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Genentech, Inc.

An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors

Official Title: 
An Open-Label, Multicenter, Expanded Access Program for Emicizumab in Patients With Hemophilia A With Inhibitors
Brief Summary: 
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.
Approved for marketing
EA Type
Treatment IND/Protocol
Hemophilia A

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
Other Name: CH5534262

Eligibility Criteria: 

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

Eligibility Gender: 
All
Eligibility Age: 
Minimum: 12 Years
Countries: 
United States
Locations: 

University of Colorado Denver, Children's Hospital
Aurora, Colorado, 80045

University of Miami Miller School of Medicine
Miami, Florida, 33136

Rush Medical Center
Chicago, Illinois, 60612

Tulane Medical Center; Investigational/Research Pharmacy
New Orleans, Louisiana, 70112

Boston Childrens Hospital
Boston, Massachusetts, 02115

University of Minnesota
Minneapolis, Minnesota, 55455

Children's Mercy Hosp Clinics
Kansas City, Missouri, 64108

Barnabas Health Newark Beth Israel Medical Center - Pulmonary Hypertension & Lung Transplant Program
Newark, New Jersey, 07112

Nationwide Children's Hospital
Columbus, Ohio, 43205

University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104

University of Texas Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, 75235

University of Texas Medical School
Houston, Texas, 77030

University of Utah
Salt Lake City, Utah, 84132

Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia
Seattle, Washington, 98104

Clinical Trials
Study Director
Hoffmann-La Roche

Genentech, Inc.
EA Number: 
MeSH Terms: 
Hemophilia A, Factor VIII, Antibodies, Bispecific
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