MPO-ANCA VasculitisPhase 1NCT00405860
CellCept in p-ANCA Vasculitis
A Phase 1 study testing CellCept (mycophenolate mofetil) in people with MPO-ANCA Vasculitis and Microscopic Polyangiitis, run by Mayo Clinic at 1 site in United States.
Trial summary
Microscopic polyangiitis (MP) is a primary systemic vasculitis predominantly affecting small blood vessels. Following the widespread introduction of ANCA testing, the primary systemic vasculitis (SV), Wegener?s granulomatosis (WG) and microscopic polyangiitis (MP) appear to be more frequent than was previously thought… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- MPO-ANCA Vasculitis, Microscopic Polyangiitis
- What is being tested?
- CellCept (mycophenolate mofetil)
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2002to Jul 2008
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
- Active microscopic polyangiitis
- Active urinary sediment (>25 rbc/hpf, red cell casts or dysmorphic red cells)
- Renal biopsy compatible with the diagnosis of microscopic polyangiitis, or diagnosis demonstrated by the presence of hematuria, proteinuria, and dysmorphic red blood cells, and / or red blood casts when biopsy is contraindicated.
- Positive p-ANCA (MPO ELISA)
- Serum creatinine < 3.0mg/dl.
- Age 18 years or over.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Pilot Study of Mycophenolate Mofetil (MMF) in Patients With p-ANCA Microscopic Polyangiitis and Mild to Moderate Renal Dysfunction.
Microscopic polyangiitis (MP) is a primary systemic vasculitis predominantly affecting small blood vessels. Following the widespread introduction of ANCA testing, the primary systemic vasculitis (SV), Wegener?s granulomatosis (WG) and microscopic polyangiitis (MP) appear to be more frequent than was previously thought (see definitions in Appendix 6). In addition, the existence of early and organ-limited forms of these diseases, such as renal-limited vasculitis (RLV) is now clearly recognized. Their annual incidence exceeds 20 per million per year and they account for at least 5 % of the causes of end stage renal failure. The two diseases share many features of their histology, serology and response to treatment, pointing to similarities in their pathogenesis, which have justified a common approach to their management. The standard treatment with corticosteroids (CS) and cyclophosphamide (CYC) is usually effective at controlling active disease but continued treatment is necessary to prevent disease relapse. Due to the cumulative toxicity associated with CYC treatment, alternatives have been looked for. Mycophenolate mofetil (MMF) has been used to treat patients with a variety of immune-mediated nephritides, including ANCA-associated vasculitis, with less toxicity than CYC but with variable outcome. The present trial will examine whether substitution of oral CYC with oral MMF is equally efficient for induction of remission with less adverse effects in cases of MP with mild to moderate renal involvement. All patients will receive the same regimen of oral prednisone + MMF. Prednisone will be tapered to a stop after 24 weeks but MMF will continue for a total of 18 months unless there is worsening or persistent disease. The trial ends after 18 months.
Read the detailed description
- Patients will receive I.V. methylprednisone, or I.V. dexamethazone, oral prednisone and oral MMF therapy as outlined in table 2.
- MMF will be initiated within the first 1-2 weeks of the start of steroids. Patients will receive CellCept, 750 mg po b.i.d for the first week. Dose will be increased to 1000 mg po b.i.d for the second week, and thereafter, according to blood levels and patient tolerance. Target blood levels are 1 ? 3.5 \g/ml. Treatment will be for a total of 18 months. This is based on the published dose-dependent adverse effect profiles in transplant patients (31-32) and on reports that lower doses are ineffective and shorter courses (less then 6 months) result in relapses or failure of therapy (25). Dose will be reduced in patient who can not tolerate MMF at the above dose.
- Relapse treatment to follow guidelines for relapse regimens. 3) After 18 months, all medications will be tapered to a full stop unless disease is active or grumbling.
- Pneumocystis pneumonia prophylaxis will be used during the trial (with sulfamethoxazole/trimethoprim, or Dapsone or Mepron if allergic to sulfa).
- Conditions
- MPO-ANCA Vasculitis, Microscopic Polyangiitis
- Being tested
- CellCept (mycophenolate mofetil)
- Medical subject headings
- Microscopic Polyangiitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CellCept (mycophenolate mofetil)Drug
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary endpoint is successful induction of remission as defined in Appendix 6 within 6 months.
Secondary outcomes (3)
Major relapse necessitating a switch to induction OCS/CYC treatment or more aggressive treatment (e.g. plasma exchange).
