Avacopan vs Reduced-dose Glucocorticoids in ANCA-associated Vasculitis

NCT ID: NCT06611696

Last Updated: 2025-05-04

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE4

Total Enrollment

160 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-11-15

Study Completion Date

2028-09-30

Brief Summary

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The goal of this clinical trial is to learn if avacopan in combination with short-term (4 weeks) reduced-dose glucocorticoid and rituximab works to treat patients with newly-onset ANCA-associated vasculitis. It will also learn about the long-term safety of avacopan. The main questions it aims to answer are:

Is avacopan in combination with short-term reduced-dose glucocorticoid and rituximab as effective as the combination of 20 week reduced-dose glucocorticoid and rituximab in the proportion of the patients achieving remission? Does avacopan lower the relapse rate compared to the 6 monthly rituximab maintenance therapy? What medical problems do participants have when taking long-term avacopan?

Participants will:

Be treated with avacopan in combination with short-term (until 4 weeks) reduced-dose glucocorticoid and rituximab (at 0 week) or reduced-dose glucocorticoid (until 20 weeks) and rituximab (at 0, 26, 52 and 78 weeks).

Be assessed at 0, 4, 8, 16, 26, 52, 78 and 104 weeks regarding disease status (remission/relapse), disease activity by Birmingham Vasculitis Activity Score ver3, disease damage by Vasculitis Damage Index and adverse events.

The primary endpoint is remission rates at 26 weeks.

Detailed Description

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Anti-neutrophil cytoplasm antibody (ANCA)-associated vasculitis is characterized by a small to medium-size vasculitis and the presence of ANCA. ANCA-associated vasculitis includes microscopic polyangiitis, granulomatosis with polyangiitis and eosinophilic granulomatosis with polyangiitis. ANCA-associated vasculitis can be a life-threatening disease and the mortality is 80% at 1 year in untreated patients. In 2010s, standard therapies for remission induction of ANCA-associated vasculitis were the combination of high-dose glucocorticoids and either cyclophosphamide or rituximab. Although those therapies have high remission rates of 80-90%, mortality at 5 years is still high at 10-20% mainly due to treatments-related adverse events.

In the LoVAS trial (2021, JAMA), the combination of reduced-dose glucocorticoid and rituximab showed non-inferiority to high-dose glucocorticoid and rituximab in remission rates at 6 months. In addition, adverse events were dramatically less in the reduced-dose group than in the high-dose group.

In the ADVOCATE trial (2021, NEJM), the combination of avacopan, newly developed complement C5a inhibitor, and rituximab or cyclophosphamide showed non-inferiority to high-dose glucocorticoid and rituximab or cyclophosphamide in remission rates at 6 months. The avacopan group was allowed to use glucocorticoid within 1 month from the trial entry, and over 80% of patients used glucocorticoid indeed. Regarding adverse events, they were less in the avacopan group than in the glucocorticoid group.

Although both the reduced-dose glucocorticoid regimen in the LoVAS trial and the avacopan regimen in the ADVOCATE trial are effective and safe for patients with ANCA-associated vasculitis, there is no trial directly comparing both regimens at the moment. Thus, in this multicenter, open-label, randomized, non-ineriority, phase 4 trial, the investigators aim to investigate if the combination of avacoapn, short-term (4 weeks) reduced-dose glucocorticoid and rituximab is non-inferior to the combination of reduced-dose glucocorticoid (20 weeks) and rituximab. The investigators also compare safety profiles and disease relapse between the two groups. A total of 160 patients with new-onset ANCA-associated vasculitis (microscopic polyangiitis and granulomatosis with polyangiitis) will be recruited and randomized to the two treatments groups. The primary end point is remission rate at 26 weeks, and the patients will be followed until 104 weeks for assessing disease relapse and long-term safety.

Conditions

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ANCA Associated Vasculitis (AAV)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Glucocorticoid group

Prednisolone will be commenced with dose of 0.5mg/kg/day and will be tapered and off within 5 months. If a patient fails to achieve BVAS=0 or normalization of CRP levels or normalization of ANCA levels, an investigator can keep 5mg/day of prednisolone and postpone the procedure of stopping prednisolone. Patients will also receive rituximab (375mg/m2/w x4).

