Clinical trial NCT03154437: 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
Study type: EXPANDED_ACCESS
Overall status: APPROVED_FOR_MARKETING
Conditions: Hemophilia A
Lead sponsor: Genentech, Inc.
Sponsor class: INDUSTRY
First posted: 2017-05-16
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT

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.


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

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Aurora
country: United States
facility: University of Colorado Denver, Children's Hospital
state: Colorado
city: Miami
country: United States
facility: University of Miami Miller School of Medicine
state: Florida
city: Chicago
country: United States
facility: Rush Medical Center
state: Illinois
city: New Orleans
country: United States
facility: Tulane Medical Center; Investigational/Research Pharmacy
state: Louisiana
city: Boston
country: United States
facility: Boston Childrens Hospital
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Kansas City
country: United States
facility: Children's Mercy Hosp Clinics
state: Missouri
city: Newark
country: United States
facility: Barnabas Health Newark Beth Israel Medical Center - Pulmonary Hypertension & Lung Transplant Program
state: New Jersey
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Oklahoma City
country: United States
facility: University of Oklahoma Health Sciences Center
state: Oklahoma
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center - Children's Medical Center Dallas
state: Texas
city: Houston
country: United States
facility: University of Texas Medical School
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Seattle
country: United States
facility: Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia
state: Washington
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References: