ACTIVE_NOT_RECRUITING

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

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Conditions

Hemophilia BFIX replacement therapy

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Official Title

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

Quick Facts

Study Start:2024-01-17
Study Completion:2026-01-21
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT05568459

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.

Ages Eligible for Study:16 Years
Sexes Eligible for Study:MALE
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. 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. 2. No known hypersensitivity to FIX replacement product
  3. 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
  1. 1. History of any coagulation disorder; requires anticoagulant therapy
  2. 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. 3. History of FIX inhibitor (clinical or laboratory-based assessment) on 2 or more occasions, as defined in the protocol
  4. 4. Bethesda inhibitor titer greater than the upper limit of normal (ULN) at screening
  5. 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. 6. Is positive for hepatitis B or C at screening, as defined in protocol
  7. 7. If any of the following pre-existing diagnoses are documented:
  8. * Cholestatic liver disease
  9. * Liver cirrhosis
  10. * Portal hypertension; or
  11. * Splenomegaly; or
  12. * Hepatic encephalopathy
  13. 8. History of arterial or venous thrombo-embolic events, as defined in the protocol
  14. 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
  15. 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

Contacts and Locations

Principal Investigator

Clinical Trial Management
STUDY_DIRECTOR
Regeneron Pharmaceuticals

Study Locations (Sites)

University of Colorado Hemophilia and Thrombosis Center
Aurora, Colorado, 80045
United States
Yale HTC
New Haven, Connecticut, 06510
United States
Georgetown University Medical Center
Washington D.C., District of Columbia, 20057
United States
University of Florida
Gainesville, Florida, 32610
United States
Rush University Medical Center
Chicago, Illinois, 60612
United States
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, 46260
United States
University of Michigan
Ann Arbor, Michigan, 48109
United States
Nationwide Children's Hospital
Columbus, Ohio, 43205
United States
University of Texas Health Science Center at Houston
Houston, Texas, 77030
United States

Collaborators and Investigators

Sponsor: Regeneron Pharmaceuticals

  • Clinical Trial Management, STUDY_DIRECTOR, Regeneron Pharmaceuticals

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 Registration Dates

Study Start Date2024-01-17
Study Completion Date2026-01-21

Study Record Updates

Study Start Date2024-01-17
Study Completion Date2026-01-21

Terms related to this study

Keywords Provided by Researchers

  • FIX replacement therapy

Additional Relevant MeSH Terms

  • Hemophilia B