During remission maintenance phase (6-24 months),, patients will receive rituximab (500mg/body) every 6 months as remission maintenance therapy.

In the case of inadequate response to the combination therapy of prednisolone and rituximab, additional administration of prednisolone 20mg/day (less than 2 weeks) can be allowed as the rescue therapy.

During remission maintenance phase, in the case of minor relapse, additional administration of prednisolone 20mg/day (less than 2 weeks) can be allowed as the rescue therapy. Minor relapse is defined as relapse with no major BVAS item.

Group Type ACTIVE_COMPARATOR

Prednisolone and rituximab

Intervention Type DRUG

Patients in the glucocorticoid arm will be treated with reduced-dose prednisolone and rituximab.

Avacopan group

Prednisolone will be commenced with dose of 0.5mg/kg/day and will be tapered and off within 1 months. Patients will also receive avacopan (60mg/day) and rituximab (375mg/m2/w x4).

During remission maintenance phase (6-24 months), patients will receive avacopan (60mg/day) as remission maintenance therapy until the trial end.

In the case of inadequate response to the combination therapy of avacopan, prednisolone and rituximab, additional administration of prednisolone 20mg/day (less than 2 weeks) can be allowed as the rescue therapy.

During remission maintenance phase, in the case of minor relapse, additional administration of prednisolone 20mg/day (less than 2 weeks) can be allowed as the rescue therapy. Minor relapse is defined as relapse with no major BVAS item.

Group Type EXPERIMENTAL

Avacopan, prednisolone and rituximab

Intervention Type DRUG

Patients in the avacoapn group will be treated with avacoapn, short-term reduced-dose prednisolone and rituximab.

Interventions

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Avacopan, prednisolone and rituximab

Patients in the avacoapn group will be treated with avacoapn, short-term reduced-dose prednisolone and rituximab.

Intervention Type DRUG

Prednisolone and rituximab

Patients in the glucocorticoid arm will be treated with reduced-dose prednisolone and rituximab.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Provision of written informed consent by a patient or a surrogate decision maker
2. Age=\>18 years
3. New clinical diagnosis of ANCA-associated vasculitis (granulomatosis with polyangiitis, microscopic polyangiitis) consistent with the 2012 Chapel Hill consensus definitions and 2022 EULAR/ACR classification criteria
4. Positive test by ELISA, CLEIA or FEIA for proteinase 3-ANCA or myeloperoxidase-ANCA

Exclusion Criteria

1. Prior treatment for ANCA-associated vasculitis before trial entry
2. ANCA-associated vasculitis related glomerulonephritis (eGFR less than 15ml/min) or alveolar hemorrhage (oxygen inhalation more than 2L/min)
3. Presence of another multisystem autoimmune disease
4. Known infection with HIV; a past or current history of hepatitis B virus or hepatitis C virus infection
5. Desire to bear children, pregnancy or lactating
6. History of malignancy within the past 5 years or any evidence of persistent malignancy
7. Ongoing or recent (last 1 year) evidence of active tuberculosis
8. History of severe allergy or anaphylaxis to monoclonal antibody therapy
9. Any concomitant condition anticipated to likely require oral systemic glucocorticoids, immunosuppressants, biologics, plasma exchange or IVIg
10. Any biological B cell depleting agent (such as rituximab or belimumab)-use within the past 6 months
11. Past history of medication of avacopan
12. Patients can not take avacopan and prednisolone orally
13. Other conditions, in the investigator\'s opinion, inappropriate for the trial entry
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Kissei Pharmaceutical Co., Ltd.

INDUSTRY

Sponsor Role collaborator

International University of Health and Welfare

OTHER

Sponsor Role collaborator

Chiba University

OTHER

Sponsor Role lead

Responsible Party

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Shunsuke Furuta

Associate Professor, Department of Allergy and Clinical Immunology

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Masayoshi Harigai, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

International University of Health and Welfare

Locations

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Fujita Health University Hospital

