POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies
Who is this study for? Pediatric patients up to age 17 years with hemophilia
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 4
SUMMARY
This is a prospective, randomized control trial in which each patient will be randomly assigned to receive either extended half-life factor VIII based replacement therapy or non-FVIII based replacement therapy, which are both standard of care treatment for persons with Hemophilia A.
Eligibility
Participation Requirements
Sex: All
Maximum Age: 17
Healthy Volunteers: f
View:
• Subjects with moderate hemophilia A (baseline factor VIII activity 1-5%) or severe hemophilia A (baseline factor VIII activity \<1%) on prophylactic standard half-life FVIII infusions OR subjects with moderate or severe hemophilia A who have not started prophylactic treatment
• Less than 18 years of age
Locations
United States
Texas
The University of Texas Southwestern Medical Center
RECRUITING
Dallas
Contact Information
Primary
Jessica Garcia, MD
Jessica.Garcia@utsouthwestern.edu
214-456-7000
Time Frame
Start Date: 2021-04-15
Estimated Completion Date: 2027-07
Participants
Target number of participants: 28
Treatments
Active_comparator: Extended half-life factor VIII-based replacement therapy
Subjects who are either already on prophylactic standard half-life FVIII products or have not started prophylactic treatment will be randomized to start prophylactic extended half-life FVIII products or non-factor product (emicizumab). Both therapies are considered the current standard of care.~The study has 4 planned visits at baseline, 1 month, 6 months, and 12 months. They will coincide with the standard of care visits.
Active_comparator: Non-Factor VIII-based replacement therapy
Subjects who are either already on prophylactic standard half-life FVIII products or have not started prophylactic treatment will be randomized to start prophylactic extended half-life FVIII products or non-factor product (emicizumab). Both therapies are considered the current standard of care.~The study has 4 planned visits at baseline, 1 month, 6 months, and 12 months. They will coincide with the standard of care visits.
Related Therapeutic Areas
Sponsors
Leads: University of Texas Southwestern Medical Center