Optic IconOptic Logo
|
SearchCollectionsBenchmarks
Clinical trial

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

ID
Name
STU-2020-1378
Description
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.
Trial arms
Trial start
2021-04-15
Estimated PCD
2025-07-01
Trial end
2025-07-01
Status
Recruiting
Phase
Early phase I
Treatment
Eloctate
Eloctate is FDA-approved to treat patients with Hemophilia A. This drug will be used for extended half-life factor VIII-based replacement therapy.
Arms:
Extended half-life factor VIII-based replacement therapy
Other names:
ELOCTATE- antihemophilic factor (recombinant), fc fusion protein
Adynovate
Adynovate is FDA-approved to treat patients with Hemophilia A. This drug will be used for extended half-life factor VIII-based replacement therapy.
Arms:
Extended half-life factor VIII-based replacement therapy
Other names:
ADYNOVATE- antihemophilic factor (recombinant) pegylated
Emicizumab
Emicizumab is FDA-approved to treat patients with Hemophilia A. This drug will be used for non-factor VIII-based replacement therapy.
Arms:
Non-Factor VIII-based replacement therapy
Other names:
HEMLIBRA- emicizumab injection, solution
Size
28
Primary endpoint
Changes in hemostatic potential
Baseline, 1 month, 6 months, and 12 months from start of study
Changes in early joint-health markers
Baseline, 1 month, 6 months, and 12 months from the start of study
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)
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE4'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Each patient will be randomly assigned to receive either extended half-life factor VIII based replacement therapy or non-FVIII based replacement therapy', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 28, 'type': 'ESTIMATED'}}
Updated at
2024-02-16

1 organization

3 products

2 indications

Organization
University of Texas Southwestern Medical Center
Product
Eloctate
Indication
Hemophilia A
Indication
Coagulation factor VIII
Product
Adynovate
Product
Emicizumab