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

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ClinicalTrials.gov Identifier: NCT04690322
Recruitment Status : Recruiting
First Posted : December 30, 2020
Last Update Posted : April 23, 2021
Sponsor:
Information provided by (Responsible Party):
Jessica Garcia, University of Texas Southwestern Medical Center

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.

Condition or disease Intervention/treatment Phase
Hemophilia A Factor VIII Drug: Eloctate Drug: Adynovate Drug: Emicizumab Phase 4

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 28 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
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
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies
Actual Study Start Date : April 15, 2021
Estimated Primary Completion Date : January 2023
Estimated Study Completion Date : January 2023


Arm Intervention/treatment
Active Comparator: Extended half-life factor VIII-based replacement therapy

Subjects who are either already on prophylactic standard half-life FVIII products or have not started prophylactic treatment will be randomized to start prophylactic extended half-life FVIII products or non-factor product (emicizumab). Both therapies are considered the current standard of care.

The study has 4 planned visits at baseline, 1 month, 6 months, and 12 months. They will coincide with the standard of care visits.

Drug: 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.
Other Name: ELOCTATE- antihemophilic factor (recombinant), fc fusion protein

Drug: 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.
Other Name: ADYNOVATE- antihemophilic factor (recombinant) pegylated

Active Comparator: Non-Factor VIII-based replacement therapy

Subjects who are either already on prophylactic standard half-life FVIII products or have not started prophylactic treatment will be randomized to start prophylactic extended half-life FVIII products or non-factor product (emicizumab). Both therapies are considered the current standard of care.

The study has 4 planned visits at baseline, 1 month, 6 months, and 12 months. They will coincide with the standard of care visits.

Drug: Emicizumab
Emicizumab is FDA-approved to treat patients with Hemophilia A. This drug will be used for non-factor VIII-based replacement therapy.
Other Name: HEMLIBRA- emicizumab injection, solution




Primary Outcome Measures :
  1. Changes in hemostatic potential [ Time Frame: Baseline, 1 month, 6 months, and 12 months from start of study ]
    Evaluate differences in hemostatic potential in persons with hemophilia A without evidence of hemophilic target joints who are receiving prophylactic FVIII-based replacement therapy versus non-FVIII replacement therapy by measuring thrombin generation potential via a plasma-based assay

  2. Changes in early joint-health markers [ Time Frame: Baseline, 1 month, 6 months, and 12 months from the start of study ]
    Identify early joint-health markers associated with bleeding in persons with hemophilia A without evidence of hemophilic target joints who are receiving prophylactic FVIII-based replacement therapy versus non-FVIII replacement by measuring soft tissue and osteochondral changes among 6 joints (bilateral elbows, knees, and ankles) using point of care ultrasound between both treatment groups


Secondary Outcome Measures :
  1. Changes in bleeding rates [ Time Frame: Baseline and 12 months after start of study ]
    Measure bleeding rates in persons with hemophilia A without evidence of hemophilic target joints on prophylactic FVIII-based replacement therapy versus non-FVIII replacement therapy by measuring the difference in annualized bleeding rates between the two treatment groups, as well as other surrogate markers of bleeding, or indirect causes of bleeding, which include FVIII inhibitor level, hemoglobin, and serum ferritin

  2. Changes in health-related quality of life and physical activity [ Time Frame: Baseline, 1 month, 6 months, and 12 months from the start of study ]
    Evaluate health-related quality of life and physical activity in persons with hemophilia A without evidence of hemophilic target joints on prophylactic FVIII-based replacement therapy versus non-FVIII replacement therapy using validated questionnaires

  3. Changes in joint health [ Time Frame: Baseline, 1 month, 6 months, and 12 months from the start of study ]
    Assess joint health in persons with hemophilia A without evidence of hemophilic target joints on prophylactic FVIII-based replacement therapy versus non-FVIII replacement therapy using validated outcome tool (Hemophilia Joint Health Score)



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Ages Eligible for Study:   up to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04690322


Contacts
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Contact: Jessica Garcia, MD 214-456-7000 Jessica.Garcia@utsouthwestern.edu

Locations
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United States, Texas
The University of Texas Southwestern Medical Center Recruiting
Dallas, Texas, United States, 75235
Contact: Jessica Garcia, MD    214-456-7000    Jessica.Garcia@UTSouthwestern.edu   
Principal Investigator: Jessica Garcia, MD         
Sub-Investigator: Ayesha Zia, MD         
Sub-Investigator: Ravi Sarode, MD         
Sponsors and Collaborators
Jessica Garcia
Investigators
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Principal Investigator: Jessica Garcia, MD University of Texas Southwestern Medical Center
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Responsible Party: Jessica Garcia, Assistant Professor, University of Texas Southwestern Medical Center
ClinicalTrials.gov Identifier: NCT04690322    
Other Study ID Numbers: STU-2020-1378
First Posted: December 30, 2020    Key Record Dates
Last Update Posted: April 23, 2021
Last Verified: April 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
BAX 855
Antibodies, Bispecific
Coagulants
Immunologic Factors
Physiological Effects of Drugs