Gene therapy offers hope for hemophilia a patients
NCT ID NCT03061201
First seen Sep 30, 2025 · Last updated May 23, 2026 · Updated 28 times
Summary
This study tested a new gene therapy (SB-525) in 13 adults with severe hemophilia A, a bleeding disorder. The goal was to see if a single infusion could safely raise the body's own clotting factor levels and reduce bleeding episodes. Participants were monitored for up to 5 years to track safety and how long the effect lasted.
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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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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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City of Hope Medical Center
Duarte, California, 91010, United States
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Emory University School of Medicine
Atlanta, Georgia, 30322, United States
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Hemophilia Center of Western PA
Pittsburgh, Pennsylvania, 15213, United States
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Midtown Ambulatory Care Center
Sacramento, California, 95817, United States
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UC Davis Ambulatory Care Clinic
Sacramento, California, 95817, United States
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UC Davis CTSC Clinical Research Center
Sacramento, California, 95817, United States
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UC Davis Comprehensive Cancer Center
Sacramento, California, 95817, United States
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UC Davis Hemophilia Treatment Center
Sacramento, California, 95817, United States
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UC Davis Investigational Drug Services Pharmacy
Sacramento, California, 95817, United States
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UC Davis Medical Center
Sacramento, California, 95817, United States
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UPMC Montefiore Clinical and Translational Research Center
Pittsburgh, Pennsylvania, 15213, United States
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UPMC, Investigational Drug Service
Pittsburgh, Pennsylvania, 15213, United States
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USF Health Morsani Center For Advanced Healthcare
Tampa, Florida, 33612, United States
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University Of Miami Hospital and Clinics/Sylvester Comprehensive Cancer Center
Miami, Florida, 33136, United States
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University of California, San Francisco - Investigational Drug Service (IDS) Pharmacy
San Francisco, California, 94143-0622, United States
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University of California, San Francisco - Outpatient Hematology Clinic
San Francisco, California, 94143, United States
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University of California, San Francisco -Moffitt Hospital
San Francisco, California, 94143, United States
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Vanderbilt Hemostasis Treatment Clinic
Nashville, Tennessee, 37232, United States
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Vanderbilt Hemostasis-Thrombosis Clinic
Nashville, Tennessee, 37232, United States
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Vanderbilt University Medical Center Clinical Research Center
Nashville, Tennessee, 37212-8646, United States
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Washington Institute for Coagulation
Seattle, Washington, 98101, United States
Conditions
Explore the condition pages connected to this study.