VasculitisPhase 3NCT06321601
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 study testing Avacopan in people with Vasculitis, run by Amgen at 34 sites in 10 countries.
Trial summary
The main objective of this study is to explore the efficacy of avacopan in participants affected by AAV. Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Vasculitis
- What is being tested?
- Avacopan
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 34 sitesin 10 countries
- Study dates
- 22 Oct 2024to 18 Jul 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 9Show fewer
- 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.
Trial locations and contacts
34 sites in 10 countries.
34 sites in 10 countries
United States10 sites
Akron Childrens Hospital
Akron, Ohio, 44308
Emory University
Atlanta, Georgia, 30322
University of North Carolina
Chapel Hill, North Carolina, 27599
Wake Forest University Health Sciences
Charlotte, North Carolina, 28203
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Texas Childrens Hospital
Houston, Texas, 77030
Riley Hospital for Children
Indianapolis, Indiana, 46202
Cohen Children Medical Center
Lake Success, New York, 11042
University of Minnesota Masonic Childrens Hospital Discovery Clinic
Minneapolis, Minnesota, 55454
University of Pittsburgh Medical Center Childrens Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Canada4 sites
Alberta Childrens Hospital
Calgary, Alberta, T3B 6A8
Stollery Childrens Hospital
Edmonton, Alberta, T6G 1C9
CHU Sainte Justine
Montreal, Quebec, H3T 1C5
British Columbia Childrens Hospital
Vancouver, British Columbia, V6H 3N1
France4 sites
Centre Hospitalier Universitaire de Bordeaux - Hopital Pellegrin
Bordeaux, 33076
Hospices Civils de Lyon Hopital Femme Mere Enfant
Bron, 69677
Hopital Necker Enfants Malades
Paris, 75015
Hopital Necker
Paris, 75015
Poland3 sites
Uniwersyteckie Centrum Kliniczne
Gdansk, 80-952
Uniwersytecki Szpital Dzieciecy w Krakowie
Krakow, 30-663
Dzieciecy Szpital Kliniczny im. Jozefa Polikarpa Brudzinskiego w Warszawie
Warsaw, 02-091
Spain3 sites
Hospital Universitari Vall d Hebron
Barcelona, Catalonia, 08035
Hospital Sant Joan de Deu
Esplugues de Llobregat, Catalonia, 08950
Hospital Universitario 12 de Octubre
Madrid, 28041
Turkey (Türkiye)3 sites
Hacettepe Universitesi Tip Fakultesi Hastanesi
Ankara, 06100
Umraniye Egitim ve Arastirma Hastanesi
Istanbul, 34764
Erciyes Universitesi Tip Fakultesi Hastanesi
Kayseri, 38030
Belgium2 sites
Universitair Ziekenhuis Gent
Ghent, 9000
Universitair Ziekenhuis Leuven - Gasthuisberg
Leuven, 3000
Czechia2 sites
Vseobecna fakultni nemocnice v Praze
Prague, 128 08
Fakultni nemocnice Motol a Homolka
Prague, 150 06
Hungary2 sites
Semmelweis Egyetem
Budapest, 1094
Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont Altalanos Orvostudomanyi Kar
Szeged, 6720
About this study
Official title: 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.
- Conditions
- Vasculitis
- Being tested
- Avacopan
- Keywords
- Pediatric, Avacopan, Tavneos®
- Medical subject headings
- Vasculitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AvacopanDrug
Also called AMG 569
Oral administration
| Study group | Role | What they receive |
|---|---|---|
| Avacopan | Experimental | Participants will receive avacopan twice-daily (BID) administered as oral tablets or liquid formula for 52 weeks. Receives: Drug: Avacopan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Participants Achieving Disease Remission at Week 26 According to the Pediatric Vasculitis Activities Score (PVAS)
Measured: Week 26
Proportion of Participants With Sustained Disease Remission at Week 52 According to the PVAS
Measured: Week 52
Secondary outcomes (13)
Plasma Concentrations of Avacopan
Measured: 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.
