One-Shot gene therapy aims to free hemophilia patients from lifelong injections
NCT ID NCT01687608
Summary
This early-stage trial is testing a single injection of gene therapy for adults with hemophilia B. The goal is to see if the treatment is safe and if it can help the body produce its own missing clotting factor, potentially reducing or eliminating the need for regular injections. The study will enroll up to 16 men to test different dose levels.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for HEMOPHILIA B are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
BloodCenter of Wisconsin
Milwaukee, Wisconsin, 53226, United States
-
Bloodworks Northwest
Seattle, Washington, 98104, United States
-
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
-
Children's Hospital of Boston
Boston, Massachusetts, 022105, United States
-
Emory University
Atlanta, Georgia, 30322, United States
-
Gulf States Hemophilia and Thrombosis Center
Houston, Texas, 77030, United States
-
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
-
Mount Sinai Medical Center
New York, New York, 10029, United States
-
Orthopaedic Hemophilia Treatment Center
Los Angeles, California, 90007, United States
-
Rush University Medical Center
Chicago, Illinois, 60612, United States
-
The Hemophilia Center, Oregon Health and Science University
Portland, Oregon, 97239, United States
-
U of Colorado School of Medicine, Hemophilia & Thrombosis Treatment Center
Aurora, Colorado, 80045, United States
-
University of California Davis Medical Center
Sacramento, California, 95817, United States
-
University of California at San Diego Medical Center
San Diego, California, 92103-8651, United States
-
University of Minnesota, Masonic Clinical Research Unit, Clinical and Translational Science Institute
Minneapolis, Minnesota, 55455, United States
Conditions
Explore the condition pages connected to this study.