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This open-label, multicenter expanded access program (EAP) is designed to provide emicizumab to eligible participants with hemophilia A with factor VIII (FVIII) inhibitors before it is commercially available in the United States for the indication of hemophilia A with FVIII inhibitors. Discontinuation may occur earlier if participant or physician decides to discontinue treatment or the sponsor discontinues emicizumab clinical development.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Emicizumab | Biological | Participants will receive emicizumab at a loading dose of 3 milligrams per kilogram (mg/kg) per week subcutaneously (SC) for 4 weeks, followed by a maintenance dose of 1.5 mg/kg per week SC thereafter. Treatment with emicizumab will continue until unacceptable toxicity, withdrawal of consent, participant or physician decision to discontinue treatment, death, the participant is able to obtain commercial drug after emicizumab becomes commercially available, or the sponsor decides to discontinue emicizumab clinical development, whichever occurs first. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Trials | Hoffmann-La Roche | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Colorado Denver, Children's Hospital | Aurora | Colorado | 80045 | United States | ||
| University of Miami Miller School of Medicine |
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| Miami |
| Florida |
| 33136 |
| United States |
| Rush Medical Center | Chicago | Illinois | 60612 | United States |
| Tulane Medical Center; Investigational/Research Pharmacy | New Orleans | Louisiana | 70112 | United States |
| Boston Childrens Hospital | Boston | Massachusetts | 02115 | United States |
| University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
| Children's Mercy Hosp Clinics | Kansas City | Missouri | 64108 | United States |
| Barnabas Health Newark Beth Israel Medical Center - Pulmonary Hypertension & Lung Transplant Program | Newark | New Jersey | 07112 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| University of Oklahoma Health Sciences Center | Oklahoma City | Oklahoma | 73104 | United States |
| University of Texas Southwestern Medical Center - Children's Medical Center Dallas | Dallas | Texas | 75235 | United States |
| University of Texas Medical School | Houston | Texas | 77030 | United States |
| University of Utah | Salt Lake City | Utah | 84132 | United States |
| Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia | Seattle | Washington | 98104 | United States |
| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C000608208 | emicizumab |
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