NCT06104826

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

Health-related Quality of Life Outcomes and Bleeding Rates Among Patients With Severe Hemophilia A on Emicizumab: Real World Data

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 50 (estimated)
• Sponsor: Newark Beth Israel Medical Center · Academic / Other
• Sex: All
• Age: 4 Years
• Healthy volunteers: Accepted

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.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
DRUG	Emicizumab	Emicizumab in Hemophilia A

Timeline

• Start date: 2023-12-01
• Primary completion: 2024-12-01
• Completion: 2025-12-01
• First posted: 2023-10-27
• Last updated: 2023-10-30

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study