Gene therapy breakthrough offers hope for hemophilia b patients
NCT ID NCT03307980
First seen Nov 01, 2025 · Last updated May 21, 2026 · Updated 28 times
Summary
This study tests a gene therapy (PF-06838435) for men with severe hemophilia B, a bleeding disorder. It aims to see if a single treatment can safely help the body produce its own clotting factor, reducing or stopping the need for regular infusions. Participants are followed for years to monitor long-term safety and how well the therapy works.
Disclaimer
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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.
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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Istanbul Universitesi Onkoloji Enstitusu Çocuk Hematoloji Onkoloji Bilim Dali
Istanbul, 34093, Turkey (Türkiye)
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Istanbul University Clinical Trials Excellence Center
Istanbul, 34452, Turkey (Türkiye)
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LA Center for Bleeding and Clotting Disorders
New Orleans, Louisiana, 70112, United States
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LA Center for Bleeding and Clotting Disorders - Metairie
Metairie, Louisiana, 70001, United States
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Louisiana Center for Advanced Medicine
Slidell, Louisiana, 70461, United States
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McGill University Health Center - Research Institute
Montreal, Quebec, H4A 3J1, Canada
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McMaster University
Hamilton, Ontario, L8S 3L8, 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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The Research Institute of the McGill University Health Centre
Montreal, Quebec, H3H 2R9, Canada
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Tulane Lakeside Hospital
Metairie, Louisiana, 70001, United States
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Tulane University Clinical Translational Unit
New Orleans, Louisiana, 70112, United States
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Tulane University Hospitals and Clinic
New Orleans, Louisiana, 70112, United States
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Tulane University School of Medicine
New Orleans, Louisiana, 70112, United States
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UC Davis Comprehensive Cancer Center
Sacramento, California, 95817, 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 Davis Midtown Cancer Center
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.