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
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: Array

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 ~ Maximum: N/A
Countries
United States
Locations

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

Genentech, Inc.
NCT Number
MeSH Terms
Hemophilia A