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

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ClinicalTrials.gov Identifier: NCT05022459
Recruitment Status : Not yet recruiting
First Posted : August 26, 2021
Last Update Posted : October 13, 2021
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by (Responsible Party):
Meera Chitlur, Wayne State University

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.


Condition or disease Intervention/treatment
Hemophilia A Drug: Emicizumab Drug: FVIII

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Study Type : Observational
Estimated Enrollment : 120 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: 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
Estimated Study Start Date : November 2021
Estimated Primary Completion Date : September 2024
Estimated Study Completion Date : October 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Bleeding Hemophilia
Drug Information available for: Emicizumab

Group/Cohort Intervention/treatment
Emicizumab Prophylaxis
This group will include patients on standard of care Emicizumab prophylaxis for Hemophilia A
Drug: Emicizumab
Patients in this group will be 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
Drug: FVIII
Patients in this group will be on standard of care FVIII prophylaxis for Hemophilia A




Primary Outcome Measures :
  1. Number of bleeds [ Time Frame: 3 years ]
    Number of bleeds without trauma, during participation in sports activities


Secondary Outcome Measures :
  1. Change in HJHS scores [ Time Frame: 3 years ]
    Change in Hemophilia Joint Health Scores (HJHS). The HJHS provides a total score (higher score is worse; max=124), joint specific scores, and a global gait score.

  2. Change in bone mineral density [ Time Frame: 3 years ]
    Change in bone mineral density as measured by DEXA Scan

  3. Change in biomarkers of bone and joint health [ Time Frame: 3 years ]
    Change in biomarkers of bone and joint health as measured by (sRANKL (osteoclastogenesis), CTX-1 (Bone resorption), CS846 (cartilage repair) and IL-1 beta, IL6 and TNF-alpha (markers of inflammation).

  4. Number of FVIII doses for breakthrough bleeding episodes [ Time Frame: 3 years ]
    Number of doses of factor needed to treat breakthrough bleeding episodes

  5. MRI changes in relevant joints [ Time Frame: 3 years ]
    MRI changes in relevant joints (The joint to be imaged will be determined based on the sport activity; joints most involved in the activity will be imaged).


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.


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   Male
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.
Criteria

Inclusion Criteria:

  1. 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
  2. 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
  3. Participants must be on Emicizumab or standard FVIII prophylaxis per institutional/primary hematologist recommendations
  4. Participants must be engaging in sports activities with moderate to high risk of bleeding as defined by the NHF- Play it safe guidelines
  5. Participants must be compliant with completing all bleed and treatment logs
  6. Participant must be compliant with use of wearable activity tracker

Exclusion Criteria:

  1. Participant/parent/LAR unwilling to provide informed consent/assent
  2. Unwilling to log or document bleeds and treatment information as per study guidelines
  3. Participants with any other bleeding disorders will be excluded
  4. Participants on concomittent FVIII replacement and emicizumab for sports participation

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): NCT05022459


Contacts
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Contact: Amy Stolinski, MPH 313-745-0148 astolins@med.wayne.edu

Locations
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United States, California
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Center for Inherited Bleeding Disorders
Orange, California, United States, 92868
University of California, San Francisco (UCSF)
San Francisco, California, United States, 94158
United States, Georgia
Children's Healthcare of Atlanta/Emory University
Atlanta, Georgia, United States, 30322
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Rush University
Chicago, Illinois, United States, 60612
United States, Indiana
Indiana Hemostasis and Thrombosis Center (IHTC)
Indianapolis, Indiana, United States, 46260
United States, Iowa
University of Iowa
Iowa City, Iowa, United States, 52246
United States, New York
Northwell Health/Cohen Children's Medical Center
New Hyde Park, New York, United States, 11040
United States, North Carolina
University of North Carolina Hemophilia Treatment Center
Chapel Hill, North Carolina, United States, 27517
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Rainbow Hemostasis & Thrombosis Center, Rainbow Babies & Children's Hospital
Cleveland, Ohio, United States, 44106
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Sponsors and Collaborators
Wayne State University
Genentech, Inc.
Investigators
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Principal Investigator: Meera Chitlur, MD Wayne State University
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Responsible Party: Meera Chitlur, Director, Hemophilia Treatment Center and Hemostasis Program, Principal Investigator, Clinical Professor, Wayne State University
ClinicalTrials.gov Identifier: NCT05022459    
Other Study ID Numbers: STEP Study
First Posted: August 26, 2021    Key Record Dates
Last Update Posted: October 13, 2021
Last Verified: October 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
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