Toyoake, Aichi-ken, Japan

Site Status NOT_YET_RECRUITING

Asahi General Hospital

Asahi, Chiba, Japan

Site Status RECRUITING

Chiba Aoba Municipal Hospital

Chiba, Chiba, Japan

Site Status RECRUITING

Chiba University

Chiba, Chiba, Japan

Site Status RECRUITING

Chiba Rosai Hospital

Ichihara, Chiba, Japan

Site Status RECRUITING

Kameda Medical Centre

Kamogawa, Chiba, Japan

Site Status RECRUITING

International University of Health and Welfare

Narita, Chiba, Japan

Site Status RECRUITING

Japanese Red Cross Narita Hospital

Narita, Chiba, Japan

Site Status RECRUITING

Gunma University

Maebashi, Gunma, Japan

Site Status RECRUITING

Kagawa University

Hiragi, Kagawa-ken, Japan

Site Status RECRUITING

St.Marianna University School of Medicine

Kawasaki, Kanagawa, Japan

Site Status RECRUITING

Tohoku Univerisity

Sendai, Miyagi, Japan

Site Status RECRUITING

Nagasaki University

Nagasaki, Nagasaki, Japan

Site Status RECRUITING

Okayama University

Okayama, Okayama-ken, Japan

Site Status RECRUITING

Kitano Hospital

Osaka, Osaka, Japan

Site Status RECRUITING

Saitama Medical University

Kawagoe, Saitama, Japan

Site Status RECRUITING

Dokkyo Medical University

Mibu, Tochigi, Japan

Site Status RECRUITING

Juntendo Univeristy

Bunkyoku, Tokyo, Japan

Site Status RECRUITING

Kyorin University

Mitaka, Tokyo, Japan

Site Status RECRUITING

Toho University

Ōta-ku, Tokyo, Japan

Site Status RECRUITING

Teikyo University

tabashi City, Tokyo, Japan

Site Status RECRUITING

Yamanashi University

Chuo-shi, Yamanashi, Japan

Site Status RECRUITING

Countries

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Japan

Central Contacts

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Shunsuke Furuta, MD, PhD

Role: CONTACT

81+43-222-7171 ext. 5531

Facility Contacts

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Naotake Tsuboi, MD, PhD

Role: primary

81+0562-93-2111

ShinIchiro Kagami, MD, PhD

Role: primary

81+0479-63-8111

Yoshihisa Kobayashi, MD, PhD

Role: primary

81+043-227-1131

Shunsuke Furuta, MD, PhD

Role: primary

81+432227171 ext. 5531

Tomohiro Tamachi, MD, PhD

Role: primary

81+0436-74-1111

Tomo Suzuki, MD

Role: primary

81+04-7092-2211

Masayoshi Harigai, MD, PhD

Role: primary

81+0476-35-5600

Masaki Hiraguri, MD

Role: primary

81+0476-22-2311

Keijyu Hiromura, MD, PhD

Role: primary

81+027-220-7111

Hiroaki Dobashi, MD, PhD

Role: primary

81+087-898-5111

Kimito Kawahata, MD, PhD

Role: primary

81+044-977-8111

Hiroshi Fujii, MD, PhD

Role: primary

81+022-717-7000

Atsushi Kawakami, MD, PhD

Role: primary

81+095-819-7200

Yoshinori Matsumoto, MD, PhD

Role: primary

81+086-235-7235

Tomomi Endo, MD

Role: primary

81+06-6312-1221

Takahiko Kurasawa, MD, PhD

Role: primary

81+049-228-3574

Kei Ikeda, MD, PhD

Role: primary

81+0282-86-1111

Naoto Tamura, MD, PhD

Role: primary

81+03-3813-3111

Yoshinori Komagata, MD, PhD

Role: primary

81+0422-47-5511

Toshihiro Nanki, MD, PhD

Role: primary

81+03-3762-4151

Hajime Kono, MD, PhD

Role: primary

81+03-3964-1211

Daiki Nakagomi, MD, PhD

Role: primary

81+055-273-3113

References

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Exley AR, Bacon PA, Luqmani RA, Kitas GD, Gordon C, Savage CO, Adu D. Development and initial validation of the Vasculitis Damage Index for the standardized clinical assessment of damage in the systemic vasculitides. Arthritis Rheum. 1997 Feb;40(2):371-80. doi: 10.1002/art.1780400222.

Reference Type BACKGROUND
PMID: 9041949 (View on PubMed)

Mukhtyar C, Lee R, Brown D, Carruthers D, Dasgupta B, Dubey S, Flossmann O, Hall C, Hollywood J, Jayne D, Jones R, Lanyon P, Muir A, Scott D, Young L, Luqmani RA. Modification and validation of the Birmingham Vasculitis Activity Score (version 3). Ann Rheum Dis. 2009 Dec;68(12):1827-32. doi: 10.1136/ard.2008.101279. Epub 2008 Dec 3.

