An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors
An Open-Label, Multicenter, Expanded Access Program for Emicizumab in Patients With Hemophilia A With Inhibitors
1 other identifier
expanded_access
N/A
1 country
14
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.
Trial Health
Trial Health Score
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14 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 12, 2017
CompletedFirst Posted
Study publicly available on registry
May 16, 2017
CompletedMarch 29, 2018
March 1, 2018
May 12, 2017
March 26, 2018
Conditions
Interventions
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.
Eligibility Criteria
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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Genentech, Inc.lead
Study Sites (14)
University of Colorado Denver, Children's Hospital
Aurora, Colorado, 80045, United States
University of Miami Miller School of Medicine
Miami, Florida, 33136, United States
Rush Medical Center
Chicago, Illinois, 60612, United States
Tulane Medical Center; Investigational/Research Pharmacy
New Orleans, Louisiana, 70112, United States
Boston Childrens Hospital
Boston, Massachusetts, 02115, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Children's Mercy Hosp Clinics
Kansas City, Missouri, 64108, United States
Barnabas Health Newark Beth Israel Medical Center - Pulmonary Hypertension & Lung Transplant Program
Newark, New Jersey, 07112, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104, United States
University of Texas Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, 75235, United States
University of Texas Medical School
Houston, Texas, 77030, United States
University of Utah
Salt Lake City, Utah, 84132, United States
Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia
Seattle, Washington, 98104, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Clinical Trials
Hoffmann-La Roche
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 12, 2017
First Posted
May 16, 2017
Last Updated
March 29, 2018
Record last verified: 2018-03