Not provided
Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| ML43506 | Other Identifier | Sponsor |
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 |
| Regional Comprehensive Hemophilia Treatment Center (Mount Sinai) | UNKNOWN |
| Massachusetts General Comprehensive Hemophilia and Thrombosis Treatment Center | UNKNOWN |
Not provided
Not provided
Not provided
Hemophilia A (HA) is a rare congenital bleeding disorder characterized by coagulation factor VIII deficiency. In severe HA, defined as plasma FVIII clotting activity < 1%, bleeding may frequently occur spontaneously, most commonly in joints, leading to painful hemophilic arthropathy and loss of joint function. Patients with moderate or mild hemophilia A, defined as FVIII clotting activity between 1-<5% and 5-40% respectively, are less likely to have spontaneous bleeding however can have significant bleeding with trauma or surgery. Perioperative management by a hematologist who specializes in hemophilia is needed to ensure hemostasis during surgery. Hemophilia is an X-linked recessive disorder affecting 1 in 5000 to 10,000 males.
A questionnaire to elucidate the dosing, frequency and indication for the use of emicizumab in patients with Hemophilia A (mild, moderate or severe) ages 0-3 years has been developed. Data on any pre-, peri and post-surgical practices while on emicizumab is being collected. Pediatricians are being asked if there are plans to introduce factor 8 to children who are already on emicizumab for primary prophylaxis as well as how and when this is planned on being done. The hope is that this data will help inform understanding of current use of emicizumab in infants and young children as a form of primary prophylaxis, especially when venous access has historically been a limiting factor.
This will be a multi-institutional, retrospective review of pediatric patients ages 0 to 36 months of age who are currently receiving and/or have received emicizumab-kxwh as part of their treatment for hemophilia A with or without inhibitors. Participating sites are part of the New England Region (plus New Jersey and New York-Region II) of hemophilia treatment centers. Each institution will be contributing subjects who have been treated or who are currently being treated with emicizumab, from October 4th, 2018 up to the point of IRB approval. Additionally, each institution will determine the best way to identify eligible patients and keep track of patients enrolled in the study. The Children's Hospital at Montefiore will be the coordinating center.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| HEMLIBRA | Drug | Emicizumab (Hemlibra, also known as ACE910 and RO5534262) is a humanized monoclonal modified immunoglobulin G4 (IgG4) antibody with a bispecific antibody structure produced by recombinant DNA technology in Chinese hamster ovary (CHO) cells. |
|
| Measure | Description | Time Frame |
|---|---|---|
| The proportion of patients that are being treated with emicizumab | The primary outcome will include the proportion of patients that are being treated with emicizumab for primary prophylaxis, as well as identifying breakthrough bleeding while on emicizumab. | Within a year. |
| Measure | Description | Time Frame |
|---|---|---|
| Patients who receive additional doses of factor concentrate while on emicizumab | Data collected will also include proportion of patients who receive additional doses of factor concentrate while on emicizumab for trauma or surgical interventions. | Within a year. |
| Measure | Description | Time Frame |
|---|---|---|
| Patients by demographics (age and race), clinical (severity of hemophilia, inhibitor history) and treatment data | Additionally, we will characterize the 37+ patients by demographics (age and race), clinical (severity of hemophilia, inhibitor history) and treatment data (indication for emicizumab ie. primary vs secondary prophylaxis. | Within a year. |
Inclusion Criteria: Patients must meet the following criteria for study entry:
Exclusion Criteria:
Not provided
Not provided
Not provided
Patients 0 to 36 months of age, diagnosed with severe, moderate or mild Hemophlia A at the time of initiating treatment with Emicizumab.
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Jennifer Davila, MD | Children's Hospital at Montefiore | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Connecticut Children's Hemophilia Treatment Center - Connecticut Children's Medical Center | Hartford | Connecticut | 06106 | United States | ||
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| 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
| Rutgers Robert Wood Johnson Medical School | OTHER |
Not provided
Not provided
Not provided
| Yale Hemophilia Treatment Center |
| New Haven |
| Connecticut |
| 06510 |
| United States |
| Dartmouth Hitchcock Hemophilia Center - Mary Hitchcock Memorial Hospital | Lebanon | New Hampshire | 03766 | United States |
| Newark Beth Israel | Newark | New Jersey | 07112 | United States |
| Western NY Blood Care - Research Foundation for SUNY | Buffalo | New York | 14202 | United States |
| Comprehensive Center for Hemophilia and Coagulation Disorders (Cornell) | New York | New York | 10065 | United States |
| Mary M Gooley Hemophilia Center, Inc. | Rochester | New York | 14621 | United States |
| Northwell Health Hemostasis and Thrombosis Center | Staten Island | New York | 11030 | United States |
| SUNY Upstate Hemophilia Treatment Center - HTC 058 | Syracuse | New York | 13210 | United States |
| Children's Hospital at Montefiore | The Bronx | New York | 10467 | United States |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |