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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified February 2009 by University Medical Center Groningen.
Recruitment status was  Recruiting
Information provided by:
University Medical Center Groningen Identifier:
First received: February 14, 2005
Last updated: February 12, 2009
Last verified: February 2009

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.

Condition Intervention Phase
Wegener's Granulomatosis
Drug: mycophenolate mofetil
Drug: cyclophosphamide
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Comparative Study of the Efficacy of Induction Therapy With Cyclophosphamide or Mycophenolate Mofetil for Non-Life-Threatening Relapses of PR3- or MPO-ANCA Associated Vasculitis

Resource links provided by NLM:

Further study details as provided by University Medical Center Groningen:

Primary Outcome Measures:
  • remission induction rate [ Time Frame: 6 months ] [ Designated as safety issue: No ]
  • disease free survival after 2 and 4 years [ Time Frame: 2 and 4 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • time to remission [ Time Frame: 9 months ] [ Designated as safety issue: No ]
  • cumulative organ damage [ Time Frame: 4 years ] [ Designated as safety issue: No ]
  • side-effects [ Time Frame: 4 years ] [ Designated as safety issue: No ]
  • ANCA titres over time [ Time Frame: 4 years ] [ Designated as safety issue: No ]

Estimated Enrollment: 90
Study Start Date: December 2004
Estimated Study Completion Date: January 2012
Estimated Primary Completion Date: January 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
mycophenolate and steroids as remission induction, followed by azathioprine maintenance therapy
Drug: mycophenolate mofetil
2000 mg mycophenolate per day combined with steroids for induction remission, followed by azathioprine standard maintenance therapy
Active Comparator: 2
Drug: cyclophosphamide
2 mg/kg/d, combined with steroids, for remission induction, followed by standard azathioprine maintenance therapy

Detailed 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.


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00103792

Contact: Patricia M. Stassen, M.D., Ph.D. +31433876543
Contact: Coen A. Stegeman, M.D., Ph.D. +31503616161

University Medical Centre Groningen Recruiting
Groningen, Netherlands, 9700 RB
Contact: Patricia Stassen, M.D.    +31503611295   
Contact: Coen Stegeman, M.D., Ph. D.    +31503616161   
Sub-Investigator: Patricia Stassen, M.D. pH.D.         
Sponsors and Collaborators
University Medical Center Groningen
Principal Investigator: Coen Stegeman, MD PhD UMCG Groningen
  More Information

Stegeman CA; Cohen Tervaert JW. Mycophenolate mofetil for remission induction in patients with active Wegener's Granulomatosis (WG) intolerant for cyclophosphamide. J Am Soc Nephrol(11):98A, 2000

Responsible Party: University Medical Center Groningen, Dept of Nephrology Identifier: NCT00103792     History of Changes
Other Study ID Numbers: WG-MMF-1  UMCG-ANCA-MMF-1 
Study First Received: February 14, 2005
Last Updated: February 12, 2009
Health Authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)

Keywords provided by University Medical Center Groningen:
Induction therapy
ANCA-associated vasculitis
Wegener's granulomatosis
microscopic polyangiitis
mycophenolate mofetil

Additional relevant MeSH terms:
Granulomatosis with Polyangiitis
Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
Microscopic Polyangiitis
Autoimmune Diseases
Brain Diseases
Cardiovascular Diseases
Central Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Immune System Diseases
Lung Diseases
Lung Diseases, Interstitial
Nervous System Diseases
Respiratory Tract Diseases
Systemic Vasculitis
Vascular Diseases
Mycophenolate mofetil
Mycophenolic Acid
Alkylating Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antineoplastic Agents, Alkylating
Antirheumatic Agents
Enzyme Inhibitors
Immunologic Factors
Immunosuppressive Agents
Molecular Mechanisms of Pharmacological Action
Myeloablative Agonists processed this record on May 24, 2016