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

May 3, 2024 updated by: Regeneron Pharmaceuticals

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

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

120

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Canada
    • Ontario
      • Hamilton, Ontario, Canada, L8N 3Z5
        • Recruiting
        • McMaster University Medical Centre, Hamilton Health Sciences
        • Contact:
        • Contact:
  • Germany
      • Hamburg, Germany, 20246
        • Recruiting
        • University Hospital Hamburg-Eppendorf
        • Contact:
          • Katharina Holstein, Dr.
          • Phone Number: +49 40 7410 52453
    • Hesse
      • Frankfurt, Hesse, Germany, 60590
        • Recruiting
        • University Hopsital Frankfurt
        • Contact:
          • Steffen Rauchfuß
          • Phone Number: +49 6963014246
  • United Kingdom
      • Glasgow, United Kingdom, G4 0SF
        • Recruiting
        • NHS Greater Glasgow and Cylde: Glasgow Royal Infirmary
        • Contact:
      • London, United Kingdom, SE1 7EH
      • London, United Kingdom, E1 1FR
        • Recruiting
        • Barts Health NHS Trust, Royal Lond Hospital
        • Contact:
          • Phone Number: (+44) 020 324 60261
    • Hampshire
      • Southampton, Hampshire, United Kingdom, SO16 6YD
  • United States
    • California
      • Los Angeles, California, United States, 90007
        • Recruiting
        • Orthopaedic Hemophilia Treatment Center
        • Principal Investigator:
          • Dr. Doris Quon, MD, PhD
        • Contact:
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • University of Colorado Hemophilia & Thrombosis Center
        • Contact:
    • District of Columbia
      • Washington, District of Columbia, United States, 20007
        • Recruiting
        • Georgetown University
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Recruiting
        • Indiana Hemophilia and Thrombosis Center
        • Contact:
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • University of Michigan
        • Contact:
    • Ohio
      • Cleveland, Ohio, United States, 44106
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hosptial of Philadelphia
        • Contact:
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Recruiting
        • Vanderbilt University Medical Center
        • Contact:
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • University of Texas Health Science Center at Houston
        • Contact:
          • Miguel Escobar
          • Phone Number: 713-500-8360

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years to 65 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

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

Description

Key Inclusion Criteria:

  1. 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
  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

Key Exclusion Criteria:

  1. History of any coagulation disorder; requires anticoagulant therapy
  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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
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
No study treatment will be administered in this study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Annualized bleeding rate (ABR)
Time Frame: At least 26 Weeks Up to 96 weeks
At least 26 Weeks Up to 96 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
FIX functional (coagulant) activity (FIX:C) in participants on prophylaxis FIX replacement therapy
Time Frame: Through the end of the study, approximately 96 Weeks
FIX:C includes pre-dose and average activity
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
Time Frame: Through the end of the study, approximately 96 weeks
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
Time Frame: Through the end of the study, approximately 96 weeks
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
Time Frame: Through the end of the study, approximately 96 weeks
Through the end of the study, approximately 96 weeks
European Quality of Life 5 Dimensions 3-levels (EQ-5D-3L) score
Time Frame: Up to 96 Weeks
EQ-5D-3L health questionnaire is a participant/relative answered questionnaire scoring 5 dimensions - mobility, self-care, usual activities, pain/discomfort and anxiety/depression. the status of each dimension had 3 possible responses (1 =no problem, 2 some problem 3 =severe problems) in the relevant health dimension. Higher score indicated a worsening health condition.
Up to 96 Weeks
Hemophilia Joint Health Score (HJHS) total and individual scores per joint
Time Frame: Up to 96 Weeks
The examination involves a physical assessment of elbow, knee, and ankle joints. Individual joints are scored based on duration and presence of swelling, joint pain, flexibility, muscle atrophy, strength, and overall gait. The HJHS is a validated 11-item scoring tool based on radiologic and clinical evaluation, sensitive to detect early signs and minor changes. HJHS ranges from 0 to 124. Higher values in the HJHS represent worse situation for the participant.
Up to 96 Weeks
Hemophilia Quality of Life (Haemo-Qol-A) total and individual domain scores
Time Frame: Up to 96 Weeks
The Haemo-QoL-A is a self-reported Hemophilia-specific health-related quality of life questionnaire which consists of 41 questions covering 6 domains (Physical Functioning, Role Functioning, Worry, Consequences of Bleeding, Emotional Impact, and Treatment Concerns). Items are answered on a 6-point Likert-type scale, ranging from 0 (None of the time) to 5 (All of the time). Higher scores mean better health-related quality of life or less impairment.
Up to 96 Weeks
Hemophilia Activities List (HAL) total and individual scores
Time Frame: Up to 96 Weeks
HAL measures of the impact of Hemophilia on functional abilities in adults. The questionnaire consists of 42 items across 7 domains. Each item is rated on a scale of 1 (Impossible) to 6 (Never). Higher scores represent lower levels of activity limitations.
Up to 96 Weeks
Annualized utilization (IU/kg) of FIX replacement therapy
Time Frame: Up to 96 Weeks
Up to 96 Weeks
Incidence of adverse event (AEs)
Time Frame: Through the end of the study, approximately 96 Weeks
Through the end of the study, approximately 96 Weeks
Severity of AEs
Time Frame: Through the end of the study, approximately 96 Weeks
Through the end of the study, approximately 96 Weeks
Annualized medically attended visit (MAV) rate for any reason
Time Frame: Up to 96 Weeks
The annual rate of MAVs including emergency room (ER) visits, urgent care center visits, physician's office visits, telemedicine visits, or hospitalizations related to bleeds treated on-demand with FIX replacement therapy
Up to 96 Weeks
Detection of antibodies to adeno-associated virus (AAV) capsid proteins
Time Frame: Up to 96 Weeks
Up to 96 Weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Regeneron Pharmaceuticals

Investigators

  • Study Director: Clinical Trial Management, Regeneron Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 17, 2024

Primary Completion (Estimated)

April 2, 2026

Study Completion (Estimated)

April 2, 2026

Study Registration Dates

First Submitted

September 23, 2022

First Submitted That Met QC Criteria

October 4, 2022

First Posted (Actual)

October 5, 2022

Study Record Updates

Last Update Posted (Actual)

May 7, 2024

Last Update Submitted That Met QC Criteria

May 3, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • R0000-HEMB-2187

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing

IPD Sharing 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, has made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry), has the legal authority to share the data, and has ensured the ability to protect participant privacy.

IPD Sharing 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

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • ANALYTIC_CODE
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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