Reference Type BACKGROUND
PMID: 19054820 (View on PubMed)

Suppiah R, Robson JC, Grayson PC, Ponte C, Craven A, Khalid S, Judge A, Hutchings A, Merkel PA, Luqmani RA, Watts RA; DCVAS INVESTIGATORS. 2022 American College of Rheumatology/European Alliance of Associations for Rheumatology classification criteria for microscopic polyangiitis. Ann Rheum Dis. 2022 Mar;81(3):321-326. doi: 10.1136/annrheumdis-2021-221796. Epub 2022 Feb 2.

Reference Type BACKGROUND
PMID: 35110332 (View on PubMed)

Robson JC, Grayson PC, Ponte C, Suppiah R, Craven A, Judge A, Khalid S, Hutchings A, Watts RA, Merkel PA, Luqmani RA; DCVAS Investigators. 2022 American College of Rheumatology/European Alliance of Associations for Rheumatology classification criteria for granulomatosis with polyangiitis. Ann Rheum Dis. 2022 Mar;81(3):315-320. doi: 10.1136/annrheumdis-2021-221795. Epub 2022 Feb 2.

Reference Type BACKGROUND
PMID: 35110333 (View on PubMed)

Hellmich B, Sanchez-Alamo B, Schirmer JH, Berti A, Blockmans D, Cid MC, Holle JU, Hollinger N, Karadag O, Kronbichler A, Little MA, Luqmani RA, Mahr A, Merkel PA, Mohammad AJ, Monti S, Mukhtyar CB, Musial J, Price-Kuehne F, Segelmark M, Teng YKO, Terrier B, Tomasson G, Vaglio A, Vassilopoulos D, Verhoeven P, Jayne D. EULAR recommendations for the management of ANCA-associated vasculitis: 2022 update. Ann Rheum Dis. 2024 Jan 2;83(1):30-47. doi: 10.1136/ard-2022-223764.

Reference Type BACKGROUND
PMID: 36927642 (View on PubMed)

Furuta S, Nakagomi D, Kobayashi Y, Hiraguri M, Sugiyama T, Amano K, Umibe T, Kono H, Kurasawa K, Kita Y, Matsumura R, Kaneko Y, Ninagawa K, Hiromura K, Kagami SI, Inaba Y, Hanaoka H, Ikeda K, Nakajima H; LoVAS Collaborators. Effect of Reduced-Dose vs High-Dose Glucocorticoids Added to Rituximab on Remission Induction in ANCA-Associated Vasculitis: A Randomized Clinical Trial. JAMA. 2021 Jun 1;325(21):2178-2187. doi: 10.1001/jama.2021.6615.

Reference Type BACKGROUND
PMID: 34061144 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 33596356 (View on PubMed)

Jennette JC, Falk RJ, Bacon PA, Basu N, Cid MC, Ferrario F, Flores-Suarez LF, Gross WL, Guillevin L, Hagen EC, Hoffman GS, Jayne DR, Kallenberg CG, Lamprecht P, Langford CA, Luqmani RA, Mahr AD, Matteson EL, Merkel PA, Ozen S, Pusey CD, Rasmussen N, Rees AJ, Scott DG, Specks U, Stone JH, Takahashi K, Watts RA. 2012 revised International Chapel Hill Consensus Conference Nomenclature of Vasculitides. Arthritis Rheum. 2013 Jan;65(1):1-11. doi: 10.1002/art.37715. No abstract available.

Reference Type BACKGROUND
PMID: 23045170 (View on PubMed)

Study Documents

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Document Type: Study Protocol

Regarding deidentified patient data, please request to Dr Shunsuke Furuta at [email protected] or Dr Masayoshi Harigai at [email protected] after publishing pre-defined trial results.

View Document

Related Links

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https://www.m.chiba-u.jp/dept/allergy-clin-immunol/researcher/clinical/

English version of the protocol is now preparing and will be shown in the above website.

Other Identifiers

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CRB0097-24

Identifier Type: -

Identifier Source: org_study_id

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