Gene therapy breakthrough? new trial targets hemophilia b patients previously excluded
NCT ID NCT06003387
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
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.
Disclaimer
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Centro de Investigacion Clinica GRAMEL S.C.
RECRUITINGMexico City, Mexico City, 3720, Mexico
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Changhua Christian Hospital (CCH)
RECRUITINGChang-hua, 500, Taiwan
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Ege University Medical Faculty
RECRUITINGBornova, 35100, Turkey (Türkiye)
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Gaziantep University Sahinbey Research and Practice Hospital
RECRUITINGGaziantep, 27310, Turkey (Türkiye)
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Haemophilia Comprehensive Care Centre
RECRUITINGJohannesburg, 2193, South Africa
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Hemophilia Center of Western Pennsylvania (HCWP)
RECRUITINGPittsburgh, Pennsylvania, 15260, United States
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Kaohsiung Medical University Chung-Ho Memorial Hospital (KMUH)
RECRUITINGKaohsiung City, Sanmin District, 80756, Taiwan
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King Faisal Specialist Hospital and Research Center
RECRUITINGRiyadh, 11471, Saudi Arabia
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Kyung Hee University Hospital at Gangdong
RECRUITINGSeoul, 05278, South Korea
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Kyungpook National University Hospital
RECRUITINGDaegu, 41944, South Korea
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McMaster University - Hamilton
RECRUITINGHamilton, Ontario, L8N 3Z5, Canada
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National Taiwan University Hospital
RECRUITINGTaipei, 100225, Taiwan
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National University Hospital
RECRUITINGSingapore, 110974, Singapore
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Prince of Wales Hospital Chinese University of Hong Kong
RECRUITINGShatin, 999077, Hong Kong
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Queen Mary Hospital
RECRUITINGHong Kong, 999077, Hong Kong
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Royal Adelaide Hospital
RECRUITINGAdelaide, South Australia, 5000, Australia
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Royal Brisbane Hospital
RECRUITINGHerston, Queensland, 4029, Australia
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Royal Prince Alfred Hospital
RECRUITINGCamperdown, New South Wales, 2050, Australia
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Severance Hospital, Yonsei University Health System
RECRUITINGSeoul, 3722, South Korea
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Sheba Medical Center
RECRUITINGTel Litwinsky, 5265601, Israel
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Singapore General Hospital
RECRUITINGSingapore, 169608, Singapore
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Taichung Veterans General Hospital -
RECRUITINGTaichung, 40705, Taiwan
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The Alfred Hospital
RECRUITINGMelbourne, Victoria, 3004, Australia
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Tri-Service General Hospital
RECRUITINGTaipei, Neihu District, 114, Taiwan
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University of California, San Diego (UCSD)
RECRUITINGSan Diego, California, 92121, United States
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University of Michigan
RECRUITINGAnn Arbor, Michigan, 48109, United States
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Özel Acibadem Adana Hastanesi
RECRUITINGSeyhan, 01130, Turkey (Türkiye)
Contact