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

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Conditions studied: Hemophilia A

In brief

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.

Key facts

Study ID

NCT03154437

Run by

Genentech, Inc.

Last updated by the study team

2018-03-29

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• 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

Full record on ClinicalTrials.gov

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