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

NCT ID NCT06321601

Ongoing Disease control Sponsor: Amgen Source: ClinicalTrials.gov ↗

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.

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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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