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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04131036
Recruitment Status : Recruiting
First Posted : October 18, 2019
Last Update Posted : April 26, 2022
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by (Responsible Party):
Rebecca Kruse-Jarres, MD, MPH, Washington Institute for Coagulation

Study Description
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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.

Condition or disease Intervention/treatment
Hemophilia A Other: assessment of joint health and bone density

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 Design
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Study Type : Observational
Estimated Enrollment : 40 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A
Actual Study Start Date : April 4, 2019
Estimated Primary Completion Date : August 2023
Estimated Study Completion Date : August 2023

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
Arm A
Male patients with severe Hemophilia A who use prophylaxis with IV factor VIII concentrate with intended trough >1%.
 Other: assessment of joint health and bone density
We propose to study longitudinal joint and bone density changes in patients with severe Hemophilia A.
Arm B
Male patients with severe Hemophilia A who use prophylaxis with SQ emicizumab.
 Other: assessment of joint health and bone density
We propose to study longitudinal joint and bone density changes in patients with severe Hemophilia A.


Outcome Measures
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Primary Outcome Measures :
  1. joint health comparison [ Time Frame: 3 years ]
    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.


Secondary Outcome Measures :
  1. bone density comparison [ Time Frame: 3 years ]
    Bone density changes over 3 years compared between group A (factor prophylaxis) vs. group B (emicizumab prophylaxis) as assessed by DEXA scan at baseline and after 3 years.

  2. comparative assessment of joint and overall health [ Time Frame: 3 years ]
    Observational comparative assessment of joint and overall health status evaluated by activity level, functional assessment, pain assessment, joint examination, and adherence.

  3. mid-point assessment of joint health and bone density [ Time Frame: 2 years ]
    Observational assessment of joint health and bone density at 1 and 2 years in the different groups.

  4. biomarkers for joint and bone health [ Time Frame: through completion of study, average 3 years ]
    Exploration of potential biomarkers for joint and bone health.


Biospecimen Retention:   Samples With DNA
Plasma samples for biomarkers for storage in the data and sample research repository at Washington Institute for Coagulation

Eligibility Criteria
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Ages Eligible for Study:   16 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Male severe Hemophilia A patients at one of the several Hemophilia Treatment Center sites participating in this study and who meet the eligibility criteria and are interested in participating in the study.
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)
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04131036


Contacts
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Contact: Rebecca Kruse-Jarres, MD, MPH 206-614-1200 RKJ@WACBD.org

Locations
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United States, California
Orthopedic Hemophilia Treatment Center Not yet recruiting
Los Angeles, California, United States, 90007
Hemophilia and Thrombosis Treatment Center, University of California, San Diego Not yet recruiting
San Diego, California, United States, 92092
United States, Louisiana
Tulane University Not yet recruiting
New Orleans, Louisiana, United States, 70112
United States, Washington
Washington Center for Bleeding Disorders at Washington Institute for Coagulation Recruiting
Seattle, Washington, United States, 98101
Contact: Rebecca Kruse-Jarres, MD, MPH    206-614-1200    RKJ@WACBD.org   
Principal Investigator: Rebecca Kruse-Jarres, MD, MPH         
Sponsors and Collaborators
Washington Institute for Coagulation
Genentech, Inc.
Investigators
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Principal Investigator: Rebecca Kruse-Jarres, MD, MPH Washington Institute for Coagulation
More Information
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Publications:

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Responsible Party: Rebecca Kruse-Jarres, MD, MPH, Executive/Medical Director, Washington Center for Bleeding Disorders at Washington Institute for Coagulation, Washington Institute for Coagulation
ClinicalTrials.gov Identifier: NCT04131036    
Other Study ID Numbers: ML40714
First Posted: October 18, 2019    Key Record Dates
Last Update Posted: April 26, 2022
Last Verified: April 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Rebecca Kruse-Jarres, MD, MPH, Washington Institute for Coagulation:
Hemophilia A
Joint Health
Bone Density
Emicizumab
factor VIII prophylaxis
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn