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

Not yet recruiting

• Conditions: Hemophilia A

• Registration Number: NCT06104826
• Lead Sponsor: Newark Beth Israel Medical Center

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.

Detailed Description

The main purpose of this study is to determine if use of emicizumab prophylaxis treatment reduces number of bleeding episodes and if it affects quality of life of individuals with hemophilia.

Patients with hemophilia A, especially those with severe disease, have recurrent bleeding in joints, muscles and deep tissue and recurrent bleeding in the same joint can result in irreversible damage in bone and cartilage and lead to disabling arthropathy. Treatment involves replacement with exogenous recombinant factor concentrates that can prevent further bleeding episodes but not the ongoing joint damage. In addition, treatment with recombinant factor concentrates can lead to inhibitory antibodies resulting in inability to use those for further treatment. The burden of this disease on patients and caregivers is well recognized.

Participant will be asked about bleeding episodes needing additional factor replacement in addition to checking bleeding log prior to starting emicizumab and at additional times after starting emicizumab. In addition, investigators would ask participant to complete questionnaire about participant's and/or their caregiver's quality of life prior to starting emicizumab and at additional times after starting emicizumab. Participant will also have joint scores measured before and at additional times after starting emicizumab. All the information collected will remain confidential and would only be shared with members of study team.

Study Timeline

• Status Verified: 2023-10
• Study First Submitted: 2023-10-23
• Study First Submitted QC: 2023-10-23
• Last Update Submitted: 2023-10-26
• Study First Posted: 2023-10-27
• Last Update Posted: 2023-10-30
• Study Start: 2023-12
• Primary Completion: 2024-12
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

Not provided

Exclusion Criteria

• 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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
To obtain and compare the annual bleeding rate in patients with hemophilia A on emicizumab before and after being on emicizumab.	12 months	Annualized Bleeding Rates (ABR)
To evaluate the health related quality of life in patients with hemophilia A before and after being on emicizumab.	12 months	Quality of Life Questionaries
To study any clinical changes in arthropathy.	12 months	Joint score measurements

Secondary Outcome Measures

Name	Time	Method
To study practitioner's preference for emicizumab schedule and how it affects compliance	12 months	ABR

Trial Locations

Locations (1)

Newark Beth Israel Medical Center; 🇺🇸 Newark, New Jersey, United States