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.

July 17, 2026 updated by: Amgen

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

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
19

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Belgium
      • Ghent, Belgium, 9000
        • Universitair Ziekenhuis Gent
      • Leuven, Belgium, 3000
        • Universitair Ziekenhuis Leuven - Gasthuisberg
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T3B 6A8
        • Alberta Childrens Hospital
      • Edmonton, Alberta, Canada, T6G 1C9
        • Stollery Childrens Hospital
    • British Columbia
      • Vancouver, British Columbia, Canada, V6H 3N1
        • British Columbia Childrens Hospital
    • Quebec
      • Montreal, Quebec, Canada, H3T 1C5
        • CHU Sainte Justine
  • Czechia
      • Prague, Czechia, 128 08
        • Vseobecna fakultni nemocnice v Praze
      • Prague, Czechia, 150 06
        • Fakultní nemocnice Motol a Homolka
  • France
      • Bordeaux, France, 33076
        • Centre Hospitalier Universitaire de Bordeaux - Hopital Pellegrin
      • Bron, France, 69677
        • Hospices Civils de Lyon Hopital Femme Mere Enfant
      • Paris, France, 75015
        • Hôpital Necker Enfants malades
      • Paris, France, 75015
        • Hopital Necker
  • Hungary
      • Budapest, Hungary, 1094
        • Semmelweis Egyetem
      • Szeged, Hungary, 6720
        • Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont Altalanos Orvostudomanyi Kar
  • Poland
      • Gdansk, Poland, 80-952
        • Uniwersyteckie Centrum Kliniczne
      • Krakow, Poland, 30-663
        • Uniwersytecki Szpital Dzieciecy w Krakowie
      • Warsaw, Poland, 02-091
        • Dzieciecy Szpital Kliniczny im. Jozefa Polikarpa Brudzinskiego w Warszawie
  • Slovakia
      • Bratislava, Slovakia, 833 40
        • Narodny Ustav Detskych Chorob
  • Spain
      • Madrid, Spain, 28041
        • Hospital Universitario 12 de Octubre
    • Catalonia
      • Barcelona, Catalonia, Spain, 08035
        • Hospital Universitari Vall D Hebron
      • Esplugues de Llobregat, Catalonia, Spain, 08950
        • Hospital Sant Joan de Déu
  • Turkey (Türkiye)
      • Ankara, Turkey (Türkiye), 06100
        • Hacettepe Universitesi Tip Fakultesi Hastanesi
      • Istanbul, Turkey (Türkiye), 34764
        • Umraniye Egitim ve Arastirma Hastanesi
      • Kayseri, Turkey (Türkiye), 38030
        • Erciyes Universitesi Tip Fakultesi Hastanesi
  • United States
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Riley Hospital for Children
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • University of Minnesota Masonic Childrens Hospital Discovery Clinic
    • New York
      • Lake Success, New York, United States, 11042
        • Cohen Children Medical Center
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • University of North Carolina
      • Charlotte, North Carolina, United States, 28203
        • Wake Forest University Health Sciences
    • Ohio
      • Akron, Ohio, United States, 44308
        • Akron Childrens Hospital
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic Foundation
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • University of Pittsburgh Medical Center Childrens Hospital of Pittsburgh
    • Texas
      • Houston, Texas, United States, 77030
        • Texas Childrens Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.
  • History or evidence of any other clinically significant disorder, condition, or disease (other than those specified above) that, in the investigator's judgment, would pose an unacceptable risk to subject safety, or interfere with study assessments or completion. The investigator may consult the Amgen medical monitor as needed. The rationale for exclusion and any consultation must be documented in the subject's source record.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Proportion of Participants Achieving Disease Remission at Week 26 According to the Pediatric Vasculitis Activities Score (PVAS)
Time Frame: Week 26
Week 26
Proportion of Participants With Sustained Disease Remission at Week 52 According to the PVAS
Time Frame: Week 52
Week 52

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Plasma Concentrations of Avacopan
Time Frame: Day 1 up to Week 52
Day 1 up to Week 52
Number of Participants Experiencing Treatment-emergent Adverse Events (TEAE)
Time Frame: Day 1 up to approximately Week 60
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)
Time Frame: Week 26
Week 26
Proportion of Participants Achieving Disease Remission at Week 52 According to the BVAS
Time Frame: Week 52
Week 52
Proportion of Participants With PVAS of 0 Over Time Through Week 52
Time Frame: Up to Week 52
Up to Week 52
Proportion of Participants With BVAS of 0 Over Time Through Week 52
Time Frame: Up to Week 52
Up to Week 52
Change From Baseline Over 52 Weeks in Urinary Albumin-Creatinine Ratio (UACR)
Time Frame: Baseline up to Week 52
Baseline up to Week 52
Change From Baseline Over 52 Weeks in Estimated Glomerular Filtration Rate (eGFR)
Time Frame: Baseline up to Week 52
Baseline up to Week 52
Change From Baseline Over 52 Weeks In Physician Global Assessment (PGA) of Disease Activity
Time Frame: Baseline up to Week 52
Baseline up to Week 52
Change From Baseline Over 52 Weeks in Pediatric Vasculitis Damage Index (PVDI)
Time Frame: Baseline up to Week 52
Baseline up to Week 52
Number of Glucocorticoid Dosages Administered
Time Frame: Screening up to Week 52
Screening up to Week 52
Proportion of Participants Across the Taste Score Categories of the TASTY Faces Scale
Time Frame: Day 1 and Week 2
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
Time Frame: Day 1 and Week 2
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

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Amgen

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: MD, Amgen

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 22, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 18, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 18, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 14, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 19, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 20, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 20, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 17, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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