Health-Related Quality of Life Outcomes and Bleeding Rates Among Patients With Severe Hemophilia A on Emicizumab

NCT06104826 | Unknown DMC FDA Drug

• 1 other identifier: 2023.20
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

The main purposes of this study are to determine if use of emicizumab prophylaxis treatment reduces number of bleeding episodes, if it improves quality of life of individuals and if improves arthropathy in persons with hemophilia A.

Conditions

Hemophilia A

Keywords

Bleeding Disorder; Hemophilia; Quality of Life Outcomes; Emicizumab

Outcome Measures

Primary Outcomes (3)

• To obtain and compare the annual bleeding rate in patients with hemophilia A on emicizumab before and after being on emicizumab.

  Annualized Bleeding Rates (ABR)

  12 months

• To evaluate the health related quality of life in patients with hemophilia A before and after being on emicizumab.

  Quality of Life Questionaries

  12 months

• To study any clinical changes in arthropathy.

  Joint score measurements

  12 months

Secondary Outcomes (1)

• To study practitioner's preference for emicizumab schedule and how it affects compliance

  12 months

Interventions

Emicizumab DRUG

Emicizumab in Hemophilia A

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population will include children and adults who are males and females with hemophilia A for all parts of the study. Children and adults are equally at risk for bleeding and hence we would collect information about bleeding events in both children and adults on emicizumab or on factor concentrates to calculate ABR. There will be no exclusion based on race or ethnicity. For HRQoL part of the study, children and their families will be included.The Quality of life was found to be poor both among children affected with hemophilia and their parents and perceived impact on family. Hemophilic arthropathy can be seen in children and adults and therefore, this outcome must be studied in both populations as it can be disabling at any age.

You may qualify if:

• Signed Informed Consent/Assent Form
• Age 4 years at time of signing Informed Consent Form
• Ability to comply with the study protocol, in the investigator's judgment
• Diagnosis of hemophilia A (any severity) on emicizumab or factor concentrate prophylaxis for at least 12 months each
• Plan to be adherent to emicizumab prophylaxis during the study
• Patient with inhibitors
• Patient without inhibitors Part B: HRQoL and Arthropathy
• Signed Informed Consent/Assent Form
• Age 4 years at time of signing Informed Consent Form
• Ability to comply with the study protocol, in the investigator's judgment
• Diagnosis of hemophilia A (any severity) who has decided to switch to emicizumab prophylaxis
• Plan to be adherent to emicizumab prophylaxis during the study
• Individuals of childbearing age will be included in this study. Their participation involves voluntary completion of a questionnaire. There are no additional precautions required for this population.
• Patient/parent must be able to read and write English/Spanish

You may not qualify if:

• Part A: ABR
• Patients who are on a clinical trial for prophylaxis
• Patients with other bleeding disorders needing any scheduled treatment
• No eligibility restrictions will be based on gender, race, ethnic background or economic status
• Part B: HRQoL and Arthropathy
• Patients who are on a clinical trial for prophylaxis
• Patients with other bleeding disorders needing any scheduled treatment
• Patients/parents who refuse to complete questionnaire or have joint health assessment by HJHS
• No eligibility restrictions will be based on gender, race, ethnic background or economic status

Sponsors & Collaborators

• Newark Beth Israel Medical Center: lead
• Genentech, Inc.: collaborator

Study Sites (1)

Newark Beth Israel Medical Center

Newark, New Jersey, 07112, United States

Location

MeSH Terms

Conditions

Hemophilia A; Hemostatic Disorders

Interventions

emicizumab

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; Vascular Diseases; Cardiovascular Diseases

Central Study Contacts

Pari Sheerer

CONTACT

973-803-7218; pari.sheerer2@rwjbh.org

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Clinical Professor, Rutgers New Jersey and Robert Wood Johnson Medical School

Study Record Dates

• First Submitted: October 23, 2023
• First Posted: October 27, 2023
• Study Start: December 1, 2023
• Primary Completion: December 1, 2024
• Study Completion: December 1, 2025
• Last Updated: October 30, 2023

Record last verified: 2023-10

Locations

US (1)