A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy

NCT05568459 | Active Not Recruiting

• 1 other identifier: R0000-HEMB-2187
• Study Type: observational
• Target: 11
• Locations: 4 countries
• Sites: 17

Brief 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

Conditions

Hemophilia B

Keywords

FIX replacement therapy

Outcome Measures

Primary Outcomes (1)

• Annualized bleeding rate (ABR)

  At least 26 Weeks Up to 96 weeks

Secondary Outcomes (13)

• Annualized utilization (IU/kg) of FIX replacement therapy

  Up to 96 Weeks

• FIX functional (coagulant) activity (FIX:C) in participants on prophylaxis FIX replacement therapy

  Through the end of the study, approximately 96 Weeks

• Difference of FIX:C in participants on prophylaxis FIX replacement therapy by one-stage and chromogenic assays

  Through the end of the study, approximately 96 weeks

• Difference of FIX:C between one-stage assay and chromogenic substrate assay by laboratory in participants on prophylaxis FIX replacement therapy

  Through the end of the study, approximately 96 weeks

• Difference of FIX:C between laboratories by assay (one-stage assay and chromogenic substrate assay) in participants on prophylaxis FIX replacement therapy

  Through the end of the study, approximately 96 weeks

Study Arms (1)

Cohort 1

Male participants with hemophilia B on current FIX Replacement Therapy prophylaxis or a documented genotype known to produce severe hemophilia B

Other: Non-Interventional

Interventions

Non-Interventional OTHER

No study treatment will be administered in this study.

Cohort 1

Eligibility Criteria

• Age: 16 Years+
• Gender Eligibility Details: Male with moderately severe to severe Hemophilia B with medical history of FIX functional activity/ FIX:C (≤2% or \<0.02 IU/mL) or a documented genotype known to produce severe hemophilia B
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Male patients with Hemophilia B with a history of FIX:C ≤2% or a documented genotype known to produce severe hemophilia B on current FIX Replacement Therapy prophylaxis

You may qualify if:

• 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

You may not qualify if:

• History of any coagulation disorder; requires anticoagulant therapy
• Lack of adherence with documentation of bleeds and/or prophylaxis replacement therapy administration in the opinion of the investigator, based on medical history
• History of FIX inhibitor (clinical or laboratory-based assessment) on 2 or more occasions, as defined in the protocol
• Bethesda inhibitor titer greater than the upper limit of normal (ULN) at screening
• 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
• Is positive for hepatitis B or C at screening, as defined in protocol
• If any of the following pre-existing diagnoses are documented:
• Cholestatic liver disease
• Liver cirrhosis
• Portal hypertension; or
• Splenomegaly; or
• Hepatic encephalopathy
• History of arterial or venous thrombo-embolic events, as defined in the protocol
• 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
• 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

Sponsors & Collaborators

• Regeneron Pharmaceuticals: lead

Study Sites (17)

University of Colorado Hemophilia and Thrombosis Center

Aurora, Colorado, 80045, United States

Location

Yale HTC

New Haven, Connecticut, 06510, United States

Location

Georgetown University Medical Center

Washington D.C., District of Columbia, 20057, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Indiana Hemophilia and Thrombosis Center

Indianapolis, Indiana, 46260, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

McMaster University Medical Centre - Hamilton Health Sciences

Hamilton, Ontario, L8N 3Z5, Canada

Location

Mcgill University Health Center (MUHC)

Montreal, Quebec, H4A 3J1, Canada

Location

University Hospital of Regensburg

Regensburg, Bavaria, 93053, Germany

Location

Klinikum der Johann Wolfgang Goethe-Universitaet Frankfurt

Frankfurt, 60590, Germany

Location

University Hospital Hamburg Eppendorf

Hamburg, 20246, Germany

Location

Southampton General Hospital

Southampton, Hampshire, SO16 6YD, United Kingdom

Location

Barts Health NHS Trust, Royal London Hospital

London, E1 1FR, United Kingdom

Location

St. Thomas' Hospital

London, SE1 7EH, United Kingdom

Location

MeSH Terms

Conditions

Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Officials

• Clinical Trial Management

  Regeneron Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 23, 2022
• First Posted: October 5, 2022
• Study Start: January 17, 2024
• Primary Completion: May 21, 2026
• Study Completion: May 21, 2026
• Last Updated: March 19, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: When Regeneron has * received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication or has globally discontinued development of the product for all indications on or after April 2020 and has no plans for future development * made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry) * the legal authority to share the data, and * ensured the ability to protect participant privacy
• Access Criteria: Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf

Locations

CA (2); DE (3); GB (3); US (9)