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.

A Phase 3, Open-label, Uncontrolled Single-arm Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Avacopan in Combination With a Rituximab or a Cyclophosphamide-containing Regimen in Children From 6 Years to < 18 Years of Age With Active ANCA-associated Vasculitis (AAV)

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

Study Overview

• Status: Recruiting
• Conditions: Vasculitis
• Intervention / Treatment: Drug: Avacopan

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Amgen Call Center; Phone Number: 866-572-6436; Email: medinfo@amgen.com

Study Locations

• Belgium
  • Ghent, Belgium, 9000
    • Recruiting
    • Universitair Ziekenhuis Gent
  • Leuven, Belgium, 3000
    • Recruiting
    • Universitair Ziekenhuis Leuven - Gasthuisberg
• Canada
  • Alberta
    • Calgary, Alberta, Canada, T3B 6A8
      • Recruiting
      • Alberta Childrens Hospital
    • Edmonton, Alberta, Canada, T6G 1C9
      • Recruiting
      • Stollery Childrens Hospital
  • British Columbia
    • Vancouver, British Columbia, Canada, V6H 3N1
      • Recruiting
      • British Columbia Childrens Hospital
  • Quebec
    • Montreal, Quebec, Canada, H3T 1C5
      • Recruiting
      • CHU Sainte Justine
• Czechia
  • Prague, Czechia, 128 08
    • Recruiting
    • Vseobecna Fakultni Nemocnice V Praze
  • Prague, Czechia, 150 06
    • Recruiting
    • Fakultni nemocnice v Motole
• France
  • Bordeaux, France, 33076
    • Recruiting
    • Centre Hospitalier Universitaire de Bordeaux - Hopital Pellegrin
  • Bron, France, 69677
    • Recruiting
    • Hospices Civils de Lyon Hopital Femme Mere Enfant
  • Paris, France, 75015
    • Recruiting
    • Hopital Necker
  • Paris, France, 75015
    • Recruiting
    • Hôpital Necker Enfants Malades
• Hungary
  • Budapest, Hungary, 1094
    • Recruiting
    • Semmelweis Egyetem
  • Szeged, Hungary, 6720
    • Terminated
    • Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont Altalanos Orvostudomanyi Kar
• Poland
  • Gdansk, Poland, 80-952
    • Recruiting
    • Uniwersyteckie Centrum Kliniczne
  • Krakow, Poland, 30-663
    • Recruiting
    • Uniwersytecki Szpital Dzieciecy w Krakowie
  • Warsaw, Poland, 02-091
    • Recruiting
    • Dzieciecy Szpital Kliniczny im. Jozefa Polikarpa Brudzinskiego w Warszawie
• Slovakia
  • Bratislava, Slovakia, 833 40
    • Recruiting
    • Narodny ustav detskych chorob
• Spain
  • Madrid, Spain, 28041
    • Recruiting
    • Hospital Universitario 12 de Octubre
  • Catalonia
    • Barcelona, Catalonia, Spain, 08035
      • Recruiting
      • Hospital Universitari Vall d Hebron
    • Esplugues de Llobregat, Catalonia, Spain, 08950
      • Recruiting
      • Hospital Sant Joan de Déu
• Turkey (Türkiye)
  • Ankara, Turkey (Türkiye), 06100
    • Recruiting
    • Hacettepe Universitesi Tip Fakultesi Hastanesi
• United States
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Recruiting
      • Emory University
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Recruiting
      • Riley Hospital for Children
  • Minnesota
    • Minneapolis, Minnesota, United States, 55454
      • Recruiting
      • University of Minnesota Masonic Childrens Hospital Discovery Clinic
  • New York
    • Lake Success, New York, United States, 11042
      • Recruiting
      • Cohen Children Medical Center
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • Recruiting
      • University of North Carolina
    • Charlotte, North Carolina, United States, 28203
      • Recruiting
      • Wake Forest University Health Sciences
  • Ohio
    • Akron, Ohio, United States, 44308
      • Recruiting
      • Akron Childrens Hospital
    • Cleveland, Ohio, United States, 44195
      • Recruiting
      • Cleveland Clinic Foundation
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15224
      • Recruiting
      • University of Pittsburgh Medical Center Childrens Hospital of Pittsburgh
  • Texas
    • Houston, Texas, United States, 77030
      • Recruiting
      • Texas Childrens Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Avacopan; Participants will receive avacopan twice-daily (BID) administered as oral tablets or liquid formula for 52 weeks.	Drug: Avacopan; Oral administration; Other Names: AMG 569

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Plasma Concentrations of Avacopan		Day 1 up to Week 52
Number of Participants Experiencing Treatment-emergent Adverse Events (TEAE)	TEAEs are any event that occurred after the participant received study treatment. Any clinically significant changes in vital signs, electrocardiograms, and clinical laboratory tests that occurred after study treatment administration were recorded as TEAEs. A serious TEAE is any untoward medical occurrence in a clinical study participant after first dose irrespective of a causal relationship with the study treatment(s) that resulted in death, was immediately life threatening, required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect, or another medically important serious event.	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
Proportion of Participants With BVAS of 0 Over Time Through Week 52		Up to Week 52
Change From Baseline Over 52 Weeks in Urinary Albumin-Creatinine Ratio (UACR)		Baseline up to Week 52
Change From Baseline Over 52 Weeks in Estimated Glomerular Filtration Rate (eGFR)		Baseline up to Week 52
Change From Baseline Over 52 Weeks In Physician Global Assessment (PGA) of Disease Activity		Baseline up to Week 52
Change From Baseline Over 52 Weeks in Pediatric Vasculitis Damage Index (PVDI)		Baseline up to Week 52
Number of Glucocorticoid Dosages Administered		Screening up to Week 52
Proportion of Participants Across the Taste Score Categories of the TASTY Faces Scale	The TASTY faces scale will be utilized to assess the taste of the oral pediatric formulation. A 7-point version of the scale will be used, where higher scores correspond to faces showing favorable tastes. Upon drug administration, the child will be shown the TASTY scale and asked to rate his/her perception of tastiness by pointing to the appropriate face on the scale.	Day 1 and Week 2
Taste and Acceptability Score of Avacopan per TASTY Faces Scale	The TASTY faces scale will be utilized to assess the taste of the oral pediatric formulation. A 7-point version of the scale will be used, where higher scores correspond to faces showing favorable tastes. Upon drug administration, the child will be shown the TASTY scale and asked to rate his/her perception of tastiness by pointing to the appropriate face on the scale.	Day 1 and Week 2

Collaborators and Investigators

• Sponsor: Amgen
• Investigators: Study Director: MD, Amgen

Publications and helpful links

Helpful Links

• AmgenTrials clinical trials website

Study record dates

Study Major Dates

• Study Start (Actual): October 22, 2024
• Primary Completion (Estimated): September 26, 2028
• Study Completion (Estimated): September 26, 2028

Study Registration Dates

• First Submitted: March 14, 2024
• First Submitted That Met QC Criteria: March 19, 2024
• First Posted (Actual): March 20, 2024

Study Record Updates

• Last Update Posted (Actual): May 14, 2026
• Last Update Submitted That Met QC Criteria: May 13, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Pediatric; Avacopan; Tavneos®
• Additional Relevant MeSH Terms: Vascular Diseases; Cardiovascular Diseases; Vasculitis; avacopan
• Other Study ID Numbers: 20230070

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
• IPD Sharing 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.
• IPD Sharing 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.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No