This open-label, multicenter expanded access program (EAP) is designed to provideemicizumab to eligible participants with hemophilia A with factor VIII (FVIII) inhibitorsbefore it is commercially available in the United States for the indication of hemophiliaA with FVIII inhibitors. Discontinuation may occur earlier if participant or physiciandecides to discontinue treatment or the sponsor discontinues emicizumab clinicaldevelopment.
Biological: Emicizumab
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.
Other Name: ACE910,CH5534262
Inclusion Criteria:
- Diagnosis of congenital hemophilia A of any severity and documented history of
high-titer inhibitor (that is [i.e.], greater than or equal to [>/=] 5 Bethesda
Units)
- History of treatment with episodic or prophylactic bypassing agents for at least the
last 24 weeks
- >/=6 (if on an episodic bypassing agent regimen) or >/=2 (if on a prophylactic
bypassing agent regimen) bleeds within 24 weeks prior to screening
- Currently using recombinant activated factor VII (rFVIIa) or are willing to switch
to rFVIIa as primary bypassing agent for the treatment of breakthrough bleeds
- Adequate hematologic function, defined as platelet count >/= 100,000 per microliters
(mcL) and hemoglobin >/=8 grams per deciliter (g/dL) at screening
- Adequate hepatic and renal function
Exclusion Criteria:
- Inherited or acquired bleeding disorder other than hemophilia A
- Ongoing (or plan to receive during the study) immune tolerance induction (ITI)
therapy or prophylaxis with FVIII with the exception of participants who have
received a treatment regimen of FVIII prophylaxis with concurrent bypassing agent
prophylaxis
- Treatment for thromboembolic disease within 12 months before Day 1 (with the
exception of previous catheter-associated thrombosis for which antithrombotic
treatment is not currently ongoing) or current signs of thromboembolic disease
- Other conditions (example [e.g.], certain autoimmune diseases) that may increase the
risk of bleeding or thrombosis
- High risk for thrombotic microangiopathy (TMA), in the investigator's judgment
- History of clinically significant hypersensitivity associated with monoclonal
antibody therapies or components of the emicizumab injection
- Use of systemic immunomodulators (e.g., interferon or rituximab) at enrollment or
planned use during the study, with the exception of antiretroviral therapy
- Treatment with any of the following: An investigational drug to treat or reduce the
risk of hemophilic bleeds within 5 half-lives of last drug administration before Day
1; A non-hemophilia-related investigational drug within the last 30 days or 5
half-lives before Day 1, whichever is longer; An investigational drug concurrently
- Any serious medical condition, treatment, or abnormality in clinical laboratory
tests that, in the investigator's judgment, precludes the participant's safe
participation in the study
University of Colorado Denver, Children's Hospital
Aurora, Colorado, United States
University of Miami Miller School of Medicine
Miami, Florida, United States
Rush Medical Center
Chicago, Illinois, United States
Tulane Medical Center; Investigational/Research Pharmacy
New Orleans, Louisiana, United States
Boston Childrens Hospital
Boston, Massachusetts, United States
University of Minnesota
Minneapolis, Minnesota, United States
Children's Mercy Hosp Clinics
Kansas City, Missouri, United States
Barnabas Health Newark Beth Israel Medical Center - Pulmonary Hypertension & Lung Transplant Program
Newark, New Jersey, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, United States
University of Texas Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, United States
University of Texas Medical School
Houston, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia
Seattle, Washington, United States
Clinical Trials, Study Director
Hoffmann-La Roche