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Gene therapy breakthrough? new trial targets hemophilia b patients previously excluded

NCT ID NCT06003387

Recruiting now Disease control Sponsor: CSL Behring Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Aug 07, 2026 · Updated 4 times

Summary

This study tests a single-dose gene therapy called CSL222 (Etranacogene dezaparvovec) in 35 adults with severe or moderately severe hemophilia B who have detectable AAV5 antibodies—a group often excluded from gene therapy. The goal is to see if it reduces bleeding episodes and is safe. Participants receive one IV infusion and are monitored for side effects and bleeding rates.

What this could mean

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

Active substance
CSL222 (Etranacogene dezaparvovec) gene therapy
What this could lead to
If successful, this could offer a one-time gene therapy option for hemophilia B patients who currently have no gene therapy options due to pre-existing antibodies.
What could go wrong
This is an early Phase 3 study with only 35 participants, so results may not apply to everyone. The gene therapy may not work as expected in people with antibodies, and there are risks like liver issues or allergic reactions.

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.

hemophilia B

As listed by the trial registrant

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

HEMOPHILIA B

Contacts and locations

Locations

  • Centro de Investigacion Clinica GRAMEL S.C.

    RECRUITING

    Mexico City, Mexico City, 3720, Mexico

  • Changhua Christian Hospital (CCH)

    RECRUITING

    Chang-hua, 500, Taiwan

  • Ege University Medical Faculty

    RECRUITING

    Bornova, 35100, Turkey (Türkiye)

  • Gaziantep University Sahinbey Research and Practice Hospital

    RECRUITING

    Gaziantep, 27310, Turkey (Türkiye)

  • Haemophilia Comprehensive Care Centre

    RECRUITING

    Johannesburg, 2193, South Africa

  • Hemophilia Center of Western Pennsylvania (HCWP)

    RECRUITING

    Pittsburgh, Pennsylvania, 15260, United States

  • Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo

    RECRUITING

    São Paulo, 05403-010, Brazil

  • Kaohsiung Medical University Chung-Ho Memorial Hospital (KMUH)

    RECRUITING

    Kaohsiung City, Sanmin District, 80756, Taiwan

  • King Faisal Specialist Hospital and Research Center

    RECRUITING

    Riyadh, 11471, Saudi Arabia

  • Klinika Zaburzen Hemostazy i Chorob Wewnetrznych

    RECRUITING

    Warsaw, 02776, Poland

  • Kyung Hee University Hospital at Gangdong

    RECRUITING

    Seoul, 05278, South Korea

  • Kyungpook National University Hospital

    RECRUITING

    Daegu, 41944, South Korea

  • McMaster University - Hamilton

    RECRUITING

    Hamilton, Ontario, L8N 3Z5, Canada

  • National Taiwan University Hospital

    RECRUITING

    Taipei, 100225, Taiwan

  • National University Hospital

    RECRUITING

    Singapore, 110974, Singapore

  • Prince of Wales Hospital Chinese University of Hong Kong

    RECRUITING

    Shatin, 999077, Hong Kong

  • Queen Mary Hospital

    RECRUITING

    Hong Kong, 999077, Hong Kong

  • Royal Adelaide Hospital

    RECRUITING

    Adelaide, South Australia, 5000, Australia

  • Royal Brisbane Hospital

    RECRUITING

    Herston, Queensland, 4029, Australia

  • Royal Prince Alfred Hospital

    RECRUITING

    Camperdown, New South Wales, 2050, Australia

  • Severance Hospital, Yonsei University Health System

    RECRUITING

    Seoul, 3722, South Korea

  • Sheba Medical Center

    RECRUITING

    Tel Litwinsky, 5265601, Israel

  • Singapore General Hospital

    RECRUITING

    Singapore, 169608, Singapore

  • Specialized Hospital for Active Treatment of Hematological Diseases

    RECRUITING

    Sofia, 1756, Bulgaria

  • Taichung Veterans General Hospital -

    RECRUITING

    Taichung, 40705, Taiwan

  • The Alfred Hospital

    RECRUITING

    Melbourne, Victoria, 3004, Australia

  • Tri-Service General Hospital

    RECRUITING

    Taipei, Neihu District, 114, Taiwan

  • UNICAMP Universidade Estadual de Campinas

    RECRUITING

    São Paulo, 13083-878, Brazil

  • University of California, San Diego (UCSD)

    RECRUITING

    San Diego, California, 92121, United States

  • University of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • Özel Acibadem Adana Hastanesi

    RECRUITING

    Seyhan, 01130, Turkey (Türkiye)

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