NCT02994927

Brief Summary

The primary objective is to evaluate the efficacy of CCX168 (avacopan) to induce and sustain remission in patients with active anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV), when used in combination with cyclophosphamide followed by azathioprine, or in combination with rituximab.

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
331

participants targeted

Target at P50-P75 for phase_3

Timeline
Completed

Started Mar 2017

Typical duration for phase_3

Geographic Reach
19 countries

183 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 11, 2016

Completed
5 days until next milestone

First Posted

Study publicly available on registry

December 16, 2016

Completed
3 months until next milestone

Study Start

First participant enrolled

March 15, 2017

Completed
2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 7, 2019

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2019

Completed
2.9 years until next milestone

Results Posted

Study results publicly available

September 9, 2022

Completed
Last Updated

March 24, 2025

Status Verified

March 1, 2025

Enrollment Period

2.5 years

First QC Date

December 11, 2016

Results QC Date

May 19, 2022

Last Update Submit

March 4, 2025

Conditions

Keywords

ANCA-associated vasculitiscomplementvasculitisC5aRavacopanCCX168MPAGPA

Outcome Measures

Primary Outcomes (2)

  • Percentage of Subjects Achieving Disease Remission at Week 26

    Disease remission at Week 26 was defined as: * Achieving a BVAS of 0 as determined by the Adjudication Committee; * No administration of glucocorticoids given for ANCA-associated vasculitis within 4 weeks prior to Week 26; * No BVAS \>0 during the 4 weeks prior to Week 26 (if collected for an unscheduled assessment).

    Week 26

  • Percentage of Subjects Achieving Sustained Disease Remission at Week 52

    Sustained remission at Week 52 was defined as: * Disease remission at Week 26 as defined above; * Disease remission at Week 52 defined as a BVAS of 0 at Week 52 as determined by the Adjudication Committee and no administration of glucocorticoids for treatment of ANCA-associated vasculitis within 4 weeks prior to Week 52; * No disease relapse between Week 26 and Week 52 as determined by the Adjudication Committee.

    Week 52

Secondary Outcomes (26)

  • Subject Incidence of Treatment-emergent SAEs, AEs, and Withdrawals Due to AEs

    From day 1 throughout the study period (day 421/week 60)

  • Glucocorticoid-induced Toxicity as Measured by Change From Baseline Over the First 26 Weeks in the GTI

    Baseline, Week 13 and 26

  • Percentage of Participants With BVAS of 0 at Week 4, Regardless of Whether the Subjects Received Glucocorticoids During This Period of Time and Based on Assessment by the Blinded AC

    Week 4

  • Change From Baseline Over 52 Weeks in Health-related Quality of Life as Measured by the Domains and Component Scores of the SF-36v2 and EQ-5D-5L VAS and Index

    Baseline, Week 26 and 52

  • Percentage of Subjects and Time to Experiencing a Relapse After Previously Achieving Remission at Week 26 in the Study

    Week 52

  • +21 more secondary outcomes

Study Arms (2)

Prednisone group

ACTIVE COMPARATOR

Avacopan-matching placebo plus cyclophosphamide/azathioprine or rituximab plus a full starting dose of prednisone.

Drug: PrednisoneDrug: CyclophosphamideBiological: RituximabDrug: Azathioprine

Avacopan group

EXPERIMENTAL

Avacopan plus cyclophosphamide/azathioprine or rituximab plus prednisone-matching placebo.

Drug: AvacopanDrug: CyclophosphamideBiological: RituximabDrug: Azathioprine

Interventions

Avacopan 30 mg twice daily orally for 52 weeks (364 days): \- Three 10 mg avacopan capsules in the morning, preferably with food, and three in the evening, preferably with food, approximately 12 hours after the morning dose. Oral prednisone-matching placebo tapering regimen over 20 weeks (140 days): * Prednisone-matching placebo capsules equivalent to 60 mg per day if the subject's body weight was ≥55 kg, or 45 mg per day if the subject's body weight was \<55 kg, starting on Day 1 with tapering according to a protocol-specified schedule. * Adolescents who weighed ≤37 kg started at a prednisone-matching placebo dose of 30 mg per day.

