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This study aims to conduct a randomized controlled trial to compare the efficacy and safety of Baricitinib and Adalimumab (ADA) in the treatment of refractory intestinal Behçet's Syndrome (BS). The objective is to demonstrate if Baricitinib is non-inferior to ADA in controlling BS inflammation, reducing BS recurrence, alleviating gastrointestinal symptoms and promoting intestinal mucosal healing.
The non-inferiority will be established by comparing the lower bound of the two-sided 95% confidence interval with the non-inferiority margin. If the lower bound was larger than the margin, Baricitinib would be regarded as non-inferiror to ADA. Superority will be further assessed in case that the non-inferiority is established. Both ITT and PP analysis will be conducted for the primary outcome given the non-inferiority design. Trial result will be primarily interpreted based on ITT analysis.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Baricitinib for IBS | Experimental |
| |
| Adalimumab for IBS | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Baricitinib | Drug | Participants randomized to this arm will maintain their original steroids and/or immunomodulators, combined with Baricitinib 4 mg/day for 6 months. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of patients with marked improvement (MI) at week 24 of follow-up | Based on the combined assessment of gastrointestinal symptoms and endoscopy scores[1], gastrointestinal symptom scoring is defined as follows: 0 points, asymptomatic; 1 point, does not affect daily life; 2 points, mildly affects daily life; 3 points, moderately affects daily life; 4 points, severely affects daily life. Endoscopy scoring is defined as follows: 0 points, mucosal healing; 1 point, maximum ulcer ≤ original 1/4; 2 points, maximum ulcer between original 1/4 and 1/2; 3 points, maximum ulcer ≥ original 1/2 or enlargement. Marked improvement is defined as gastrointestinal symptom and endoscopy scores both ≤ 1. [1]Sugimura, N. et al. Real-world efficacy of adalimumab and infliximab for refractory intestinal Behçet's disease. Dig. Liver Dis. 51, 967-971 (2019). | Baseline to week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of patients with CR (complete response) at week 24 of follow-up | CR is defined as both gastrointestinal symptoms and endoscopy scores being 0. | Baseline to week 24 |
| Proportion of patients with improvement of ≥1 point in gastrointestinal symptom scores compared to baseline at week 24 of follow-up |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Jinjing Liu, M.D. | Contact | 86-10-69151188 | pumch_followup@126.com | |
| Wenjie Zheng, M.D. | Contact |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking Union Medical College Hospital | Not yet recruiting | Beijing | Beijing Municipality | 100730 | China |
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| ID | Term |
|---|---|
| D001528 | Behcet Syndrome |
| ID | Term |
|---|---|
| D009059 | Mouth Diseases |
| D009057 | Stomatognathic Diseases |
| D014606 | Uveitis, Anterior |
| D015864 | Panuveitis |
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| ID | Term |
|---|---|
| C000596027 | baricitinib |
| D000068879 | Adalimumab |
| ID | Term |
|---|---|
| D061067 | Antibodies, Monoclonal, Humanized |
| D000911 | Antibodies, Monoclonal |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
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|
| Adalimumab | Drug | Participants randomized to this arm will maintain their original steroids and/or immunomodulators, combined with ADA (initially ADA 160 mg subcutaneous injection, followed by 80 mg ADA after 2 weeks, then 40 mg ADA every 2 weeks thereafter) for 6 months. |
|
|
| Baseline to week 24 |
| Changes in C-reactive protein (CRP) compared to baseline | The hs-CRP is acute phase reactants that are indicative of the patient's disease activity. | Baseline to week 24 |
| Changes in erythrocyte sedimentation rate (ESR) compared to baseline | The ESR is acute phase reactants that are indicative of the patient's disease activity. | Baseline to week 24 |
| Changes in DAIBD scores compared to baseline | Disease Activity Index for Intestinal Behcet's Disease (DAIBD); | Baseline to week 24 |
| Changes in BDCAF scores compared to baseline | Behçet's Disease Current Activity Index (BDCAF); | Baseline to week 24 |
| Changes in SF-36 of life questionnaires compared to baseline | Short Form (36) Health Survey(SF-36); | Baseline to week 24 |
| Changes in IBDQ quality of life questionnaires compared to baseline | Inflammatory Bowel Disease Questionnaire (IBDQ) | Baseline to week 24 |
| Incidence of Treatment-Emergent Adverse Events | Record the types, frequency, and severity of adverse events, as well as the number of study participants who were drop out due to any adverse events. Adverse event: Any medical condition that affects the health of the participant during the study or worsens a preexisting medical condition, regardless of whether it is causally related to the study, is considered an adverse event. This can be symptoms, signs, or abnormal laboratory tests. Serious adverse event (SAE): Any significant medical event that causes death, is life-threatening, requires hospitalization, results in permanent or severe disability or loss of function, or the investigator deems it a significant medical event that may seriously harm the participant or require medical intervention to prevent the above outcomes. The causal relationship between the serious adverse event and the study is determined by the investigator's discussion. | Baseline to week 24 |
| Peking Union Medical College Hospital | Recruiting | Beijing | China |
|
| D014605 |
| Uveitis |
| D014603 | Uveal Diseases |
| D005128 | Eye Diseases |
| D014657 | Vasculitis |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D056660 | Hereditary Autoinflammatory Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D017445 | Skin Diseases, Vascular |
| D007162 |
| Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |