Recruiting

Emicizumab vs. Factor VIII

Sponsor:

Washington Institute for Coagulation

Code:

NCT04131036

Conditions

Hemophilia A

Eligibility Criteria

Sex: Male

Age: 16+

Healthy Volunteers: Not accepted

Interventions

assessment of joint health and bone density

Study Details

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.

Conditions

Hemophilia A

Study ID

NCT04131036

Start date

Apr 4, 2019

Status verified date

Aug, 2023

Completion date

Aug, 2026

Anticipated

Primary completion date

Aug, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 16+

Healthy Volunteers: Not accepted

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 Design

Enrollment

40 participants

Anticipated

Interventions and Outcome Measures

Arms

Arm A

Male patients with severe Hemophilia A who use prophylaxis with IV factor VIII concentrate with intended trough >1%.

Arm B

Male patients with severe Hemophilia A who use prophylaxis with SQ emicizumab.

Interventions

assessment of joint health and bone density

We propose to study longitudinal joint and bone density changes in patients with severe Hemophilia A.

Primary outcome measure

  • joint health comparison [ Time Frame: 3 years ]

Central Contacts and Locations

Central contacts

Rebecca Kruse-Jarres, MD, MPH

206-614-1200RKJ@WACBD.org

Cassandra Bryan

206-681-9072cassandra.bryan@wacbd.org

Locations

Hemophilia and Thrombosis Treatment Center, University of California, San Diego

Recruiting

San Diego, California, United States, 92092

Contacts

Isabel Chang

i3chang@health.ucsd.edu

Principal Investigator:

Annette Von Drygalski, MD, PharmD

The University of Texas Health Science Center at San Antonio

Recruiting

San Antonio, Texas, United States, 78229

Contacts

Jeffrey Kallberg, DPT

kallberg@uthscsa.edu

Washington Center for Bleeding Disorders at Washington Institute for Coagulation

Recruiting

Seattle, Washington, United States, 98101

Contacts

Rebecca Kruse-Jarres, MD, MPH

206-614-1200RKJ@WACBD.org

Principal Investigator:

Rebecca Kruse-Jarres, MD, MPH

More Information

Sponsor

Washington Institute for Coagulation

Last update posted

Sep 5, 2023

Last verified

Aug, 2023

Keywords

  • Hemophilia A
  • Joint Health
  • Bone Density
  • Emicizumab
  • factor VIII prophylaxis

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Washington Institute for Coagulation on 2023-09-05.