Gene therapy breakthrough: hemophilia b patients may need fewer treatments
NCT ID NCT03307980
First seen Nov 01, 2025 · Last updated May 09, 2026 · Updated 27 times
Summary
This study looks at the long-term safety and effectiveness of a gene therapy (PF-06838435) for people with severe hemophilia B, a bleeding disorder. It follows patients who already received the therapy and also tests a higher dose in new participants. The goal is to see if the treatment can reduce or eliminate the need for regular factor IX infusions.
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This is a summary of
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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Contacts and locations
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Locations
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Ege Universitesi Tip Fakultesi Cocuk Sagligi ve Hastaliklari Anabilim Dali
Izmir, 35100, Turkey (Türkiye)
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McGill University Health Center - Research Institute
Montreal, Quebec, H4A 3J1, Canada
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McMaster University Medical Centre - Hamilton Health Sciences
Hamilton, Ontario, L8N 3Z5, Canada
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Mississippi Center for Advanced Medicine
Madison, Mississippi, 39110, United States
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Royal Prince Alfred Hospital
Camperdown, New South Wales, 2050, Australia
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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UC Davis Ellison Ambulatory Care Clinic
Sacramento, California, 95817, United States
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UC Davis Medical Center
Sacramento, California, 95817, United States
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UC Davis Medical Center department of Radiology
Sacramento, California, 95817, United States
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UC DavisHealth Main Hospital
Sacramento, California, 95817, United States
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University Medical Center New Orleans
New Orleans, Louisiana, 70112, United States
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Weill Cornell Medicine - New York Presbyterian Hospital
New York, New York, 10065, United States
Conditions
Explore the condition pages connected to this study.