

2. 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.

3. 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.).

4. 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.

5. Pregnant and lactating women, women of reproductive age who cannot guarantee
contraception.

6. Patients with allergic constitution, who have been allergic to various drugs in the
past.

7. Mental disorders, or other patients unable to cooperate with treatment.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Abatacept

Dermatomyositis

Intervention(s)

Drug: Abatacept

Primary Outcome(s)

IMACS score improved [Time Frame: 12 weeks after treat with Abatacept]

Secondary Outcome(s)

Dose of glucocorticoids used [Time Frame: 12 weeks after treat with Abatacept]

Secondary ID(s)

ABA-DM

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Natural Science Foundation of China