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

NCT04690322 | Recruiting Phase 4 FDA Drug

• 1 other identifier: STU-2020-1378
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 1

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.

Conditions

Hemophilia A; Factor VIII

Outcome Measures

Primary Outcomes (2)

• Changes in hemostatic potential

  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

  Baseline, 1 month, 6 months, and 12 months from start of study

• Changes in early joint-health markers

  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

  Baseline, 1 month, 6 months, and 12 months from the start of study

Secondary Outcomes (3)

• Changes in bleeding rates

  Baseline and 12 months after start of study

• Changes in health-related quality of life and physical activity

  Baseline, 1 month, 6 months, and 12 months from the start of study

• Changes in joint health

  Baseline, 1 month, 6 months, and 12 months from the start of study

Study Arms (2)

Extended half-life factor VIII-based replacement therapy

ACTIVE COMPARATOR

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; Drug: Adynovate

Non-Factor VIII-based replacement therapy

ACTIVE COMPARATOR

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

Interventions

Eloctate DRUG

Eloctate is FDA-approved to treat patients with Hemophilia A. This drug will be used for extended half-life factor VIII-based replacement therapy.

Also known as: ELOCTATE- antihemophilic factor (recombinant), fc fusion protein

Extended half-life factor VIII-based replacement therapy

Adynovate DRUG

Adynovate is FDA-approved to treat patients with Hemophilia A. This drug will be used for extended half-life factor VIII-based replacement therapy.

Also known as: ADYNOVATE- antihemophilic factor (recombinant) pegylated

Extended half-life factor VIII-based replacement therapy

Emicizumab DRUG

Emicizumab is FDA-approved to treat patients with Hemophilia A. This drug will be used for non-factor VIII-based replacement therapy.

Also known as: HEMLIBRA- emicizumab injection, solution

Non-Factor VIII-based replacement therapy

Eligibility Criteria

• Age: Up to 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• University of Texas Southwestern Medical Center: lead

Study Sites (1)

The University of Texas Southwestern Medical Center

Dallas, Texas, 75235, United States

RECRUITING

MeSH Terms

Conditions

Hemophilia A

Interventions

factor VIII-Fc fusion protein; BAX 855; emicizumab; Solutions

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pharmaceutical Preparations

Study Officials

• Jessica Garcia, MD

  University of Texas Southwestern Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jessica Garcia, MD

CONTACT

214-456-7000; Jessica.Garcia@utsouthwestern.edu

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Model Details: Each patient will be randomly assigned to receive either extended half-life factor VIII based replacement therapy or non-FVIII based replacement therapy

Study Record Dates

• First Submitted: December 27, 2020
• First Posted: December 30, 2020
• Study Start: April 15, 2021
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: March 3, 2025

Record last verified: 2025-02

Locations

US (1)