RECRUITING

Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A

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

The investigators propose to study longitudinal joint and bone density changes in patients with severe Hemophilia A. Per current standard of care, most patients are on prophylactic FVIII replacement therapy intravenously several times weekly with a goal of keeping the trough \>1% FVIII. Recent phase 3 data suggest superior bleed protection with emicizumab prophylaxis every 1-2 weeks. It is the purpose of this study to longitudinally assess joint health and bone density over 3 years and to compare the effect of routine factor VIII prophylaxis with emicizumab prophylaxis.

Official Title

Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A

Quick Facts

Study Start:2019-04-04
Study Completion:2026-08
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04131036

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. * Male gender
  2. * Severe hemophilia A (factor VIII \< 1%)
  3. * Age ≥ 16 year
  4. * Either on prophylaxis with factor VIII or emicizumab with the intention to stay on the current regimen for the next 3 years
  5. * Willing and able to give written informed consent/assent
  6. * Willing to undergo MSKUS, DEXA scan +/- collection of blood sampling for repository biomarkers
  7. * Willing to come in for baseline and 3 yearly visits
  8. * Willing to answer phone survey for bleeding and safety every 3 months
  1. * Current FVIII inhibitor of \> 0.6 BU
  2. * Unable to take FVIII replacement
  3. * Other known bleeding disorder
  4. * Other rheumatologic disorder affecting joints
  5. * Other known neuromotor defect (making physical exam difficult)

Contacts and Locations

Study Contact

Rebecca Kruse-Jarres, MD, MPH
CONTACT
206-614-1200
RKJ@WACBD.org
Cassandra Bryan
CONTACT
206-681-9072
cassandra.bryan@wacbd.org

Principal Investigator

Rebecca Kruse-Jarres, MD, MPH
PRINCIPAL_INVESTIGATOR
Washington Institute for Coagulation

Study Locations (Sites)

Orthopedic Hemophilia Treatment Center
Los Angeles, California, 90007
United States
Hemophilia and Thrombosis Treatment Center, University of California, San Diego
San Diego, California, 92092
United States
The University of Texas Health Science Center at San Antonio
San Antonio, Texas, 78229
United States
Washington Center for Bleeding Disorders at Washington Institute for Coagulation
Seattle, Washington, 98101
United States

Collaborators and Investigators

Sponsor: Washington Institute for Coagulation

  • Rebecca Kruse-Jarres, MD, MPH, PRINCIPAL_INVESTIGATOR, Washington Institute for Coagulation

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 Date2019-04-04
Study Completion Date2026-08

Study Record Updates

Study Start Date2019-04-04
Study Completion Date2026-08

Terms related to this study

Keywords Provided by Researchers

  • Hemophilia A
  • Joint Health
  • Bone Density
  • Emicizumab
  • factor VIII prophylaxis

Additional Relevant MeSH Terms

  • Hemophilia A