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CompletedNCT00103792Updated Aug 27, 2024

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

A Phase 3 interventional study of mycophenolate mofetil and cyclophosphamide in Wegener's Granulomatosis and Vasculitis, sponsored by University Medical Center Groningen. Completed at 1 site in Netherlands. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-08-27.

Sponsored by University Medical Center Groningen · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
84
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study summary

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.

Read the 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.

02

Conditions studied

  • Wegener's Granulomatosis
  • Vasculitis

Keywords

  • Induction therapy
  • ANCA-associated vasculitis
  • Wegener's granulomatosis
  • microscopic polyangiitis
  • mycophenolate mofetil
  • cyclophosphamide
03

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
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

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
04

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
84 participants (actual)

Study arms

  • Experimental
    1 MMF induction

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

    Drug: mycophenolate mofetil

  • Active comparator
    2 CYC induction

    cyclophosphamide and steroids, followed by maintenance therapy (azathioprine)

    Drug: cyclophosphamide

Interventions

  • Drugmycophenolate mofetil

    2000 mg mycophenolate mofetil per day combined with steroids for induction remission, followed by azathioprine standard maintenance therapy

  • Drugcyclophosphamide

    2 mg/kg/d, combined with steroids, for remission induction, followed by standard azathioprine maintenance therapy

05

What researchers measure

Primary outcomes

  1. remission induction rate

    Time frame: 6 months

  2. disease free survival after 2 and 4 years

    Time frame: 2 and 4 years

Secondary outcomes

  1. time to remission

    Time frame: 9 months

  2. cumulative organ damage

    Time frame: 4 years

  3. side-effects

    Time frame: 4 years

  4. ANCA titres over time

    Time frame: 4 years

06

Study locations

1 site
  • University Medical Centre Groningen
    Groningen, 9700 RB, Netherlands
07

References and documents

Publications

  • 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

Individual participant data

Plan to share: Undecided

08

Registry details

Key details

Study ID
NCT00103792
Lead sponsor
University Medical Center Groningen
Responsible party
Sponsor
First posted
Feb 15, 2005
Start date
Dec 2004
Primary completion
Dec 2014
Completion
Jan 2015
Last update
Aug 27, 2024

Study contacts

Coen Stegeman, MD PhD
principal investigator · UMCG Groningen

Oversight

Data monitoring committee
No
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