ANCA Associated VasculitisNCT04973033
Effect of Tofacitinib in Treating ANCA-associated Vasculitis
A study testing Tofacitinib in people with ANCA Associated Vasculitis and Drug Use, run by Shanghai Zhongshan Hospital at 1 site in China.
Trial summary
The goal of this study is to evaluate the efficacy and safety of tofacitinib 5 mg twice daily in AAV patients. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- ANCA Associated Vasculitis, Drug Use, JAK-STAT Pathway Deregulation
- What is being tested?
- Tofacitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 1 Dec 2019to 31 Jan 2021
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 12Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
China1 site
Department of Rheumatology in Zhongshan hospital, Fudan University
Shanghai, Shanghai Municipality, 200032
About this study
Official title: 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.
Read the 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.
- Being tested
- Tofacitinib
- Keywords
- ANCA associated vasculitis, tofacitinib, safety, efficacy
- Medical subject headings
- Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TofacitinibDrug
Also called Tofacitinib 5mg.bid.po.
patients enrolled were prescribed tofacitinib 5mg twice a day orally.
| Study group | Role | What they receive |
|---|---|---|
| Tofactitinib | Experimental | Tofacitinib 5mg twice a day Receives: Drug: Tofacitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
The response rate (CR, PR and TR)
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.
Measured: From the enrollment to the end of follow-up [0 to 13 months.]
Secondary outcomes (4)
The rate of adverse event
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.
Measured: From the enrollment to the end of follow-up [0 to 13 months].
Changes in erythrocyte sedimentation rate (ESR)
The change of ESR in different follow-up point compared with the baseline.
Measured: From the enrollment to the end of follow-up [0 to 13 months].
Changes in CRP
The change of CRP in different follow-up point compared with the baseline.
Measured: From the enrollment to the end of follow-up [0 to 13 months].
Changes in glucocorticoids steroids (GCs) dosage
The change of the prednisone or its equivalent drug in different follow-up point compared with the baseline.
Measured: From the enrollment to the end of follow-up [0 to 13 months].
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2021
- Study start1 Dec 2019
- Primary completion31 Jan 2021
- Study completion31 Jan 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ANCA Associated VasculitisStudies of the same condition, open and under way.
- More trials run by Shanghai Zhongshan HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shanghai Zhongshan Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Lindi Jiang, PhD, Study chair, Fudan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04973033
- Sponsor's protocol ID
- TofAV
- Status last verified
- Jul 2021
- First submitted
- 5 Jul 2021
- Last updated
- 22 Jul 2021
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tofacitinib for the Treatment of Anti-Neutrophil Cytoplasm Antibody-associated Vasculitis: a Pilot Study
- ClinicalTrials.gov ID
- NCT04973033
- Acronym / sponsor ID
- TofAV
- Keywords
- ANCA associated vasculitis, tofacitinib, safety, efficacy
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Shanghai Zhongshan HospitalUniversity, hospital or other
- Study start
- 1 Dec 2019 (actual)
- Primary completion
- 31 Jan 2021 (actual)
- Study completion
- 31 Jan 2021 (actual)
- First posted
- 22 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this study is to evaluate the efficacy and safety of tofacitinib 5 mg twice daily in AAV patients.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tofactitinib | Experimental | Drug: TofacitinibTofacitinib 5mg twice a day |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (12 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TofacitinibAlso: Tofacitinib 5mg.bid.po.patients enrolled were prescribed tofacitinib 5mg twice a day orally. | Drug | Tofactitinib |
Primary outcome
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.
4 secondary outcomes
The rate of adverse event
Time frame: 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].
Changes in CRP
Time frame: 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].
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jul 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lindi Jiang, PhD study chair
Fudan University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Tofacitinib in people with ANCA Associated Vasculitis and Drug Use, run by Shanghai Zhongshan Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The response rate (CR, PR and TR) (time frame: From the enrollment to the end of follow-up [0 to 13 months.]). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jul 2021.