Mycophenolate Mofetil for Treatment of Relapses of Wegener's Disease or Microscopic Polyangiitis (MPA)

University Medical Center Groningen (UMCG)

Status and phase: Completed; Phase 3

Conditions: Vasculitis; Wegener's Granulomatosis

Treatments: Drug: cyclophosphamide; Drug: mycophenolate mofetil

Study type: Interventional

Funder types: Other

Identifiers

NCT00103792

WG-MMF-1

UMCG-ANCA-MMF-1

Details and patient eligibility

About

The purpose of this study is to determine the efficacy and safety of a new drug, mycophenolate mofetil, for the treatment of relapses of ANCA-associated vasculitis (Wegener's granulomatosis or microscopic polyangiitis). Therefore, we compare the standard therapy with cyclophosphamide to mycophenolate mofetil.

The investigators expect mycophenolate mofetil to be less toxic and almost equally effective as cyclophosphamide.

Full description

Treatment of ANCA-associated vasculitis consists of two phases: remission induction with highly effective, but also relatively toxic drugs, and, secondly, after remission is achieved, maintenance therapy with less toxic drugs. The standard induction therapy of a relapse of Wegener's granulomatosis or microscopic polyangiitis consists of the combination of cyclophosphamide and prednisolone. Although this induction therapy is very effective, it is very toxic as well.

Searching for an alternative for cyclophosphamide, we will test the efficacy and safety of a new combination therapy with mycophenolate mofetil and prednisolone. We will compare the effect and safety of the standard induction therapy with the new therapy. When relapses occur, patients will be randomized for either the standard therapy with cyclophosphamide or for mycophenolate mofetil.

Enrollment: 84 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• First or second relapse ANCA-associated vasculitis
• PR3- or MPO-ANCA antibodies present or histological proof of relapse
• Adult

Exclusion criteria

• Severe alveolar bleeding or (imminent) respiratory failure
• Renal failure (serum creatinine >500 umol/L or dialysis)
• Maintenance therapy before start of study consisting of: cyclophosphamide > 100 mg/day or prednisolone >25 mg/day
• Intolerance or allergy for cyclophosphamide, mycophenolate mofetil or azathioprine
• Gravidity or inadequate anticonception

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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84 participants in 2 patient groups

1 MMF induction

Experimental group

Description:

mycophenolate and steroids as remission induction, followed by azathioprine as maintenance therapy

Treatment:

Drug: mycophenolate mofetil

2 CYC induction

Active Comparator group

Description:

cyclophosphamide and steroids, followed by maintenance therapy (azathioprine)

Treatment:

Drug: cyclophosphamide