One-Shot gene therapy aims to free hemophilia patients from constant treatments
NCT ID NCT03307980
Summary
This study is testing whether a single gene therapy treatment can provide long-term control of Hemophilia B, a bleeding disorder. It follows men who received the therapy in an earlier trial to see how long the benefits last and monitors for any delayed side effects. A small group of new participants will also receive a higher dose to see if it's safe and more effective.
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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.