Hemophilia BHoney-BNCT05568459
A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy
An observational study of Hemophilia B, run by Regeneron Pharmaceuticals at 17 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 years and older, men only
- Conditions the study is for
- Hemophilia B
- What is being tested?
- Non-Interventional
- How many people
- 11 participants
- Where
- 17 sitesin 4 countries
- Study dates
- 17 Jan 2024to 21 Aug 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
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
Who may be able to join
- 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
Who cannot join
- 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
Show all 16Show fewer
- 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
- NOTE: Other Inclusion/Exclusion Protocol Defined Criteria Apply
Trial locations and contacts
17 sites in 4 countries.
17 sites in 4 countries
United States9 sites
University of Michigan
Ann Arbor, Michigan, 48109
University of Colorado Hemophilia and Thrombosis Center
Aurora, Colorado, 80045
Rush University Medical Center
Chicago, Illinois, 60612
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of Florida
Gainesville, Florida, 32610
University of Texas Health Science Center at Houston
Houston, Texas, 77030
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, 46260
Yale HTC
New Haven, Connecticut, 06510
Georgetown University Medical Center
Washington D.C., District of Columbia, 20057
Germany3 sites
Klinikum der Johann Wolfgang Goethe-Universitaet Frankfurt
Frankfurt, 60590
University Hospital Hamburg Eppendorf
Hamburg, 20246
University Hospital of Regensburg
Regensburg, Bavaria, 93053
United Kingdom3 sites
Barts Health NHS Trust, Royal London Hospital
London, E1 1FR
St. Thomas' Hospital
London, SE1 7EH
Southampton General Hospital
Southampton, Hampshire, SO16 6YD
Canada2 sites
McMaster University Medical Centre - Hamilton Health Sciences
Hamilton, Ontario, L8N 3Z5
Mcgill University Health Center (MUHC)
Montreal, Quebec, H4A 3J1
About this study
Official title: 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
- Conditions
- Hemophilia B
- Being tested
- Non-Interventional
- Keywords
- FIX replacement therapy
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Non-InterventionalOther
No study treatment will be administered in this study.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Male participants with hemophilia B on current FIX Replacement Therapy prophylaxis or a documented genotype known to produce severe hemophilia B Receives: Other: Non-Interventional |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized bleeding rate (ABR)
Measured: At least 26 Weeks Up to 96 weeks
Secondary outcomes (13)
Annualized utilization (IU/kg) of FIX replacement therapy
Measured: Up to 96 Weeks
FIX functional (coagulant) activity (FIX:C) in participants on prophylaxis FIX replacement therapy
FIX:C includes pre-dose and average activity
Measured: 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
Measured: 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
Measured: 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
Measured: Through the end of the study, approximately 96 weeks
Incidence of adverse event (AEs)
Measured: Through the end of the study, approximately 96 Weeks
Severity of AEs
Measured: Through the end of the study, approximately 96 Weeks
Hemophilia Quality of Life (Haemo-Qol-A) total and individual domain scores
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.
Measured: Up to 96 Weeks
Hemophilia Activities List (HAL) total and individual scores
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.
Measured: Up to 96 Weeks
European Quality of Life 5 Dimensions 3-levels (EQ-5D-3L) score
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.
Measured: Up to 96 Weeks
Hemophilia Joint Health Score (HJHS) total and individual scores per joint
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.
Measured: Up to 96 Weeks
Annualized medically attended visit (MAV) rate for any reason
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
Measured: Up to 96 Weeks
Detection of antibodies to adeno-associated virus (AAV) capsid proteins
Measured: Up to 96 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Oct 2022
- Study start17 Jan 2024
- Primary completion21 Aug 2026 (estimated)
- Study completion21 Aug 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia BStudies of the same condition, open and under way.
- More trials run by Regeneron PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Regeneron Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trial Management, Study director, Regeneron Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05568459
- Sponsor's protocol ID
- R0000-HEMB-2187
- Acronym
- Honey-B
- Status last verified
- Jul 2026
- First submitted
- 23 Sep 2022
- Last updated
- 7 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Study to Evaluate Disease Characteristics in Hemophilia B Participants Receiving Prophylaxis With Standard of Care FIX Replacement Therapy
- ClinicalTrials.gov ID
- NCT05568459
- Acronym / sponsor ID
- Honey-B / R0000-HEMB-2187
- Conditions
- Hemophilia B
- Keywords
- FIX replacement therapy
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 17 Jan 2024 (actual)
- Primary completion
- 21 Aug 2026 (estimated)
- Study completion
- 21 Aug 2026 (estimated)
- First posted
- 5 Oct 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | – | Other: Non-InterventionalMale participants with hemophilia B on current FIX Replacement Therapy prophylaxis or a documented genotype known to produce severe hemophilia B |
Who may qualify
- Ages
- 16 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (16 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Non-InterventionalNo study treatment will be administered in this study. | Other | Cohort 1 |
Primary outcome
Annualized bleeding rate (ABR)
Time frame: At least 26 Weeks Up to 96 weeks
13 secondary outcomes
Annualized utilization (IU/kg) of FIX replacement therapy
Time frame: Up to 96 Weeks
FIX functional (coagulant) activity (FIX:C) in participants on prophylaxis FIX replacement therapy
Time frame: 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
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
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
Incidence of adverse event (AEs)
Time frame: Through the end of the study, approximately 96 Weeks
Severity of AEs
Time frame: Through the end of the study, approximately 96 Weeks
Hemophilia Quality of Life (Haemo-Qol-A) total and individual domain scores
Time frame: Up to 96 Weeks
Hemophilia Activities List (HAL) total and individual scores
Time frame: Up to 96 Weeks
European Quality of Life 5 Dimensions 3-levels (EQ-5D-3L) score
Time frame: Up to 96 Weeks
Hemophilia Joint Health Score (HJHS) total and individual scores per joint
Time frame: Up to 96 Weeks
Annualized medically attended visit (MAV) rate for any reason
Time frame: Up to 96 Weeks
Detection of antibodies to adeno-associated virus (AAV) capsid proteins
Time frame: Up to 96 Weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2026.
- Individual participant dataSharing planned: Yes. All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
Related trials for Hemophilia B
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
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Not yet recruitingPhase 1
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RecruitingPhase 3
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trial Management study director
Regeneron Pharmaceuticals
17 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia B, run by Regeneron Pharmaceuticals at 17 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 7 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States, Germany, United Kingdom, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized bleeding rate (ABR) (time frame: At least 26 Weeks Up to 96 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Aug 2026.