Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis -STEP: SporTs Emicizumab Prophylaxis

Status: Recruiting
Location: See all (7) locations...
Intervention Type: Drug
Study Type: Observational
SUMMARY

Hemophilia A (HA) is a genetic bleeding disorder resulting from a deficiency or absence of factor VIII (FVIII), which is necessary in the clotting process. This disorder occurs mostly in males and in severe cases causes frequent bleeding episodes in joints and muscles which can lead to progressive damage that affects mobility and quality of life. Prophylactic FVIII administered intravenously every other day has been the standard of care treatment for HA for the past few decades. Sports and physical activity are generally encouraged in patients with hemophilia on appropriate prophylactic treatment to increase strength, prevent or decrease obesity, accrue and maintain bone density and encourage normal socialization. To ensure safety with participation in sports in persons with hemophilia A (PWHA), timing of FVIII administration is often adjusted to maximize FVIII at the time of sports. The exact factor level that is needed to safely participate in sports and minimize bleeding risk is not yet known. Based on clinical practice, infusion of FVIII to near the lower limit of normal right before participation in sports generally works to prevent bleeding. The study is looking at how well the newly approved medication Emicizumab works compared to Factor VIII to prevent bleeding in patients with Hemophilia A who play sports. The study will enroll children and adolescents who are already on Emicizumab or Factor VIII who are currently playing sports.

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 6
Maximum Age: 19
View:

• Participant (if 18 years of age or older) or parent/LAR is willing and able to provide written informed consent; minor participant is willing and able to provide assent, if applicable based on site and local regulations

• Male participants with moderate to severe Hemophilia A ( FVIII activity \</= 5%) between 6 to 19 years of age without inhibitors are eligible for participation in this study

• Participants must be on Emicizumab or standard FVIII prophylaxis per institutional/primary hematologist recommendations

• Participants must be engaging in sports activities with moderate to high risk of bleeding as defined by the NHF- Playing it Safe guidelines (numerical rating \>/= 2).

• Participants must be compliant with completing all standard of care bleed and treatment logs

• Participant must be willing to keep daily activity logs for the duration of the study.

Locations
United States
Arkansas
Arkansas Children's Hospital
RECRUITING
Little Rock
California
Stanford University
RECRUITING
Palo Alto
Florida
Johns Hopkins All Children's Hospital
RECRUITING
St. Petersburg
Indiana
Indiana Hemophilia & Thrombosis Center
RECRUITING
Indianapolis
Massachusetts
Boston Children's Hospital
RECRUITING
Boston
Michigan
Children's Hospital of Michigan
RECRUITING
Detroit
Ohio
Cincinnati Children's Hospital Medical Center
RECRUITING
Cincinnati
Contact Information
Primary
Amy Stolinski, MPH
stepstudy@wayne.edu
313-577-7500
Time Frame
Start Date: 2023-08-16
Estimated Completion Date: 2029-03
Participants
Target number of participants: 120
Treatments
Emicizumab Prophylaxis
This group will include patients on standard of care Emicizumab prophylaxis for Hemophilia A
FVIII Prophylaxis
This group will include patients on standard of care FVIII prophylaxis for Hemophilia A
Related Therapeutic Areas
Sponsors
Collaborators: Genentech, Inc.
Leads: Wayne State University

This content was sourced from clinicaltrials.gov