Evaluating clot formation and joint health in children with severe hemophilia A on different therapies
POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies
This study is testing whether two different treatments for severe hemophilia A can help children have better clotting and joint health.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 28 (estimated) |
| Ages | N/A to 17 Years |
| Sex | All |
| Sponsor | University of Texas Southwestern Medical Center Academic / other |
| Locations | 1 site (Dallas, Texas) |
| Trial ID | NCT04690322 on ClinicalTrials.gov |
What this trial studies
This research investigates the differences in clot formation and joint health in children with severe hemophilia A receiving either extended half-life factor VIII replacement therapy or non-FVIII based replacement therapy. The study is a prospective, randomized control trial where participants will be assigned to one of the two treatment groups. It aims to assess the occurrence of joint bleeds and early joint changes through standard care procedures, including thrombin generation assays and ultrasound evaluations. Both therapies are recognized as standard treatments for hemophilia A.
Who should consider this trial
Good fit: Ideal candidates for this study are children under 18 years old with moderate to severe hemophilia A who are on prophylactic treatment or have not yet started treatment.
Not a fit: Patients with documented FVIII inhibitors or significant joint history, such as multiple target joints or previous synovectomy, may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved treatment strategies for children with hemophilia A, enhancing their joint health and reducing bleeding episodes.
How similar studies have performed: Other studies have explored similar therapeutic approaches in hemophilia A, but this specific comparison of therapies is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Exclusion Criteria: * Subjects with documented FVIII inhibitor * Subjects with a history of ≥ 2 target joints * Subjects with a history of synovectomy * Currently using medications known to impact bone and mineral metabolism (e.g., bisphosphonates, corticosteroids, estrogen, testosterone, calcitonin, thyroid hormone therapy); * Disease states known to affect bone integrity (e.g., primary hyperparathyroidism, Paget's disease, clinically significant liver disease)
Where this trial is running
Dallas, Texas
- The University of Texas Southwestern Medical Center — Dallas, Texas, United States (Recruiting)
Study contacts
- Principal investigator: Jessica Garcia, MD — University of Texas Southwestern Medical Center
- Study coordinator: Jessica Garcia, MD
- Email: Jessica.Garcia@utsouthwestern.edu
- Phone: 214-456-7000
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.