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



Status:Approved for marketing
Conditions:Anemia, Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:12 - Any
Updated:4/17/2018

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An Open-Label, Multicenter, Expanded Access Program for Emicizumab in Patients With Hemophilia A With Inhibitors

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.


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
We found this trial at
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1415 Tulane Ave
New Orleans, Louisiana 70112
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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1601 Northwest 12th Avenue
Miami, Florida 33136
(305) 243-6545
University of Miami Miller School of Medicine The University of Miami Leonard M. Miller School...
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Minneapolis, Minnesota 55455
(612) 625-5000
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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201 Presidents Circle
Salt Lake City, Utah 84108
801) 581-7200
University of Utah Research is a major component in the life of the U benefiting...
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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Chicago, Illinois 60612
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Houston, Texas 77030
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Kansas City, Missouri 64108
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Newark, NJ
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940 NE 13th St
Oklahoma City, Oklahoma 73190
(405) 271-6458
University of Oklahoma Health Sciences Center The OU Health Sciences Center is composed of seven...
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