Clinical trial NCT04690322: POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies
Official title: POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Hemophilia A
Factor VIII
Lead sponsor: University of Texas Southwestern Medical Center
Sponsor class: OTHER
First posted: 2020-12-30
Enrollment: 28
Sex: ALL
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Each patient will be randomly assigned to receive either extended half-life factor VIII based replacement therapy or non-FVIII based replacement therapy
Primary purpose: TREATMENT

Brief 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.

Detailed description: This is a research study to find out if there is a difference in the way children with hemophilia A form a clot and also evaluate if they develop tiny bleeds within the joint and subsequently early joint changes when receiving extended half-life factor VIII based replacement therapy vs non-FVIII based replacement as part of their hemophilia treatment to prevent spontaneous joint bleeds.

Both therapies are standard of care therapies for patients with hemophilia A. The only experimental/research procedures as part of this study include the thrombin generation assay and ultrasound. All other procedures are standard of care.

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)

Primary outcomes:
measure: Changes in hemostatic potential
timeFrame: Baseline, 1 month, 6 months, and 12 months from start of study
measure: Changes in early joint-health markers
timeFrame: Baseline, 1 month, 6 months, and 12 months from the start of study
Secondary outcomes:
measure: Changes in bleeding rates
timeFrame: Baseline and 12 months after start of study
measure: Changes in health-related quality of life and physical activity
timeFrame: Baseline, 1 month, 6 months, and 12 months from the start of study
measure: Changes in joint health
timeFrame: Baseline, 1 month, 6 months, and 12 months from the start of study
Other outcomes:
Locations:
city: Dallas
country: United States
facility: The University of Texas Southwestern Medical Center
state: Texas
Study officials:
affiliation: University of Texas Southwestern Medical Center
name: Jessica Garcia, MD
role: PRINCIPAL_INVESTIGATOR
References: