5524: PEXIVAS: Plasma exchange and glucocorticoid dosing in the treatment of anti-neutrophil cytoplasm antibody associated vasculitis: an international randomized controlled trial

Status: Closed to Enrollment

For Diseases:

Granulomatosis with Polyangiitis (Wegener's)


Granulomatosis with polyangiitis (GPA, Wegener's) and microscopic polyangiitis (MPA) are types of vasculitis that are known as ANCA-associated vasculitis (AAV). Vasculitis is inflammation of the small blood vessels of your body. There is often an antibody present in the blood of patients with vasculitis, called ANCA, which damages blood vessels in vasculitis. When the kidneys or lungs are affected by vasculitis a treatment called plasma exchange can be used.

The aim of the plasma exchange is to quickly remove ANCA and other harmful substances from the blood. This may help control the disease and the kidneys or lungs can recover. Patients involved in this study have ANCA vasculitis, and their kidney and/or lungs are affected. This study aims to determine how good plasma exchange is, and whether it can be a routine treatment for vasculitis.

Standard treatment of vasculitis is a combination of drugs including glucocorticoids (steroids) in combination with a second drug to suppress the body's immune system. These drugs are effective at controlling the disease but have toxic side effects, particularly infections. It is important to use the correct dose of these drugs in order to balance their benefit with their side effects. We are unsure about how much steroids to use. This study will also compare a standard steroid dose to a reduced steroid dose.

About this Study

This is an interventional study that will be conducted at multiple hospitals and vasculitis centers around the world. About 500 participants with GPA and MPA will be enrolled. All participants will be randomized to receive plasma exchange with standard immunosuppressive medication and steroids or standard immunosuppressive medication and steroids (without plasma exchange). The amount of steroids participants receive will also be randomized. Participants will either receive a reduced-dose tapering schedule or a standard-dose tapering schedule.

Participants will be in this study between 2 to 7 years. During the first year, there will be 7 clinic visits. After the first year, there will only be visits every 6 months until the end of the study.

Target Enrollment:

To be eligible to participate, you must:

  1. Have a diagnosis of granulomatosis with polyangiitis (Wegener's) or microscopic polyangiitis
  2. Positive test for proteinase 3-ANCA or myeloperoxidase-ANCA
  3. Severe vasculitis defined by kidney involvement or lung hemorrhage <
  4. Provision of informed consent by patient or a surrogate decision maker

You are not eligible to participate if:

  • Age <15 years (age <18 years at centers that do not treat pediatric patients)
  • A diagnosis of vasculitis other than GPA or MPA
  • Receipt of dialysis for >21 days immediately prior to randomization or prior renal transplant
  • Pregnancy at study entry, or unwillingness to comply with birth control/abstinence
  • Treatment with >1 IV dose of cyclophosphamide and/or >14 days of oral cyclophosphamide and/or >14 days of prednisone/prednisolone (>30 mg/day) and/or >1 dose of rituximab within the 28 days immediately prior to randomization
  • Plasma exchange in 3 months prior to randomization

Additional inclusion and exclusion criteria apply.

Study Participation

This study is taking place in the following US cities:

  • Boston, MA
  • Chapel Hill, NC
  • Cleveland, OH
  • Los Angeles, CA
  • New York City NY
  • Philadelphia, PA
  • Pittsburgh, PA
  • Rochester, MN
  • St. Louis, MO

Please contact Carol McAlear for additional information.

This study is taking place in the following Canadian centers:

  • Hospital Saint-Luc, Montreal
  • London Health Sciences Centre, London
  • Mount Sinai Hospital, Toronto
  • St Joseph's Hospital, Hamilton
  • St Michael's Hospital, Toronto
  • St Paul's Hospital, Vancouver
  • The Ottawa Hospital, Ottawa
  • University of Alberta, Edmonton
  • University of Calgary, Calgary

Please contact Andrea Mazzettiat for additional information.

For information about the European, Japanese, Australian and New Zealand sites participating in this study:
Please contact Biljana Brezina

For additional information on this trial, please visit:
University of Birmingham: PEXIVAS Trial
Clinicaltrials.gov: clinicaltrials.gov/ct2/show/NCT00987389