A Prospective Study to Evaluate Disease Characteristics in Hemophilia B Participants Receiving Prophylaxis With Standard of Care FIX Replacement Therapy

Status: Recruiting
Location: See all (22) locations...
Intervention Type: Other
Study Type: Observational
SUMMARY

This study is focused on males who have Hemophilia B and who need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent and also to control their bleeding events. The aim of the study is to gather at least 6 months of information on bleeding events for each individual participant while they continue to use their usual FIX replacement therapy. There is no experimental treatment being tested in this study. The study is informational, and part of a larger program to understand and treat Hemophilia B with a potential experimental new therapy in the future. There is no obligation to agree to taking part in this future study. The study is looking to answer several other research questions to help understand each participant's individual disease characteristics, including: * How often to use FIX replacement therapy, both on a regular basis (prophylaxis) and as needed to treat bleeding events * Measurement of FIX activity (factor IX is a clotting factor) by different laboratories using different types of tests in Hemophilia B participants * Possible complications from the FIX replacement therapy the patient receives (usual standard of care will continue to be used) * How quality of life is affected by Hemophilia B * How joint health is affected by Hemophilia B * How often the participant visits the emergency room, urgent care center, physician's office, hospital, or has a telemedicine visit as a result of bleeding events * Whether the body makes antibodies (a protein produced by the body's immune system) against the FIX replacement therapy you receive, which could make the drug less effective or could lead to side effects

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 16
Healthy Volunteers: f
View:

• Previous experience with FIX therapy (≥50 documented exposure days to a FIX protein product such as recombinant, plasma-derived or extended half-life FIX product) with a current stable prophylaxis regimen for \>2 months prior to enrollment and intention to use FIX replacement therapy for the duration of the study

• No known hypersensitivity to FIX replacement product

• Willing to be contacted about a potential future clustered regularly interspaced short palindromic repeats (CRISPR)-based Factor 9 (F9) gene insertion clinical trial in which they may have the opportunity to screen for enrollment

Locations
United States
California
Orthopaedic Hemophilia Treatment Center
COMPLETED
Los Angeles
Colorado
University of Colorado Hemophilia and Thrombosis Center
RECRUITING
Aurora
Connecticut
Yale HTC
RECRUITING
New Haven
Washington, D.c.
Georgetown University Medical Center
RECRUITING
Washington D.c.
Florida
University of Florida
RECRUITING
Gainesville
Illinois
Rush University Medical Center
RECRUITING
Chicago
Indiana
Indiana Hemophilia and Thrombosis Center
RECRUITING
Indianapolis
Michigan
University of Michigan
RECRUITING
Ann Arbor
Ohio
University Hospitals Cleveland Medical Center
RECRUITING
Cleveland
Nationwide Children's Hospital
RECRUITING
Columbus
Pennsylvania
Children's Hospital of Philadelphia (CHOP)
RECRUITING
Philadelphia
Tennessee
Vanderbilt University Medical Center
RECRUITING
Nashville
Texas
University of Texas Health Science Center at Houston
RECRUITING
Houston
Other Locations
Canada
McMaster University Medical Centre - Hamilton Health Sciences
RECRUITING
Hamilton
Mcgill University Health Center (MUHC)
RECRUITING
Montreal
Germany
Klinikum der Johann Wolfgang Goethe-Universitaet Frankfurt
RECRUITING
Frankfurt
University Hospital Hamburg Eppendorf
RECRUITING
Hamburg
University Hospital of Regensburg
RECRUITING
Regensburg
United Kingdom
Glasgow Royal Infirmary - Clinical Research Facility
RECRUITING
Glasgow
Barts Health NHS Trust, Royal London Hospital
RECRUITING
London
St. Thomas' Hospital
RECRUITING
London
Southampton General Hospital
RECRUITING
Southampton
Contact Information
Primary
Clinical Trials Administrator
clinicaltrials@regeneron.com
844-734-6643
Time Frame
Start Date: 2024-01-17
Estimated Completion Date: 2026-04-02
Participants
Target number of participants: 120
Treatments
Cohort 1
Male participants with hemophilia B on current FIX Replacement Therapy prophylaxis or a documented genotype known to produce severe hemophilia B
Related Therapeutic Areas
Sponsors
Leads: Regeneron Pharmaceuticals

This content was sourced from clinicaltrials.gov