Minor relapses that can effectively be controlled by a transient, non-toxic increase in OCS
Intolerance to trial medications and adverse effects. Adverse effects will be monitored
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Nov 2006 (estimated)
- Study startDec 2002
- Primary completionJul 2008
- Study completionJul 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for MPO-ANCA VasculitisStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Collaborators
- Roche Pharma AG
- Investigators
- Fernando C. Fervenza, M.D., Ph.D., Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00405860
- Sponsor's protocol ID
- 1679-02
- Status last verified
- Mar 2011
- First submitted
- 29 Nov 2006
- Last updated
- 23 Mar 2011
- Results posted
- No
Publications
Silva F, Specks U, Kalra S, Hogan MC, Leung N, Sethi S, Fervenza FC. Mycophenolate mofetil for induction and maintenance of remission in microscopic polyangiitis with mild to moderate renal involvement--a prospective, open-label pilot trial. Clin J Am Soc Nephrol. 2010 Mar;5(3):445-53. doi: 10.2215/CJN.06010809. Epub 2010 Jan 21.
PubMed 20093349
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Mycophenolate Mofetil (MMF) in Patients With p-ANCA Microscopic Polyangiitis and Mild to Moderate Renal Dysfunction.
- ClinicalTrials.gov ID
- NCT00405860
- Acronym / sponsor ID
- 1679-02
- Conditions
- MPO-ANCA Vasculitis, Microscopic Polyangiitis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Collaborators
- Roche Pharma AG
- Study start
- Dec 2002
- Primary completion
- Jul 2008 (actual)
- Study completion
- Jul 2008 (actual)
- First posted
- 30 Nov 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Microscopic polyangiitis (MP) is a primary systemic vasculitis predominantly affecting small blood vessels. Following the widespread introduction of ANCA testing, the primary systemic vasculitis (SV), Wegener?s granulomatosis (WG) and microscopic polyangiitis (MP) appear to be more frequent than was previously thought (see definitions in Appendix 6). In addition, the existence of early and organ-limited forms of these diseases, such as renal-limited vasculitis (RLV) is now clearly recognized. Their annual incidence exceeds 20 per million per year and they account for at least 5 % of the causes of end stage renal failure. The two diseases share many features of their histology, serology and response to treatment, pointing to similarities in their pathogenesis, which have justified a common approach to their management. The standard treatment with corticosteroids (CS) and cyclophosphamide (CYC) is usually effective at controlling active disease but continued treatment is necessary to prevent disease relapse. Due to the cumulative toxicity associated with CYC treatment, alternatives have been looked for. Mycophenolate mofetil (MMF) has been used to treat patients with a variety of immune-mediated nephritides, including ANCA-associated vasculitis, with less toxicity than CYC but with variable outcome. The present trial will examine whether substitution of oral CYC with oral MMF is equally efficient for induction of remission with less adverse effects in cases of MP with mild to moderate renal involvement. All patients will receive the same regimen of oral prednisone + MMF. Prednisone will be tapered to a stop after 24 weeks but MMF will continue for a total of 18 months unless there is worsening or persistent disease. The trial ends after 18 months.
Detailed description
- Patients will receive I.V. methylprednisone, or I.V. dexamethazone, oral prednisone and oral MMF therapy as outlined in table 2.
- MMF will be initiated within the first 1-2 weeks of the start of steroids. Patients will receive CellCept, 750 mg po b.i.d for the first week. Dose will be increased to 1000 mg po b.i.d for the second week, and thereafter, according to blood levels and patient tolerance. Target blood levels are 1 ? 3.5 \g/ml. Treatment will be for a total of 18 months. This is based on the published dose-dependent adverse effect profiles in transplant patients (31-32) and on reports that lower doses are ineffective and shorter courses (less then 6 months) result in relapses or failure of therapy (25). Dose will be reduced in patient who can not tolerate MMF at the above dose.
- Relapse treatment to follow guidelines for relapse regimens. 3) After 18 months, all medications will be tapered to a full stop unless disease is active or grumbling.
- Pneumocystis pneumonia prophylaxis will be used during the trial (with sulfamethoxazole/trimethoprim, or Dapsone or Mepron if allergic to sulfa).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CellCept (mycophenolate mofetil) | Drug | – |
Primary outcome
The primary endpoint is successful induction of remission as defined in Appendix 6 within 6 months.
3 secondary outcomes
Major relapse necessitating a switch to induction OCS/CYC treatment or more aggressive treatment (e.g. plasma exchange).
Minor relapses that can effectively be controlled by a transient, non-toxic increase in OCS
Intolerance to trial medications and adverse effects. Adverse effects will be monitored
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2011.
- Derived from this study: PubMed 20093349Silva F, Specks U, Kalra S, Hogan MC, Leung N, Sethi S, Fervenza FC. Mycophenolate mofetil for induction and maintenance of remission in microscopic polyangiitis with mild to moderate renal involvement--a prospective, open-label pilot trial. Clin J Am Soc Nephrol. 2010 Mar;5(3):445-53. doi: 10.2215/CJN.06010809. Epub 2010 Jan 21.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Fernando C. Fervenza, M.D., Ph.D. principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CellCept (mycophenolate mofetil) in people with MPO-ANCA Vasculitis and Microscopic Polyangiitis, run by Mayo Clinic at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Mar 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The primary endpoint is successful induction of remission as defined in Appendix 6 within 6 months.. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00405860, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Mar 2011; AzTrials last refreshed its copy on 31 Oct 2026.