An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors
- Conditions
- Hemophilia A
- Registration Number
- NCT03154437
- Lead Sponsor
- Genentech, Inc.
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- APPROVED_FOR_MARKETING
- Sex
- All
- Target Recruitment
- Not specified
- 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
- 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
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (14)
Tulane Medical Center; Investigational/Research Pharmacy
🇺🇸New Orleans, Louisiana, United States
Children's Mercy Hosp Clinics
🇺🇸Kansas City, Missouri, United States
University of Texas Medical School
🇺🇸Houston, Texas, United States
University of Miami Miller School of Medicine
🇺🇸Miami, Florida, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Rush Medical Center
🇺🇸Chicago, Illinois, United States
Boston Childrens Hospital
🇺🇸Boston, Massachusetts, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Barnabas Health Newark Beth Israel Medical Center - Pulmonary Hypertension & Lung Transplant Program
🇺🇸Newark, New Jersey, United States
University of Texas Southwestern Medical Center - Children's Medical Center Dallas
🇺🇸Dallas, Texas, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia
🇺🇸Seattle, Washington, United States
University of Colorado Denver, Children's Hospital
🇺🇸Aurora, Colorado, United States
University of Oklahoma Health Sciences Center
🇺🇸Oklahoma City, Oklahoma, United States