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

Phase

N/A

Condition

Hemophilia

Treatment

assessment of joint health and bone density

Clinical Study ID

NCT04131036

ML40714

Ages > 16
Male

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion 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 thecurrent 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 repositorybiomarkers
Willing to come in for baseline and 3 yearly visits
Willing to answer phone survey for bleeding and safety every 3 months

Exclusion

Exclusion Criteria:

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)

Study Design

assessment of joint health and bone density

April 04, 2019

August 31, 2026

Study Description

Summary: This retrospective/prospective, non-randomized controlled study, whereby patients will be included in 1 of 2 groups depending on their current treatments as outlined below. Patients will be assigned to a specific group by the clinician/PI based on their current regimen with the intent to stay on this regimen for the next 3 years.

Duration of Study and Subject Participation: Study subject enrollment is anticipated to occur over about 12 months and study duration per subject will be 3 years. Thus a total study duration of 4 years is anticipated.

Intervention: By using musculoskeletal ultrasound, the investigators will measure joint health among participants and assess joint health changes over 3 years compared between group A (factor prophylaxis) vs. group B (emicizumab prophylaxis) as assessed by MSKUS at baseline and after 3 years. Other outcomes such as bone density, comparative assessment of joint and overall health status, and exploration of potential biomarkers for joint and bone health will also be assessed.

Connect with a study center

Orthopedic Hemophilia Treatment Center
Los Angeles, California 90007
United States
Site Not Available
Hemophilia and Thrombosis Treatment Center, University of California, San Diego
San Diego, California 92092
United States
Active - Recruiting
Tulane University
New Orleans, Louisiana 70112
United States
Site Not Available
The University of Texas Health Science Center at San Antonio
San Antonio, Texas 78229
United States
Active - Recruiting
Washington Center for Bleeding Disorders at Washington Institute for Coagulation
Seattle, Washington 98101
United States
Active - Recruiting

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.