Measured: Day 1 up to approximately Week 60
Proportion of Participants Achieving Disease Remission at Week 26 According to the Birmingham Vasculitis Activity Score (BVAS)
Measured: Week 26
Proportion of Participants Achieving Disease Remission at Week 52 According to the BVAS
Measured: Week 52
Proportion of Participants With PVAS of 0 Over Time Through Week 52
Measured: Up to Week 52
Proportion of Participants With BVAS of 0 Over Time Through Week 52
Measured: Up to Week 52
Change From Baseline Over 52 Weeks in Urinary Albumin-Creatinine Ratio (UACR)
Measured: Baseline up to Week 52
Change From Baseline Over 52 Weeks in Estimated Glomerular Filtration Rate (eGFR)
Measured: Baseline up to Week 52
Change From Baseline Over 52 Weeks In Physician Global Assessment (PGA) of Disease Activity
Measured: Baseline up to Week 52
Change From Baseline Over 52 Weeks in Pediatric Vasculitis Damage Index (PVDI)
Measured: Baseline up to Week 52
Number of Glucocorticoid Dosages Administered
Measured: 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.
Measured: 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.
Measured: Day 1 and Week 2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Mar 2024
- Study start22 Oct 2024
- Primary completion18 Jul 2027 (estimated)
- Study completion18 Jul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for VasculitisStudies of the same condition, open and under way.
- More trials run by AmgenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amgen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- MD, Study director, Amgen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06321601
- Sponsor's protocol ID
- 20230070
- Status last verified
- Jul 2026
- First submitted
- 14 Mar 2024
- Last updated
- 20 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
Study at a glance
From the registry record, last updated by the sponsor on 20 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT06321601
- Acronym / sponsor ID
- 20230070
- Conditions
- Vasculitis
- Keywords
- Pediatric, Avacopan, Tavneos®
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 19 participants (actual)
- Lead sponsor
- AmgenIndustry (pharma, biotech, devices)
- Study start
- 22 Oct 2024 (actual)
- Primary completion
- 18 Jul 2027 (estimated)
- Study completion
- 18 Jul 2027 (estimated)
- First posted
- 20 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main objective of this study is to explore the efficacy of avacopan in participants affected by AAV.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Avacopan | Experimental | Drug: AvacopanParticipants will receive avacopan twice-daily (BID) administered as oral tablets or liquid formula for 52 weeks. |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AvacopanAlso: AMG 569Oral administration | Drug | Avacopan |
Primary outcome
Proportion of Participants Achieving Disease Remission at Week 26 According to the Pediatric Vasculitis Activities Score (PVAS)
Time frame: Week 26
Proportion of Participants With Sustained Disease Remission at Week 52 According to the PVAS
Time frame: Week 52
13 secondary outcomes
Plasma Concentrations of Avacopan
Time frame: Day 1 up to Week 52
Number of Participants Experiencing Treatment-emergent Adverse Events (TEAE)
Time frame: 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
Proportion of Participants Achieving Disease Remission at Week 52 According to the BVAS
Time frame: Week 52
Proportion of Participants With PVAS of 0 Over Time Through Week 52
Time frame: Up to Week 52
Proportion of Participants With BVAS of 0 Over Time Through Week 52
Time frame: Up to Week 52
Change From Baseline Over 52 Weeks in Urinary Albumin-Creatinine Ratio (UACR)
Time frame: Baseline up to Week 52
Change From Baseline Over 52 Weeks in Estimated Glomerular Filtration Rate (eGFR)
Time frame: 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
Change From Baseline Over 52 Weeks in Pediatric Vasculitis Damage Index (PVDI)
Time frame: Baseline up to Week 52
Number of Glucocorticoid Dosages Administered
Time frame: 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
Taste and Acceptability Score of Avacopan per TASTY Faces Scale
Time frame: Day 1 and Week 2
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jul 2026.
- AmgenTrials clinical trials websiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
MD study director
Amgen
34 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Avacopan in people with Vasculitis, run by Amgen at 34 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 20 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 34 sites in United States, Canada, France, Poland, Spain and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of Participants Achieving Disease Remission at Week 26 According to the Pediatric Vasculitis Activities Score (PVAS) (time frame: Week 26). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jul 2026.