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

NCT05022459 | Recruiting FDA Drug

• 1 other identifier: STEP Study
• Study Type: observational
• Target: 72
• Locations: 1 country
• Sites: 9

Brief 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.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

• Number of bleeds

  Number of bleeds without trauma, during participation in sports activities

  2 years

Secondary Outcomes (5)

• Change in HJHS scores

  3 years

• Change in bone mineral density

  3 years

• Change in biomarkers of bone and joint health

  3 years

• Number of FVIII doses for breakthrough bleeding episodes

  3 years

• MRI changes in relevant joints

  3 years

Study Arms (2)

Emicizumab Prophylaxis

This group will include patients on standard of care Emicizumab prophylaxis for Hemophilia A

Drug: Emicizumab

FVIII Prophylaxis

This group will include patients on standard of care FVIII prophylaxis for Hemophilia A

Drug: FVIII

Interventions

FVIII DRUG

Patients in this group will be on standard of care FVIII prophylaxis for Hemophilia A

FVIII Prophylaxis

Emicizumab DRUG

Patients in this group will be on standard of care Emicizumab prophylaxis for Hemophilia A

Emicizumab Prophylaxis

Eligibility Criteria

• Age: 6 Years - 19 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

This study will enroll patients who are already on Emicizumab or FVIII prophylaxis as per standard of care and who are already participating in sports activities with moderate to high risk of bleeding.

You may qualify if:

• 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
• Males and females between 6 to ≤ 19 years of age at time of enrollment with moderate to severe Hemophilia A (FVIII activity ≤ 5%) 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 or registered to start participating in one or more sports activities with moderate to high risk of bleeding as defined by the NHF- Playing it Safe guidelines (numerical rating \>/= 2).
• Participant must be willing to keep activity, bleed, and treatment logs for the duration of the study

You may not qualify if:

• Participant/parent/LAR unwilling to provide informed consent/assent
• Unwilling to log or document bleeds and treatment information as per study guidelines
• Participants with any other bleeding disorders will be excluded
• Patients who are pregnant, planning to become pregnant, or breastfeeding should not be enrolled in the study
• Participants on concomittent FVIII replacement and emicizumab for sports participation

Sponsors & Collaborators

• Wayne State University: lead
• Genentech, Inc.: collaborator

Study Sites (9)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

RECRUITING

Stanford University

Palo Alto, California, 94304, United States

RECRUITING

Rady Children's Hospital

San Diego, California, 92123, United States

RECRUITING

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States

RECRUITING

Indiana Hemophilia & Thrombosis Center

Indianapolis, Indiana, 46260, United States

RECRUITING

Boston Children's Hospital

Boston, Massachusetts, 02215, United States

RECRUITING

Children's Hospital of Michigan

Detroit, Michigan, 48201, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

Oklahoma Center for Bleeding and Clotting Disorders

Oklahoma City, Oklahoma, 73104, United States

RECRUITING

Biospecimen

Retention: SAMPLES WITHOUT DNA

Participants will have the option to agree to allow any blood leftover from study testing to be stored for future research.

MeSH Terms

Conditions

Hemophilia A

Interventions

emicizumab

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Meera Chitlur, MD

  Wayne State University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Amy Stolinski, MPH

CONTACT

313-577-7500; stepstudy@wayne.edu

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Hemophilia Treatment Center and Hemostasis Program, Principal Investigator, Clinical Professor

Study Record Dates

• First Submitted: August 23, 2021
• First Posted: August 26, 2021
• Study Start: August 16, 2023
• Primary Completion (Estimated): January 1, 2029
• Study Completion (Estimated): March 1, 2029
• Last Updated: January 16, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (9)