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

August 31, 2023 updated by: Rebecca Kruse-Jarres, MD, MPH, Washington Institute for Coagulation
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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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

Enrollment (Estimated)

40

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Rebecca Kruse-Jarres, MD, MPH
  • Phone Number: 206-614-1200
  • Email: RKJ@WACBD.org

Study Contact Backup

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90007
        • Not yet recruiting
        • Orthopedic Hemophilia Treatment Center
      • San Diego, California, United States, 92092
        • Recruiting
        • Hemophilia and Thrombosis Treatment Center, University of California, San Diego
        • Contact:
        • Principal Investigator:
          • Annette Von Drygalski, MD, PharmD
    • Texas
      • San Antonio, Texas, United States, 78229
        • Recruiting
        • The University of Texas Health Science Center at San Antonio
        • Contact:
    • Washington
      • Seattle, Washington, United States, 98101
        • Recruiting
        • Washington Center for Bleeding Disorders at Washington Institute for Coagulation
        • Contact:
          • Rebecca Kruse-Jarres, MD, MPH
          • Phone Number: 206-614-1200
          • Email: RKJ@WACBD.org
        • Principal Investigator:
          • Rebecca Kruse-Jarres, MD, MPH

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.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Child, Adult, Older Adult)

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.

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
3 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
3 years
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 years
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.
2 years
biomarkers for joint and bone health
Time Frame: through completion of study, average 3 years
Exploration of potential biomarkers for joint and bone health.
through completion of study, average 3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Washington Institute for Coagulation

Collaborators

Genentech, Inc.

Investigators

  • Principal Investigator: Rebecca Kruse-Jarres, MD, MPH, Washington Institute for Coagulation

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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 Major Dates

Study Start (Actual)

April 4, 2019

Primary Completion (Estimated)

August 1, 2026

Study Completion (Estimated)

August 1, 2026

Study Registration Dates

First Submitted

October 14, 2019

First Submitted That Met QC Criteria

October 16, 2019

First Posted (Actual)

October 18, 2019

Study Record Updates

Last Update Posted (Actual)

September 5, 2023

Last Update Submitted That Met QC Criteria

August 31, 2023

Last Verified

August 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ML40714

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Hemophilia A

Clinical Trials on assessment of joint health and bone density

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Gabon

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe