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Phase 4
Age: 0 - 17Years
All Genders
ID04690322

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

Led by University of Texas Southwestern Medical Center · Updated on 2026-04-07

N/A

Participants Needed

1

Research Sites

N/A

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 0 - 17Years
All Genders

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)

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