Brief Title
An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors
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.
Study Type
Expanded Access
Condition
Hemophilia A
Intervention
Emicizumab
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
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
Gender
All
Ages
12 Years - N/A
Contacts
Clinical Trials, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT03154437
Organization ID
ML39356
Responsible Party
Sponsor
Study Sponsor
Genentech, Inc.
Study Sponsor
Clinical Trials, Study Director, Hoffmann-La Roche
Verification Date
March 2018