Clinical trial NCT02169219: Pilot Study of Short-Course Glucocorticoids and Rituximab for Treatment of ANCA-Associated Vasculitis
Official title: Short-Course Glucocorticoids and Rituximab in ANCA-Associated Vasculitis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Granulomatosis With Polyangiitis
Microscopic Polyangiitis
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: Genentech, Inc.
First posted: 2014-06-23
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this pilot study is to test whether an 8-week course of glucocorticoids, combined with rituximab, is effective in treating ANCA-associated vasculitis.

Detailed description: The primary aim of this pilot study is to examine whether an 8 week course of glucocorticoids, in combination with rituximab, is effective in inducing and maintaining disease remission for up to 6 months in a subset of patients with ANCA-associated vasculitis (AAV) who have a more favorable prognosis.

This pilot study will enroll 20 patients with active AAV. Close patient follow-up will insure that any patients who require courses of glucocorticoids longer than two months will receive longer therapy, if appropriate for their well-being.

Eligibility criteria: Inclusion Criteria:

* Patients ages 18-85 years old
* Diagnosis of GPA or MPA according to the definitions of the Chapel Hill Consensus Conference
* New diagnosis or disease flare with a Birmingham Vasculitis Activity Score/Wegener's granulomatosis (BVAS/WG) of \> 3

Exclusion Criteria:

* Renal disease in patients with PR3-ANCA as defined by any of the following:
* Urinary red blood cell casts
* Biopsy-proven glomerulonephritis
* Increase in serum creatinine of \>30% over baseline
* Severe renal disease in patients with MPO-ANCA as defined by both of the following:
* Urinary red blood cell casts or biopsy-proven glomerulonephritis
* Estimated glomerular filtration rate \< 30 ml/min/1.73m2
* Diffuse alveolar hemorrhage requiring ventilatory support
* GC treatment for longer than 14 days prior to enrollment unless patient has been on a stable maintenance dose of prednisone at the time of the flare
* Daily oral cyclophosphamide within 1 month prior to enrollment
* Completed a remission induction course of cyclophosphamide or rituximab within 4 months of enrollment
* Hepatitis B infection
* HIV infection
* History of anti-GBM disease
* Other uncontrolled disease, including drug and alcohol abuse, that may interfere with the study
* Pregnancy or breastfeeding
* History of severe allergic reactions to human or chimeric monoclonal antibodies

Primary outcomes:
measure: Complete Remission
timeFrame: 6 months
Secondary outcomes:
measure: Disease Response
timeFrame: 4 weeks
measure: Partial Remission
timeFrame: 8 weeks
measure: Sustained Complete Remission
timeFrame: 6 months
measure: Limited Flares
timeFrame: 6 months
measure: Severe Flares
timeFrame: 6 months
measure: Early Treatment Failures
timeFrame: 4 weeks
measure: Vasculitis Damage Index (VDI)
timeFrame: 24 months
Other outcomes:
Locations:
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
Study officials:
affiliation: Massachusetts General Hospital and Harvard Medical School
name: John H Stone, MD
role: PRINCIPAL_INVESTIGATOR
References: