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New hope for kids with rare blood vessel disease: avacopan trial launches

NCT ID NCT06321601

Disease control Sponsor: Amgen Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jul 21, 2026 · Updated 3 times

Summary

This study tests whether the drug avacopan can help children aged 6 to 18 with a rare autoimmune disease called ANCA-associated vasculitis, which causes inflammation of blood vessels. About 20 participants will receive avacopan alongside standard treatments like rituximab or cyclophosphamide. The main goal is to see if the disease goes into remission by 26 weeks and stays under control for a year.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
avacopan (a drug that blocks inflammation, taken by mouth)
What this could lead to
If successful, this could provide a new treatment option for children with this rare autoimmune disease, potentially reducing the need for steroids.
What could go wrong
This is a small, single-arm study with no placebo group, so results may be less reliable. Avacopan may cause side effects like infections or liver issues.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

19 people

The number who actually took part.

Started

Oct 2024

Expected to finish

Jul 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

granulomatosis with polyangiitis microscopic polyangiitis vasculitis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Vasculitis

Contacts and locations

Locations

  • Akron Childrens Hospital

    Akron, Ohio, 44308, United States

  • Alberta Childrens Hospital

    Calgary, Alberta, T3B 6A8, Canada

  • British Columbia Childrens Hospital

    Vancouver, British Columbia, V6H 3N1, Canada

  • CHU Sainte Justine

    Montreal, Quebec, H3T 1C5, Canada

  • Centre Hospitalier Universitaire de Bordeaux - Hopital Pellegrin

    Bordeaux, 33076, France

  • Cleveland Clinic Foundation

    Cleveland, Ohio, 44195, United States

  • Cohen Children Medical Center

    Lake Success, New York, 11042, United States

  • Dzieciecy Szpital Kliniczny im. Jozefa Polikarpa Brudzinskiego w Warszawie

    Warsaw, 02-091, Poland

  • Emory University

    Atlanta, Georgia, 30322, United States

  • Erciyes Universitesi Tip Fakultesi Hastanesi

    Kayseri, 38030, Turkey (Türkiye)

  • Fakultni nemocnice Motol a Homolka

    Prague, 150 06, Czechia

  • Hacettepe Universitesi Tip Fakultesi Hastanesi

    Ankara, 06100, Turkey (Türkiye)

  • Hopital Necker

    Paris, 75015, France

  • Hopital Necker Enfants Malades

    Paris, 75015, France

  • Hospices Civils de Lyon Hopital Femme Mere Enfant

    Bron, 69677, France

  • Hospital Sant Joan de Deu

    Esplugues de Llobregat, Catalonia, 08950, Spain

  • Hospital Universitari Vall d Hebron

    Barcelona, Catalonia, 08035, Spain

  • Hospital Universitario 12 de Octubre

    Madrid, 28041, Spain

  • Narodny ustav detskych chorob

    Bratislava, 833 40, Slovakia

  • Riley Hospital for Children

    Indianapolis, Indiana, 46202, United States

  • Semmelweis Egyetem

    Budapest, 1094, Hungary

  • Stollery Childrens Hospital

    Edmonton, Alberta, T6G 1C9, Canada

  • Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont Altalanos Orvostudomanyi Kar

    Szeged, 6720, Hungary

  • Texas Childrens Hospital

    Houston, Texas, 77030, United States

  • Umraniye Egitim ve Arastirma Hastanesi

    Istanbul, 34764, Turkey (Türkiye)

  • Universitair Ziekenhuis Gent

    Ghent, 9000, Belgium

  • Universitair Ziekenhuis Leuven - Gasthuisberg

    Leuven, 3000, Belgium

  • University of Minnesota Masonic Childrens Hospital Discovery Clinic

    Minneapolis, Minnesota, 55454, United States

  • University of North Carolina

    Chapel Hill, North Carolina, 27599, United States

  • University of Pittsburgh Medical Center Childrens Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • Uniwersytecki Szpital Dzieciecy w Krakowie

    Krakow, 30-663, Poland

  • Uniwersyteckie Centrum Kliniczne

    Gdansk, 80-952, Poland

  • Vseobecna fakultni nemocnice v Praze

    Prague, 128 08, Czechia

  • Wake Forest University Health Sciences

    Charlotte, North Carolina, 28203, United States

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