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

Description

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 Start Date

Not specified

Estimated Completion Date

Not specified

Interventions

  • Biological: Emicizumab

Study ID

Genentech, Inc. -- ML39356

Status

Available

Trial ID

NCT03154437

Study Type

Expanded Access

Trial Phase

N/A

Enrollment Quota

Not Listed

Sponsor

Genentech, Inc.

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 and older

Accepts Healthy Volunteers

Not Listed

Study Locations and Contact Information (49)

Study Location Distance Name Phone Email
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles None None None
Barnabas Health Newark Beth Israel Medical Center Pulmonary Hypertension Lung Transplant Program - Newark, New Jersey 197.7 miles None None None
Barnabas Health Newark Beth Israel Medical Center Pulmonary Hypertension Lung Transplant Program - Newark, New Jersey 197.7 miles None None None
Barnabas Health Newark Beth Israel Medical Center Pulmonary Hypertension Lung Transplant Program - Newark, New Jersey 197.7 miles None None None
Barnabas Health Newark Beth Israel Medical Center Pulmonary Hypertension Lung Transplant Program - Newark, New Jersey 197.7 miles None None None
Barnabas Health Newark Beth Israel Medical Center Pulmonary Hypertension Lung Transplant Program - Newark, New Jersey 197.7 miles None None None
Nationwide Childrens Hospital - Columbus, Ohio 640.0 miles None None None
Nationwide Childrens Hospital - Columbus, Ohio 640.0 miles None None None
Nationwide Childrens Hospital - Columbus, Ohio 640.0 miles None None None
Nationwide Childrens Hospital - Columbus, Ohio 640.0 miles None None None
Nationwide Childrens Hospital - Columbus, Ohio 640.0 miles None None None
Rush Medical Center - Chicago, Illinois 851.3 miles None None None
University of Minnesota - Minneapolis, Minnesota 1,120.0 miles None None None
University of Minnesota - Minneapolis, Minnesota 1,120.0 miles None None None
University of Minnesota - Minneapolis, Minnesota 1,120.0 miles None None None
Childrens Mercy Hosp Clinics - Kansas City, Missouri 1,249.3 miles None None None
Childrens Mercy Hosp Clinics - Kansas City, Missouri 1,249.3 miles None None None
Childrens Mercy Hosp Clinics - Kansas City, Missouri 1,249.3 miles None None None
Childrens Mercy Hosp Clinics - Kansas City, Missouri 1,249.3 miles None None None
Childrens Mercy Hosp Clinics - Kansas City, Missouri 1,249.3 miles None None None
University of Miami Miller School of Medicine - Miami, Florida 1,259.8 miles None None None
Tulane Medical Center InvestigationalResearch Pharmacy - New Orleans, Louisiana 1,360.0 miles None None None
Tulane Medical Center InvestigationalResearch Pharmacy - New Orleans, Louisiana 1,360.0 miles None None None
Tulane Medical Center InvestigationalResearch Pharmacy - New Orleans, Louisiana 1,360.0 miles None None None
Tulane Medical Center InvestigationalResearch Pharmacy - New Orleans, Louisiana 1,360.0 miles None None None
Tulane Medical Center InvestigationalResearch Pharmacy - New Orleans, Louisiana 1,360.0 miles None None None
University of Oklahoma Health Sciences Center - Oklahoma City, Oklahoma 1,493.6 miles None None None
University of Oklahoma Health Sciences Center - Oklahoma City, Oklahoma 1,493.6 miles None None None
University of Oklahoma Health Sciences Center - Oklahoma City, Oklahoma 1,493.6 miles None None None
University of Texas Southwestern Medical Center Childrens Medical Center Dallas - Dallas, Texas 1,551.5 miles None None None
University of Texas Southwestern Medical Center Childrens Medical Center Dallas - Dallas, Texas 1,551.5 miles None None None
University of Texas Southwestern Medical Center Childrens Medical Center Dallas - Dallas, Texas 1,551.5 miles None None None
University of Texas Southwestern Medical Center Childrens Medical Center Dallas - Dallas, Texas 1,551.5 miles None None None
University of Texas Southwestern Medical Center Childrens Medical Center Dallas - Dallas, Texas 1,551.5 miles None None None
University of Texas Medical School - Houston, Texas 1,609.9 miles None None None
University of Texas Medical School - Houston, Texas 1,609.9 miles None None None
University of Texas Medical School - Houston, Texas 1,609.9 miles None None None
University of Colorado Denver Childrens Hospital - Aurora, Colorado 1,759.8 miles None None None
University of Colorado Denver Childrens Hospital - Aurora, Colorado 1,759.8 miles None None None
University of Colorado Denver Childrens Hospital - Aurora, Colorado 1,759.8 miles None None None
University of Colorado Denver Childrens Hospital - Aurora, Colorado 1,759.8 miles None None None
University of Colorado Denver Childrens Hospital - Aurora, Colorado 1,759.8 miles None None None
University of Utah - Salt Lake City, Utah 2,094.1 miles None None None
University of Utah - Salt Lake City, Utah 2,094.1 miles None None None
Bloodworks Northwest formerly Puget Sound Blood Center Hemophilia - Seattle, Washington 2,489.6 miles None None None
Bloodworks Northwest formerly Puget Sound Blood Center Hemophilia - Seattle, Washington 2,489.6 miles None None None
Bloodworks Northwest formerly Puget Sound Blood Center Hemophilia - Seattle, Washington 2,489.6 miles None None None
Bloodworks Northwest formerly Puget Sound Blood Center Hemophilia - Seattle, Washington 2,489.6 miles None None None
Bloodworks Northwest formerly Puget Sound Blood Center Hemophilia - Seattle, Washington 2,489.6 miles None None None

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