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 | Active Not Recruiting Phase 3 FDA Drug

• 1 other identifier: 20230070
• Study Type: interventional
• Target: 19
• Locations: 10 countries
• Sites: 34

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 old of age.
• Clinical diagnosis of Granulomatosis with Polyangiitis (GPA) or microscopic polyangiitis (MPA), consistent with Chapel-Hill Consensus Conference definitions (Jennette et al, 2013).
• Positive anti-PR3(Anti-Proteinase 3) or anti-MPO(Anti-Myeloperoxidase) antibody documented at Screening or historically. Historical positivity is acceptable (even if Screening is negative) if supported by verifiable lab source documentation obtained during AAV diagnosis or disease course; use the most recent positive result.
• At least 1 PVAS major item, at least 3 PVAS nonmajor items, or atleast the 2 renal items of proteinuria and hematuria.
• Estimated glomerular filtration rate (eGFR) of ≥ 15 mL/minute/1.73 m\^2 at screening and day 1.
• Participants must have a bodyweight of ≥ 15 kg at day 1.

You may not qualify if:

• Any other known multisystem autoimmune disease including and not limited to eosinophilic granulomatosis with polyangiitis (EGPA, previously, Churg-Strauss disease), systemic lupus erythematosus, IgA vasculitis / Henoch-Schönlein, Purpura, rheumatoid vasculitis, Sjögren's syndrome, anti-glomerular basement membrane disease, or cryoglobulinemic vasculitis.
• Renal replacement therapy / plasmapheresis: subjects will be excluded who received, require, or initiate CRRT (continuous renal replacement therapy), hemodialysis, any renal dialysis, or plasmapheresis within 14 days prior to Screening or between Screening and Day 1.
• History of kidney transplantation or is anticipated to require renal transplantation during the study.
• 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, ongoing treatment with immunosuppressive, therapy (including systemic glucocorticoids) for a non-AAV indication that in the judgment of the investigator, could confound study assessments or interpretation of study results.
• Female subjects of childbearing potential must have a negative highly sensitive serum pregnancy test at Screening and a negative sensitive urine pregnancy test, on day 1, with results confirmed prior to the first administration of investigational product.
• Known hypersensitivity or contraindication to avacopan, its excipients, or to any investigational product or required concomitant medication used in this study.
• Subject likely to not be available to complete all protocol-required study visits or procedures, and/or to comply with all required study procedures (eg, Clinical Outcome Assessments) to the best of the subject and investigator's knowledge.

Sponsors & Collaborators

• Amgen: lead

Study Sites (34)

Emory University

Atlanta, Georgia, 30322, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

University of Minnesota Masonic Childrens Hospital Discovery Clinic

Minneapolis, Minnesota, 55454, United States

Location

Cohen Children Medical Center

Lake Success, New York, 11042, United States

Location

University of North Carolina

Chapel Hill, North Carolina, 27599, United States

Location

Wake Forest University Health Sciences

Charlotte, North Carolina, 28203, United States

Location

Akron Childrens Hospital

Akron, Ohio, 44308, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

University of Pittsburgh Medical Center Childrens Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Texas Childrens Hospital

Houston, Texas, 77030, United States

Location

Universitair Ziekenhuis Gent

Ghent, 9000, Belgium

Location

Universitair Ziekenhuis Leuven - Gasthuisberg

Leuven, 3000, Belgium

Location

Alberta Childrens Hospital

Calgary, Alberta, T3B 6A8, Canada

Location

Stollery Childrens Hospital

Edmonton, Alberta, T6G 1C9, Canada

Location

British Columbia Childrens Hospital

Vancouver, British Columbia, V6H 3N1, Canada

Location

CHU Sainte Justine

Montreal, Quebec, H3T 1C5, Canada

Location

Vseobecna fakultni nemocnice v Praze

Prague, 128 08, Czechia

Location

Fakultni nemocnice Motol a Homolka

Prague, 150 06, Czechia

Location

Centre Hospitalier Universitaire de Bordeaux - Hopital Pellegrin

Bordeaux, 33076, France

Location

Hospices Civils de Lyon Hopital Femme Mere Enfant

Bron, 69677, France

Location

Hopital Necker Enfants Malades

Paris, 75015, France

Location

Hopital Necker

Paris, 75015, France

Location

Semmelweis Egyetem

Budapest, 1094, Hungary

Location

Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont Altalanos Orvostudomanyi Kar

Szeged, 6720, Hungary

Location

Uniwersyteckie Centrum Kliniczne

Gdansk, 80-952, Poland

Location

Uniwersytecki Szpital Dzieciecy w Krakowie

Krakow, 30-663, Poland

Location

Dzieciecy Szpital Kliniczny im. Jozefa Polikarpa Brudzinskiego w Warszawie

Warsaw, 02-091, Poland

Location

Narodny ustav detskych chorob

Bratislava, 833 40, Slovakia

Location

Hospital Universitari Vall d Hebron

Barcelona, Catalonia, 08035, Spain

Location

Hospital Sant Joan de Deu

Esplugues de Llobregat, Catalonia, 08950, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Hacettepe Universitesi Tip Fakultesi Hastanesi

Ankara, 06100, Turkey (Türkiye)

Location

Umraniye Egitim ve Arastirma Hastanesi

Istanbul, 34764, Turkey (Türkiye)

Location

Erciyes Universitesi Tip Fakultesi Hastanesi

Kayseri, 38030, Turkey (Türkiye)

Location

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

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): July 18, 2027
• Study Completion (Estimated): July 18, 2027
• Last Updated: July 20, 2026

Record last verified: 2026-07

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

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