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

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.

Conditions

Hemophilia A

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Study Overview

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Study Details

Study overview

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.

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

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

Condition
Hemophilia A
Intervention / Treatment

-

Contacts and Locations

Los Angeles

Orthopedic Hemophilia Treatment Center, Los Angeles, California, United States, 90007

San Diego

Hemophilia and Thrombosis Treatment Center, University of California, San Diego, San Diego, California, United States, 92092

San Antonio

The University of Texas Health Science Center at San Antonio, San Antonio, Texas, United States, 78229

Seattle

Washington Center for Bleeding Disorders at Washington Institute for Coagulation, Seattle, Washington, United States, 98101

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.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

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

Ages Eligible for Study

16 Years to

Sexes Eligible for Study

MALE

Accepts Healthy Volunteers

No

Collaborators and Investigators

Washington Institute for Coagulation,

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

Study Record Dates

2026-08