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
14
sites
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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1415 Tulane Ave
New Orleans, Louisiana 70112
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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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Seattle, WA
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