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

Genentech, Inc.14 sites in 1 countryMay 16, 2017

ConditionsHemophilia A

Overview

Phase: Not Applicable
Intervention: Not specified
Conditions: Hemophilia A
Sponsor: Genentech, Inc.
Locations: 14
Status: Approved For Marketing
Last Updated: 8 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

May 16, 2017

End Date

TBD

Last Updated

8 years ago

Study Type

Expanded Access

Sex

All

Investigators

Sponsor

Genentech, Inc.

Responsible Party

Sponsor

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