Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Genentech, Inc. | INDUSTRY |
Not provided
Not provided
Not provided
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.
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.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Emicizumab | Drug | Emicizumab in Hemophilia A |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| To study practitioner's preference for emicizumab schedule and how it affects compliance | ABR | 12 months |
Not provided
Inclusion Criteria Part A: ABR
Exclusion Criteria:
Part A: ABR
Part B: HRQoL and Arthropathy
Not provided
Not provided
Not provided
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.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Pari Sheerer | Contact | 973-803-7218 | pari.sheerer2@rwjbh.org |
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Newark Beth Israel Medical Center | Newark | New Jersey | 07112 | United States |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| D020141 | Hemostatic Disorders |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000608208 | emicizumab |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |