- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04973033
Effect of Tofacitinib in Treating ANCA-associated Vasculitis
July 20, 2021 updated by: Shanghai Zhongshan Hospital
Tofacitinib for the Treatment of Anti-Neutrophil Cytoplasm Antibody-associated Vasculitis: a Pilot Study
The goal of this study is to evaluate the efficacy and safety of tofacitinib 5 mg twice daily in AAV patients.
Study Overview
Status
Completed
Intervention / Treatment
Detailed Description
Anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) represents a group of small vessel vasculitides characterized by granulomatous and neutrophilic tissue inflammation, often associated with the production of antibodies that target neutrophil antigens.
The predominantly used treatment for induction of remission in AAV consisted of cyclophosphamide (CYC) plus corticosteroids (GCs) which leads to remission in about 90% of patients.
However, relapses are frequent and remain a challenge.
The optimal drug for maintenance treatment is not determined.
Tofacitinib is a Jak inhibitor which has been proved to be effective in multiple inflammatory diseases such as rheumatoid arthritis.
Considering that T cells and associated cytokine production play an important role in the pathogenesis of AAV via activation of the JAK/ STAT pathway, we hypothesized that tofacitinib-mediated inhibition of JAK signaling may represent an effective therapy for active AAV.
In this prospective, open label, single arm study, tofacitinib 5mg twice a day will be added to the background treatment of GCs and immunosuppressants in AAV, the safety and efficacy of tofacitinib will be assessed.
Study Type
Interventional
Enrollment (Actual)
10
Phase
- Not Applicable
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
Shanghai
-
Shanghai, Shanghai, China, 200032
- Department of Rheumatology in Zhongshan hospital, Fudan University
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 75 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Patients with active AAV met the criteria of 1990 ACR and 2012 Chapel Hill criteria
- Age 18 to 75 years
- Written informed consent obtained before taking part in the study
Exclusion Criteria:
- Severe AAV defined as potentially organ- or life-threatening disease (i.e. alveolar haemorrhage, heart failure caused by myocarditis or pericarditis, progressive neurological symptoms, deaf, blindness, et al.)
- Serum creatinine>120umol/L or proteinuria>1.0g/d
- Receipt of a JAKi therapy previously
- Co-existence of another systemic autoimmune disease
- Secondary vasculitis (following neoplastic disease, an infection or antithyroid drugs)
- Malignancy or history of malignancy
- Infection by HIV, HCV, HBV or tuberculosis-
- Severe uncontrolled cardiovascular, pulmonary, liver, gastrointestinal, endocrine, hematological, neurological, or psychiatric diseases that are not related to systemic vasculitis
- Allergic to JAKi
- Blood dyscrasias including confirmed: Hemoglobin <9 g/dL or Hematocrit <30%; White blood cell count <3.0 x 109/L; Absolute neutrophil count <1.5 x 109/L; Platelet count <100 x 109/L; Alanine transaminase or aspartate aminotransferase or total bilirubin>1.5 upper normal limit; Estimated glomerular filtration rate<60ml/min/1.73m2
- Incapacity or refusal to understand or sign the informed consent form.
- Pregnancy, breastfeeding.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Tofactitinib
Tofacitinib 5mg twice a day
|
patients enrolled were prescribed tofacitinib 5mg twice a day orally.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
The response rate (CR, PR and TR)
Time Frame: From the enrollment to the end of follow-up [0 to 13 months.]
|
The percent of patients who achieved disease response.
The disease response includes:(1) complete remission (CR), defined as the absence of disease activity (BVAS = 0); (2) partial remission (PR) defined as at least 50% reduction of BVAS and no new manifestations; (3) treatment resistance (TR) was defined as less than a 50% reduction or increased disease activity after 4 ~ 6 weeks of treatment.
|
From the enrollment to the end of follow-up [0 to 13 months.]
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
The rate of adverse event
Time Frame: From the enrollment to the end of follow-up [0 to 13 months].
|
The percent of different kinds of adverse events occurred during follow-up.
The adverse event was evaluated according to the CTC-AE 4.0 standard.
|
From the enrollment to the end of follow-up [0 to 13 months].
|
Changes in erythrocyte sedimentation rate (ESR)
Time Frame: From the enrollment to the end of follow-up [0 to 13 months].
|
The change of ESR in different follow-up point compared with the baseline.
|
From the enrollment to the end of follow-up [0 to 13 months].
|
Changes in CRP
Time Frame: From the enrollment to the end of follow-up [0 to 13 months].
|
The change of CRP in different follow-up point compared with the baseline.
|
From the enrollment to the end of follow-up [0 to 13 months].
|
Changes in glucocorticoids steroids (GCs) dosage
Time Frame: From the enrollment to the end of follow-up [0 to 13 months].
|
The change of the prednisone or its equivalent drug in different follow-up point compared with the baseline.
|
From the enrollment to the end of follow-up [0 to 13 months].
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
December 1, 2019
Primary Completion (Actual)
January 31, 2021
Study Completion (Actual)
January 31, 2021
Study Registration Dates
First Submitted
July 5, 2021
First Submitted That Met QC Criteria
July 20, 2021
First Posted (Actual)
July 22, 2021
Study Record Updates
Last Update Posted (Actual)
July 22, 2021
Last Update Submitted That Met QC Criteria
July 20, 2021
Last Verified
July 1, 2021
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- TofAV
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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