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No new drug: study tracks bleeding in hemophilia b patients on usual care

NCT ID NCT05568459

Knowledge-focused Sponsor: Regeneron Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 07, 2026 · Updated 1 time

Summary

This study follows 11 males with Hemophilia B who already receive regular factor IX therapy to prevent bleeding. Researchers will collect information on bleeding events, treatment use, and quality of life over at least 6 months. No experimental treatment is given; the goal is to better understand the disease for future studies.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

11 people

The number who actually took part.

Started

Jan 2024

Expected to finish

Aug 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

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

Ages

16 years and older

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

hemophilia B

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hemophilia B

Contacts and locations

Locations

  • Barts Health NHS Trust, Royal London Hospital

    London, E1 1FR, United Kingdom

  • Georgetown University Medical Center

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

  • Indiana Hemophilia and Thrombosis Center

    Indianapolis, Indiana, 46260, United States

  • Klinikum der Johann Wolfgang Goethe-Universitaet Frankfurt

    Frankfurt, 60590, Germany

  • McMaster University Medical Centre - Hamilton Health Sciences

    Hamilton, Ontario, L8N 3Z5, Canada

  • Mcgill University Health Center (MUHC)

    Montreal, Quebec, H4A 3J1, Canada

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Rush University Medical Center

    Chicago, Illinois, 60612, United States

  • Southampton General Hospital

    Southampton, Hampshire, SO16 6YD, United Kingdom

  • St. Thomas' Hospital

    London, SE1 7EH, United Kingdom

  • University Hospital Hamburg Eppendorf

    Hamburg, 20246, Germany

  • University Hospital of Regensburg

    Regensburg, Bavaria, 93053, Germany

  • University of Colorado Hemophilia and Thrombosis Center

    Aurora, Colorado, 80045, United States

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Michigan

    Ann Arbor, Michigan, 48109, United States

  • University of Texas Health Science Center at Houston

    Houston, Texas, 77030, United States

  • Yale HTC

    New Haven, Connecticut, 06510, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.