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

Primary Purpose

Hemophilia A

Status
Approved for marketing
Phase
Locations
United States
Study Type
Expanded Access
Intervention
Emicizumab
Sponsored by
Genentech, Inc.
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Hemophilia A

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All Sexes

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

Sites / Locations

  • University of Colorado Denver, Children's Hospital
  • University of Miami Miller School of Medicine
  • Rush Medical Center
  • Tulane Medical Center; Investigational/Research Pharmacy
  • Boston Childrens Hospital
  • University of Minnesota
  • Children's Mercy Hosp Clinics
  • Barnabas Health Newark Beth Israel Medical Center - Pulmonary Hypertension & Lung Transplant Program
  • Nationwide Children's Hospital
  • University of Oklahoma Health Sciences Center
  • University of Texas Southwestern Medical Center - Children's Medical Center Dallas
  • University of Texas Medical School
  • University of Utah
  • Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
May 12, 2017
Last Updated
March 26, 2018
Sponsor
Genentech, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03154437
Brief Title
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

2. Study Status

Record Verification Date
March 2018
Overall Recruitment Status
Approved for marketing
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genentech, Inc.

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type
Biological
Intervention Name(s)
Emicizumab
Other Intervention Name(s)
ACE910, CH5534262
Intervention Description
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.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Trials
Organizational Affiliation
Hoffmann-La Roche
Official's Role
Study Director
Facility Information:
Facility Name
University of Colorado Denver, Children's Hospital
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of Miami Miller School of Medicine
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Rush Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Tulane Medical Center; Investigational/Research Pharmacy
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70112
Country
United States
Facility Name
Boston Childrens Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Children's Mercy Hosp Clinics
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Barnabas Health Newark Beth Israel Medical Center - Pulmonary Hypertension & Lung Transplant Program
City
Newark
State/Province
New Jersey
ZIP/Postal Code
07112
Country
United States
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
University of Oklahoma Health Sciences Center
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
Country
United States
Facility Name
University of Texas Southwestern Medical Center - Children's Medical Center Dallas
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Facility Name
University of Texas Medical School
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia
City
Seattle
State/Province
Washington
ZIP/Postal Code
98104
Country
United States

12. IPD Sharing Statement

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

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