An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors
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| ClinicalTrials.gov Identifier: NCT03154437 |
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Expanded Access Status :
Approved for marketing
First Posted : May 16, 2017
Last Update Posted : March 29, 2018
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Sponsor:
Genentech, Inc.
Information provided by (Responsible Party):
Genentech, Inc.
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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.
| Condition or disease | Intervention/treatment |
|---|---|
| Hemophilia A | Biological: Emicizumab |
| Study Type : | Expanded Access |
| Expanded Access Type : | Treatment IND/Protocol |
| See clinical trials of the intervention/treatment in this expanded access record. | |
| Official Title: | An Open-Label, Multicenter, Expanded Access Program for Emicizumab in Patients With Hemophilia A With Inhibitors |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Hemophilia
MedlinePlus related topics:
Hemophilia
Drug Information
available for:
Emicizumab
Intervention Details:
- 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.Other Names:
- ACE910
- CH5534262
Information from the National Library of Medicine
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 12 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
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
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03154437
Locations
| United States, Colorado | |
| University of Colorado Denver, Children's Hospital | |
| Aurora, Colorado, United States, 80045 | |
| United States, Florida | |
| University of Miami Miller School of Medicine | |
| Miami, Florida, United States, 33136 | |
| United States, Illinois | |
| Rush Medical Center | |
| Chicago, Illinois, United States, 60612 | |
| United States, Louisiana | |
| Tulane Medical Center; Investigational/Research Pharmacy | |
| New Orleans, Louisiana, United States, 70112 | |
| United States, Massachusetts | |
| Boston Childrens Hospital | |
| Boston, Massachusetts, United States, 02115 | |
| United States, Minnesota | |
| University of Minnesota | |
| Minneapolis, Minnesota, United States, 55455 | |
| United States, Missouri | |
| Children's Mercy Hosp Clinics | |
| Kansas City, Missouri, United States, 64108 | |
| United States, New Jersey | |
| Barnabas Health Newark Beth Israel Medical Center - Pulmonary Hypertension & Lung Transplant Program | |
| Newark, New Jersey, United States, 07112 | |
| United States, Ohio | |
| Nationwide Children's Hospital | |
| Columbus, Ohio, United States, 43205 | |
| United States, Oklahoma | |
| University of Oklahoma Health Sciences Center | |
| Oklahoma City, Oklahoma, United States, 73104 | |
| United States, Texas | |
| University of Texas Southwestern Medical Center - Children's Medical Center Dallas | |
| Dallas, Texas, United States, 75235 | |
| University of Texas Medical School | |
| Houston, Texas, United States, 77030 | |
| United States, Utah | |
| University of Utah | |
| Salt Lake City, Utah, United States, 84132 | |
| United States, Washington | |
| Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia | |
| Seattle, Washington, United States, 98104 | |
Sponsors and Collaborators
Genentech, Inc.
Investigators
| Study Director: | Clinical Trials | Hoffmann-La Roche |
| Responsible Party: | Genentech, Inc. |
| ClinicalTrials.gov Identifier: | NCT03154437 |
| Other Study ID Numbers: |
ML39356 |
| First Posted: | May 16, 2017 Key Record Dates |
| Last Update Posted: | March 29, 2018 |
| Last Verified: | March 2018 |
Additional relevant MeSH terms:
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Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn |