Effect of Tofacitinib in Treating ANCA-associated Vasculitis | NCT04973033

Studies
Effect of Tofacitinib in Treating ANCA-associated Vasculitis

NCT04973033

Sponsor

Shanghai Zhongshan Hospital

Status

Completed

Last Update Posted

Jul 22, 2021Actual

Enrollment

10Actual

Phase

Not Applicable

Conditions

ANCA Associated Vasculitis
Drug Use
JAK-STAT Pathway Deregulation

Interventions

Tofacitinib

Countries

China

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jul 2021

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Dec 1, 2019Actual

Primary Completion Date

Jan 31, 2021Actual

Completion Date

Jan 31, 2021Actual

First Submitted Date

Jul 5, 2021

First Submission Date that Met QC Criteria

Jul 20, 2021

First Posted Date

Jul 22, 2021Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jul 20, 2021

Last Update Posted Date

Jul 22, 2021Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Not provided

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

10Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Tofactitinib	Experimental	Tofacitinib 5mg twice a day	Drug: Tofacitinib

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Tofacitinib	Drug	patients enrolled were prescribed tofacitinib 5mg twice a day orally.	Tofactitinib

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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.	From the enrollment to the end of follow-up [0 to 13 months.]

Secondary Outcomes

Measure	Description	Time Frame
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.	From the enrollment to the end of follow-up [0 to 13 months].
Changes in erythrocyte sedimentation rate (ESR)

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

75 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Name	Affiliation	Role
Lindi Jiang, PhD	Fudan University	Study Chair

Facility	Status	City	State	ZIP	Country	Contacts
Department of Rheumatology in Zhongshan hospital, Fudan University		Shanghai	Shanghai Municipality	200032	China

Condition Browse Module

MeSH Terms

ID	Term
D056648	Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

Ancestor Terms

ID	Term
D056647	Systemic Vasculitis
D014657	Vasculitis
D014652	Vascular Diseases
D002318	Cardiovascular Diseases

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Browse Branches

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