Brief Title
Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A
Official Title
Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A
Brief 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.
Detailed 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.
Study Type
Observational
Primary Outcome
joint health comparison
Secondary Outcome
bone density comparison
Condition
Hemophilia A
Intervention
assessment of joint health and bone density
Study Arms / Comparison Groups
Arm A
Description: Male patients with severe Hemophilia A who use prophylaxis with IV factor VIII concentrate with intended trough >1%.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Other
Estimated Enrollment
40
Start Date
April 4, 2019
Completion Date
August 2023
Primary Completion Date
August 2023
Eligibility Criteria
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 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 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)
Gender
Male
Ages
16 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Rebecca Kruse-Jarres, MD, MPH, (206) 292-6500, [email protected]
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT04131036
Organization ID
ML40714
Responsible Party
Principal Investigator
Study Sponsor
Bloodworks
Collaborators
Genentech, Inc.
Study Sponsor
Rebecca Kruse-Jarres, MD, MPH, Principal Investigator, Bloodworks NW
Verification Date
October 2019