Study to Evaluate Avacopan in Combination With a Rituximab or Cyclophosphamide-containing Regimen, in Children From 6 Years to < 18 Years of Age With AAV.

NCT06321601 | Recruiting Phase 3 FDA Drug

• 1 other identifier: 20230070
• Study Type: interventional
• Target: 20
• Locations: 10 countries
• Sites: 31

Brief Summary

The main objective of this study is to explore the efficacy of avacopan in participants affected by AAV.

Conditions

Vasculitis

Keywords

Pediatric; Avacopan; Tavneos®

Outcome Measures

Primary Outcomes (2)

• Proportion of Participants Achieving Disease Remission at Week 26 According to the Pediatric Vasculitis Activities Score (PVAS)

  Week 26

• Proportion of Participants With Sustained Disease Remission at Week 52 According to the PVAS

  Week 52

Secondary Outcomes (13)

• Plasma Concentrations of Avacopan

  Day 1 up to Week 52

• Number of Participants Experiencing Treatment-emergent Adverse Events (TEAE)

  Day 1 up to approximately Week 60

• Proportion of Participants Achieving Disease Remission at Week 26 According to the Birmingham Vasculitis Activity Score (BVAS)

  Week 26

• Proportion of Participants Achieving Disease Remission at Week 52 According to the BVAS

  Week 52

• Proportion of Participants With PVAS of 0 Over Time Through Week 52

  Up to Week 52

Study Arms (1)

Avacopan

EXPERIMENTAL

Participants will receive avacopan twice-daily (BID) administered as oral tablets or liquid formula for 52 weeks.

Drug: Avacopan

Interventions

Avacopan DRUG

Oral administration

Also known as: AMG 569

Avacopan

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male and female children and adolescents from 6 to \< 18 years of age
• Clinical diagnosis of granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA), consistent with Chapel-Hill Consensus Conference definitions (Jennette et al, 2013)
• Newly diagnosed or relapsed AAV with positive test for anti-PR3 or anti-MPO antibodies
• At least 1 PVAS major item, at least 3 PVAS nonmajor items, or at least the 2 renal items of proteinuria and hematuria.
• eGFR \> 15 mL/min/1.73 m2 (using modified Schwartz equation per central lab guidelines)
• Participants must have a bodyweight of ≥ 15 kg at day 1.

You may not qualify if:

• Any other known multisystem autoimmune disease including eosinophilic granulomatosis with polyangiitis (Churg-Strauss), systemic lupus erythematosus , IgA vasculitis (Henoch-Schönlein), rheumatoid vasculitis, Sjogren's syndrome, anti-glomerular basement membrane disease, or cryoglobulinemic vasculitis
• Alveolar hemorrhage requiring invasive pulmonary ventilation support anticipated to last beyond the screening period of the study
• Any medical condition requiring or expected to require continued use of immunosuppressive treatments, including corticosteroids that may cause confoundment with study assessments and study conclusions.

Sponsors & Collaborators

• Amgen: lead

Study Sites (31)

Emory University

Atlanta, Georgia, 30322, United States

RECRUITING

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

RECRUITING

University of Minnesota Masonic Childrens Hospital Discovery Clinic

Minneapolis, Minnesota, 55454, United States

RECRUITING

Cohen Children Medical Center

Lake Success, New York, 11042, United States

RECRUITING

University of North Carolina

Chapel Hill, North Carolina, 27599, United States

RECRUITING

Wake Forest University Health Sciences

Charlotte, North Carolina, 28203, United States

RECRUITING

Akron Childrens Hospital

Akron, Ohio, 44308, United States

RECRUITING

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

RECRUITING

University of Pittsburgh Medical Center Childrens Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

RECRUITING

Texas Childrens Hospital

Houston, Texas, 77030, United States

RECRUITING

Universitair Ziekenhuis Gent

Ghent, 9000, Belgium

RECRUITING

Universitair Ziekenhuis Leuven - Gasthuisberg

Leuven, 3000, Belgium

RECRUITING

Alberta Childrens Hospital

Calgary, Alberta, T3B 6A8, Canada

RECRUITING

Stollery Childrens Hospital

Edmonton, Alberta, T6G 1C9, Canada

RECRUITING

CHU Sainte Justine

Montreal, Quebec, H3T 1C5, Canada

RECRUITING

Vseobecna fakultni nemocnice v Praze

Prague, 128 08, Czechia

RECRUITING

Fakultni nemocnice v Motole

Prague, 150 06, Czechia

RECRUITING

Centre Hospitalier Universitaire de Bordeaux - Hopital Pellegrin

Bordeaux, 33076, France

RECRUITING

Hospices Civils de Lyon Hopital Femme Mere Enfant

Bron, 69677, France

RECRUITING

Hopital Necker Enfants Malades

Paris, 75015, France

RECRUITING

Hopital Necker

Paris, 75015, France

RECRUITING

Semmelweis Egyetem

Budapest, 1094, Hungary

RECRUITING

Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont Altalanos Orvostudomanyi Kar

Szeged, 6720, Hungary

RECRUITING

Uniwersyteckie Centrum Kliniczne

Gdansk, 80-952, Poland

RECRUITING

Uniwersytecki Szpital Dzieciecy w Krakowie

Krakow, 30-663, Poland

RECRUITING

Dzieciecy Szpital Kliniczny im. Jozefa Polikarpa Brudzinskiego w Warszawie

Warsaw, 02-091, Poland

RECRUITING

Narodny ustav detskych chorob

Bratislava, 833 40, Slovakia

RECRUITING

Hospital Universitari Vall d Hebron

Barcelona, Catalonia, 08035, Spain

RECRUITING

Hospital Sant Joan de Deu

Esplugues de Llobregat, Catalonia, 08950, Spain

RECRUITING

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

RECRUITING

Hacettepe Universitesi Tip Fakultesi Hastanesi

Ankara, 06100, Turkey (Türkiye)

RECRUITING

Related Links

• AmgenTrials clinical trials website

MeSH Terms

Conditions

Vasculitis

Interventions

avacopan

Condition Hierarchy (Ancestors)

Vascular Diseases; Cardiovascular Diseases

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Central Study Contacts

Amgen Call Center

CONTACT

866-572-6436; medinfo@amgen.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 14, 2024
• First Posted: March 20, 2024
• Study Start: October 22, 2024
• Primary Completion (Estimated): September 26, 2028
• Study Completion (Estimated): September 26, 2028
• Last Updated: May 1, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• 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 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 reevaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. 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.

Locations

TU (1); BE (2); ES (3); HU (2); US (10); FR (4); SL (1); CA (3); CZ (2); PL (3)