POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies

Launched by UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER · Dec 27, 2020

Keywords

ClinConnect Summary

This clinical trial, called POCUS, is studying the effects of two different types of treatments for patients with severe hemophilia A. Hemophilia A is a condition where people have low levels of a protein called Factor VIII, which is important for blood clotting. In this trial, participants will be randomly assigned to receive either an extended half-life Factor VIII treatment or a non-FVIII treatment, both of which are standard options for managing hemophilia A. The goal is to understand how these treatments affect bleeding control and joint health.

To be eligible for this trial, participants must be under 18 years old and have moderate to severe hemophilia A. This means they should have a very low level of Factor VIII activity. However, some individuals may not qualify if they have certain health conditions, such as having a specific type of blood protein that can complicate treatment or a history of joint surgeries. If you decide to join the study, you can expect regular check-ups and monitoring to ensure your safety and assess how well the treatments are working. This trial is currently looking for participants, so it could be a great opportunity to contribute to important research in hemophilia A.

Gender

ALL

Eligibility 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)