Gene therapy offers hope for hemophilia b patients
NCT ID NCT03307980
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a gene therapy (PF-06838435) for people with severe hemophilia B, a bleeding disorder. The therapy uses a harmless virus to deliver a working gene for clotting factor IX. Researchers are following 21 participants long-term to see if it is safe and reduces bleeding. Some participants receive a higher dose in a substudy.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
PF-06838435 (a gene therapy using a modified virus to deliver a working factor IX gene)
What this could lead to
If successful, this gene therapy could allow people with hemophilia B to produce their own clotting factor, reducing or eliminating the need for regular infusions.
What could go wrong
This is a small, early-phase study (21 participants) focused on safety. Gene therapy carries risks like immune reactions or the treatment not lasting long-term. It may not work for everyone.
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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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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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