Also known as: CCX168
Avacopan group

Avacopan-matching placebo twice daily orally for 52 weeks (364 days): \- Three avacopan-matching placebo capsules in the morning, preferably with food, and three in the evening, preferably with food, approximately 12 hours after the morning dose. Oral prednisone tapering regimen over 20 weeks (140 days): * Prednisone 60 mg per day if the subject's body weight was ≥55 kg, or 45 mg per day if the subject's body weight was \<55 kg, starting on Day 1 with tapering according to the protocol-specified schedule. * Adolescents who weighed ≤37 kg started at a prednisone dose of 30 mg per day.

Prednisone group

Orally or intravenously administered

Avacopan groupPrednisone group
RituximabBIOLOGICAL

Intravenously administered

Avacopan groupPrednisone group

Orally administered

Avacopan groupPrednisone group

Eligibility Criteria

Age12 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Clinical diagnosis of granulomatosis with polyangiitis (Wegener's) or microscopic polyangiitis
  • Male and female subjects, aged at least 18 years, with newly-diagnosed or relapsed associated vasculitis (AAV) where treatment with cyclophosphamide or rituximab is needed; where approved by Regulatory Agencies, adolescents (12-17 year old) may be enrolled
  • Use of adequate contraception
  • Positive test for anti-proteinase 3 (PR3) or anti-myeloperoxidase (MPO)
  • At least 1 major item, or at least 3 non-major items, or at least the 2 renal items of proteinuria and hematuria on Birmingham Vasculitis Activity Score (BVAS)
  • Estimated glomerular filtration rate ≥15 mL/minute/1.73 m\^2 at screening

You may not qualify if:

  • Pregnant or breast-feeding
  • Alveolar hemorrhage requiring pulmonary ventilation support at screening
  • Any other known multi-system autoimmune disease
  • Required dialysis or plasma exchange within 12 weeks prior to screening
  • Have a kidney transplant
  • Received cyclophosphamide within 12 weeks prior to screening; if on azathioprine, mycophenolate mofetil or methotrexate at the time of screening, these drugs must be withdrawn prior to receiving the cyclophosphamide or rituximab dose on Day 1
  • Received intravenous glucocorticoids, \>3000 mg methylprednisolone equivalent, within 4 weeks prior to screening
  • Have been taking an oral daily dose of a glucocorticoid of more than 10 mg prednisone-equivalent for more than 6 weeks continuously prior to screening
  • Received rituximab or other B-cell antibody within 52 weeks of screening or 26 weeks provided B cell reconstitution has occurred (i.e., CD19 count \> 0.01x10\^9/L); received anti-tumor necrosis factor (TNF) treatment, abatacept, alemtuzumab, intravenous immunoglobulin (IVIg), belimumab, tocilizumab, or eculizumab within 12 weeks prior to screening
  • For patients scheduled to receive cyclophosphamide treatment, urinary outflow obstruction, active infection (especially varicella zoster infection), or platelet count \<50,000/μL before start of dosing
  • Participated previously in a CCX168 study

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (198)

Clinical Trial Site

Huntsville, Alabama, 35801, United States

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Phoenix, Arizona, 85012, United States

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Phoenix, Arizona, 85032, United States

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Los Angeles, California, 90048, United States

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Santa Monica, California, 90404, United States

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Aurora, Colorado, 80045, United States

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Washington D.C., District of Columbia, 20007, United States

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Daytona Beach, Florida, 32117, United States

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Tampa, Florida, 33612, United States

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Atlanta, Georgia, 30322, United States

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Meridian, Idaho, 83605, United States

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Chicago, Illinois, 60637, United States

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Indianapolis, Indiana, 46202, United States

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Kansas City, Kansas, 66160, United States

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Lexington, Kentucky, 40536, United States

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Shreveport, Louisiana, 71101, United States

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Baltimore, Maryland, 21224, United States

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Boston, Massachusetts, 02114, United States

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Boston, Massachusetts, 02118, United States

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Ann Arbor, Michigan, 48109, United States

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Minneapolis, Minnesota, 55414, United States

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Rochester, Minnesota, 55905, United States

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St Louis, Missouri, 63110, United States

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Great Neck, New York, 11021, United States

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Mineola, New York, 11501, United States

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New York, New York, 10021, United States

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New York, New York, 10032, United States

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Chapel Hill, North Carolina, 27599, United States

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Greenville, North Carolina, 27834, United States

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Winston-Salem, North Carolina, 27103, United States

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Cleveland, Ohio, 44195, United States

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Columbus, Ohio, 43210, United States

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Duncansville, Pennsylvania, 16635, United States

