A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy
NCT ID: NCT05568459
Last Updated: 2025-12-19
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
11 participants
OBSERVATIONAL
2024-01-17
2026-01-21
Brief Summary
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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
Detailed Description
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Conditions
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Keywords
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Cohort 1
Male participants with hemophilia B on current FIX Replacement Therapy prophylaxis or a documented genotype known to produce severe hemophilia B
Non-Interventional
No study treatment will be administered in this study.
Interventions
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Non-Interventional
No study treatment will be administered in this study.
Eligibility Criteria
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Inclusion Criteria
2. No known hypersensitivity to FIX replacement product
3. 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
Exclusion Criteria
2. Lack of adherence with documentation of bleeds and/or prophylaxis replacement therapy administration in the opinion of the investigator, based on medical history
3. History of FIX inhibitor (clinical or laboratory-based assessment) on 2 or more occasions, as defined in the protocol
4. Bethesda inhibitor titer greater than the upper limit of normal (ULN) at screening
5. Any detectable pre-existing antibodies to the Adeno-associated virus serotype 8 (AAV8) capsid; as measured by an assay at prescreening, as defined in the protocol
6. Is positive for hepatitis B or C at screening, as defined in protocol
7. If any of the following pre-existing diagnoses are documented:
* Cholestatic liver disease
* Liver cirrhosis
* Portal hypertension; or
* Splenomegaly; or
* Hepatic encephalopathy
8. History of arterial or venous thrombo-embolic events, as defined in the protocol
9. History of clinically significant cardiovascular, respiratory, hepatic, renal (including nephrotic syndrome), gastrointestinal (including protein-losing enteropathy), endocrine, hematological (including thrombophilia), psychiatric, or neurological disease, as assessed by the investigator that may confound the results of the study or poses an additional risk to the participant by study participation
10. Previously received of any AAV-gene based therapy with a marketed gene therapy or in a clinical trial or intent to receive approved or investigational AAV-gene based therapy during the study period
NOTE: Other Inclusion/Exclusion Protocol Defined Criteria Apply
16 Years
MALE
No
Sponsors
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Regeneron Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trial Management
Role: STUDY_DIRECTOR
Regeneron Pharmaceuticals
Locations
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University of Colorado Hemophilia and Thrombosis Center
Aurora, Colorado, United States
Yale HTC
New Haven, Connecticut, United States
Georgetown University Medical Center
Washington D.C., District of Columbia, United States
University of Florida
Gainesville, Florida, United States
Rush University Medical Center
Chicago, Illinois, United States
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, United States
University of Michigan
Ann Arbor, Michigan, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
University of Texas Health Science Center at Houston
Houston, Texas, United States
McMaster University Medical Centre - Hamilton Health Sciences
Hamilton, Ontario, Canada
Mcgill University Health Center (MUHC)
Montreal, Quebec, Canada
University Hospital of Regensburg
Regensburg, Bavaria, Germany
Klinikum der Johann Wolfgang Goethe-Universitaet Frankfurt
Frankfurt, , Germany
University Hospital Hamburg Eppendorf
Hamburg, , Germany
Southampton General Hospital
Southampton, Hampshire, United Kingdom
Barts Health NHS Trust, Royal London Hospital
London, , United Kingdom
St. Thomas' Hospital
London, , United Kingdom
Countries
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Other Identifiers
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R0000-HEMB-2187
Identifier Type: -
Identifier Source: org_study_id