PREP-ANCA - Comparison of a strategy based on clinic-biological surveillance versus pre-emptive treatment with rituximab in the event of ANCA repositivation in patients with granulomatosis with polyangiitis and microscopic polyangiitis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Assistance Publique Hopitaux De Paris

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Decision date (initial)

2026-06-08

Transition trial

No

Low-intervention

Yes

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

French Ministry of Health National PHRC 2023

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

To evaluate the superiority of pre-emptive treatment with rituximab compared to clinic-biological monitoring every 3 months in the event of ANCA reactivation in patients with GPA and MPA who were previously treated with rituximab for 18 months and are in complete remission. The primary outcome is the maintenance of remission, defined by a Birmingham Vasculitis Activity Score (BVAS) of 0.

Secondary objectives 9

1. 1. To compare the proportion of participants with a minor and/or major relapse
2. 2. To compare the treatment tolerance profile, especially the proportion of severe infections
3. 3. To compare the number of rituximab perfusions required in both arms during follow-up
4. 4. To compare the cumulative dose of rituximab in both arms
5. 5. To compare sequeale during follow-up
6. 6. To compare the quality of life during follow-up
7. 7. To evaluate the mortality rate
8. 8. To compare the cumulative dose of corticosteroids in both arms
9. 9. To compare changes in ANCA and CD19 in both arms

Conditions and MedDRA coding

granulomatosis with polyangiitis and microscopic polyangiitis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. 1. Age ≥ 18 years
2. 2. Diagnosis of GPA or MPA according to the ACR/EULAR 2022 classification criteria
3. 3. Patients previously treated with rituximab as maintenance therapy, following the recommended regimen: an intravenous infusion of 500 mg on Day 1, possibly repeated on Day 15, then every 6 months for 18 months (4 to 5 infusions of 500 mg)
4. 4. Complete remission (BVAS = 0) at randomization
5. 5. Patients with ANCA repositivation on a specific antigenic test within 3 months prior to randomization
6. 6. Ability to provide written informed consent before participating in the study
7. 7. Affiliation with a social security system

Exclusion criteria 12

1. 1. Diagnosis of another vasculitis
2. 2. Active vasculitis
3. 3. Acute or chronic active infection requiring hospitalization or treatment with an intravenous anti-infective drug in the 4 weeks prior to study selection, or with an oral anti-infective drug in the 2 weeks prior to selection for the trial
4. 4. History of deep tissue infection in the year prior to inclusion in the trial
5. 5. History of severe chronic or recurrent infections, or any underlying disease predisposing to serious infections
6. 6. Administration of a live vaccine within 4 weeks prior to enrolment in the trial
7. 7. Evolving cancer or recent hematological malignancy (< 5 years), except localized prostate cancer and basal cell skin cancers
8. 8. Patients with a systemic disease for which the treatments used in the trial could have unpredictable or inappropriate consequences
9. 9. History of allergic reactions, severe anaphylaxis, or known hypersensitivity to humanized or murine monoclonal antibodies and/or corticosteroids
10. 10. Suspicion of non-compliance with treatment and foreseeable inability or refusal to complete the follow-up exams required by the protocol
11. 11. Inability to provide written informed consent before participating in the study
12. 12. Pregnant women and breastfeeding mothers. Women of childbearing age must use reliable contraception throughout treatment and for 6 months after the last infusion.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary endpoint is survival without relapse in each arm after 24 months of follow-up. Relapse is defined as a BVAS >0.

Secondary endpoints 9

1. 1. Number of participants with a major or minor relapse during follow-
2. 2. Number of adverse events
3. 3. Number of serious adverse events: potentially fatal, requiring hospitalization, causing significant disability or leading to death
4. 4. Number of rituximab perfusions performed in both arms
5. 5. Sequalae according to the VDI classification during follow-up
6. 6. Quality of life according of HAQ and SF-36 classifications during follow-up
7. 7. Mortality in both arms
8. 8. The cumulative dose and duration of treatment with corticosteroids in both arms
9. 9. Changes in ANCA and B CD19+ cells rate in both arms and their correlation with clinical events

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Comparator 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Assistance Publique Hopitaux De Paris

Sponsor organisation

Assistance Publique Hopitaux De Paris

Address

Porte 23, 1 Avenue Claude Vellefaux 1 Avenue Claude Vellefaux

City

Paris Cedex 10

Postcode

75475

Country

France

Scientific contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Dr Florence DELESTRE

Public contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Dr Florence DELESTRE

Locations

1 EU/EEA country · 22 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 16 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications