An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors

Hemophilia A Clinical Trial

Official title:

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

Clinical Trial Details — Status: Approved for marketing

Administrative data

• NCT number: NCT03154437
• Other study ID #: ML39356
• Status: Approved for marketing
• First received: May 12, 2017
• Last updated: March 26, 2018

Study information

• Verified date: March 2018
• Source: Genentech, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial 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.

Recruitment information / eligibility

• Status: Approved for marketing
• Enrollment: 0
• Gender: All
• Age group: 12 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Biological:
• Emicizumab
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.

Locations

Country	Name	City	State
United States	University of Colorado Denver, Children's Hospital	Aurora	Colorado
United States	Boston Childrens Hospital	Boston	Massachusetts
United States	Rush Medical Center	Chicago	Illinois
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	University of Texas Southwestern Medical Center - Children's Medical Center Dallas	Dallas	Texas
United States	University of Texas Medical School	Houston	Texas
United States	Children's Mercy Hosp Clinics	Kansas City	Missouri
United States	University of Miami Miller School of Medicine	Miami	Florida
United States	University of Minnesota	Minneapolis	Minnesota
United States	Tulane Medical Center; Investigational/Research Pharmacy	New Orleans	Louisiana
United States	Barnabas Health Newark Beth Israel Medical Center - Pulmonary Hypertension & Lung Transplant Program	Newark	New Jersey
United States	University of Oklahoma Health Sciences Center	Oklahoma City	Oklahoma
United States	University of Utah	Salt Lake City	Utah
United States	Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Genentech, Inc.

Country where clinical trial is conducted

United States,