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Philadelphia, Pennsylvania, 19104, United States

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Pittsburgh, Pennsylvania, 15225, United States

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Providence, Rhode Island, 02903, United States

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Charleston, South Carolina, 29406, United States

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Dallas, Texas, 75235, United States

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Houston, Texas, 77030, United States

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Salt Lake City, Utah, 84132, United States

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Seattle, Washington, 98101, United States

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Adelaide, Australia

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Auchenflower, Australia

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Brisbane, Australia

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Clayton, Australia

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Concord, Australia

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Heidelberg, Australia

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Liverpool, Australia

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Nambour, Australia

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Nedlands, Australia

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Randwick, Australia

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Saint Albans, Australia

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Southport, Australia

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St Leonards, Australia

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Westmead, Australia

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Woolloongabba, Australia

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Feldkirch, Austria

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Graz, Austria

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Innsbruck, Austria

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Antwerp, Belgium

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Brussels, Belgium

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Leuven, Belgium

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Liège, Belgium

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Calgary, Canada

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Greenfield Park, Canada

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Hamilton, Canada

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Montreal, Canada

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Québec, Canada

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Sherbrooke, Canada

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Toronto, Canada

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Vancouver, Canada

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Olomouc, Czechia

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Prague, Czechia

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Aalborg, Denmark

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Aarhus, Denmark

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Copenhagen, Denmark

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Odense, Denmark

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Roskilde, Denmark

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Angers, France

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Bordeaux, France

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Boulogne-sur-Mer, France

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Brest, France

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Bron, France

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Caen, France

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Colmar, France

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Grenoble, France

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Marseille, France

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Metz, France

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Nantes, France

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Nîmes, France

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Paris, France

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Toulouse, France

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Valenciennes, France

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Aachen, Germany

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Bad Bramstedt, Germany

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Berlin, Germany

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Cologne, Germany

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Dresden, Germany

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Essen, Germany

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Freiburg im Breisgau, Germany

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Fulda, Germany

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Hamburg, Germany

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Hanover, Germany

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Heidelberg, Germany

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Jena, Germany

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Kirchheim unter Teck, Germany

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Leipzig, Germany

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Ludwigshafen, Germany

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Lübeck, Germany

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Mannheim, Germany

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Munich, Germany

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Tübingen, Germany

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Villingen-Schwenningen, Germany

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Budapest, Hungary

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Debrecen, Hungary

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Cork, Ireland

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Dublin, Ireland

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Ancona, Italy

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Florence, Italy

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Genova, Italy

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Milan, Italy

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Monza, Italy

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Parma, Italy

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Reggio Emilia, Italy

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Torino, Italy

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Udine, Italy

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Aichi, Japan

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Akita, Japan

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Chiba, Japan

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Hiroshima, Japan

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Hokkaido, Japan

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Ishikawa, Japan

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Kagawa, Japan

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Kanagawa, Japan

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Kobe, Japan

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Miyazaki, Japan

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Nagoya, Japan

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Okayama, Japan

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Osaka, Japan

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Saitama, Japan

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Shimane, Japan

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Shizuoka, Japan

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Tokyo, Japan

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Toyama, Japan

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Yokohama, Japan

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Groningen, Netherlands

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Leiden, Netherlands

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Rotterdam, Netherlands

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Christchurch, New Zealand

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Dunedin, New Zealand

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Grafton, New Zealand

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Hamilton, New Zealand

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Takapuna, New Zealand

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Nordbyhagen, Norway

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Oslo, Norway

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Tromsø, Norway

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Badalona, Spain

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Barcelona, Spain

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Burela de Cabo, Spain

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Lleida, Spain

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Madrid, Spain

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San Sebastián, Spain

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Linköping, Sweden

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Lund, Sweden

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Örebro, Sweden

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Stockholm, Sweden

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Uppsala, Sweden

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Basel, Switzerland

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Bern, Switzerland

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Fribourg, Switzerland

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Lausanne, Switzerland

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Sankt Gallen, Switzerland

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Zurich, Switzerland

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Aberdeen, United Kingdom

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Basildon, United Kingdom

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Birmingham, United Kingdom

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Bristol, United Kingdom

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Cambridge, United Kingdom

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Canterbury, United Kingdom

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Cardiff, United Kingdom

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Carshalton, United Kingdom

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Dorchester, United Kingdom

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Dudley, United Kingdom

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Exeter, United Kingdom

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Glasgow, United Kingdom

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Inverness, United Kingdom

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Kirkcaldy, United Kingdom

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Leeds, United Kingdom

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Leicester, United Kingdom

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Liverpool, United Kingdom

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London, United Kingdom

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Manchester, United Kingdom

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Newcastle, United Kingdom

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Nottingham, United Kingdom

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Oxford, United Kingdom

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Reading, United Kingdom

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Salford, United Kingdom

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Westcliff-on-Sea, United Kingdom

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Related Publications (5)

  • Geetha D, Neumann T, Karras A, Cid MC, Merkel PA, Bray S, Bozeman AM, Jayne DRW; Members of the ADVOCATE Study Group. Efficacy and safety of avacopan for treatment of patients with ANCA-associated vasculitis receiving cyclophosphamide. RMD Open. 2025 Oct 5;11(4):e005743. doi: 10.1136/rmdopen-2025-005743.

  • Geetha D, Dua A, Yue H, Springer J, Salvarani C, Jayne D, Merkel P; ADVOCATE Study Group. Efficacy and safety of avacopan in patients with ANCA-associated vasculitis receiving rituximab in a randomised trial. Ann Rheum Dis. 2024 Jan 11;83(2):223-232. doi: 10.1136/ard-2023-224816.

  • Soulsby WD. Journal Club Review of "Avacopan for the Treatment of ANCA-Associated Vasculitis". ACR Open Rheumatol. 2022 Jul;4(7):558-561. doi: 10.1002/acr2.11412. Epub 2022 Feb 15.

  • Jayne DRW, Merkel PA, Schall TJ, Bekker P; ADVOCATE Study Group. Avacopan for the Treatment of ANCA-Associated Vasculitis. N Engl J Med. 2021 Feb 18;384(7):599-609. doi: 10.1056/NEJMoa2023386.

  • Merkel PA, Jayne DR, Wang C, Hillson J, Bekker P. Evaluation of the Safety and Efficacy of Avacopan, a C5a Receptor Inhibitor, in Patients With Antineutrophil Cytoplasmic Antibody-Associated Vasculitis Treated Concomitantly With Rituximab or Cyclophosphamide/Azathioprine: Protocol for a Randomized, Double-Blind, Active-Controlled, Phase 3 Trial. JMIR Res Protoc. 2020 Apr 7;9(4):e16664. doi: 10.2196/16664.

MeSH Terms

Conditions

Anti-Neutrophil Cytoplasmic Antibody-Associated VasculitisVasculitis

Interventions

avacopanPrednisoneCyclophosphamideRituximabAzathioprine

Condition Hierarchy (Ancestors)

Systemic VasculitisVascular DiseasesCardiovascular DiseasesSkin Diseases, VascularSkin DiseasesSkin and Connective Tissue DiseasesAutoimmune DiseasesImmune System Diseases

Intervention Hierarchy (Ancestors)

PregnadienediolsPregnadienesPregnanesSteroidsFused-Ring CompoundsPolycyclic CompoundsPhosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus CompoundsAntibodies, Monoclonal, Murine-DerivedAntibodies, MonoclonalAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsThionucleosidesSulfur CompoundsMercaptopurinePurinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic CompoundsNucleosidesNucleic Acids, Nucleotides, and Nucleosides

Results Point of Contact

Title
Clinical trial disclosure
Organization
ChemoCentryx, Inc.

Study Officials

  • MD

    Amgen

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Masking Details
This study was double-blind, double-dummy, i.e., placebo capsules were identical in appearance to the avacopan capsules, and prednisone capsules also had matching placebo capsules. To maintain the blind, multiple measures were taken (i.e., randomization code was not accessible to study personnel who had contact with study centers or who were involved in data management and analysis for the duration of the study).
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 11, 2016

First Posted

December 16, 2016

Study Start

March 15, 2017

Primary Completion

September 7, 2019

Study Completion

November 1, 2019

Last Updated

March 24, 2025

Results First Posted

September 9, 2022

Record last verified: 2025-03

Data Sharing

IPD Sharing
Will share

De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request

Shared Documents
STUDY PROTOCOL, SAP, ICF, CSR
Time Frame
Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication (or other new use) have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
Access Criteria
Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors, and if not approved, may be further arbitrated by a Data Sharing Independent Review Panel. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below.
More information

Locations