Brief Title
A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors
Official Title
A Randomized, Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Patients Without Inhibitors
Brief Summary
This is a randomized, global, multicenter, open-label, Phase 3 clinical study in participants with severe hemophilia A without inhibitors against Factor VIII (FVIII) who are 12 years or older. The study evaluates two prophylactic emicizumab regimens versus no prophylaxis in this population with emphasis on efficacy, safety, and pharmacokinetics.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Annualized Bleeding Rate (ABR) for Treated Bleeds
Secondary Outcome
Annualized Bleeding Rate (ABR) for All Bleeds
Condition
Hemophilia A
Intervention
Emicizumab
Study Arms / Comparison Groups
A: Emicizumab 1.5 mg/kg/week
Description: Participants who received episodic treatment with FVIII prior to study entry will receive emicizumab prophylaxis at a dose of 3 milligrams per kilogram per week (mg/kg/week) subcutaneously for 4 weeks, followed by 1.5 mg/kg/week emicizumab subcutaneously until the end of study (maximum up to 6 years).
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
152
Start Date
September 27, 2016
Completion Date
May 12, 2022
Primary Completion Date
September 15, 2017
Eligibility Criteria
Inclusion Criteria: - Body weight >/= 40 kilogram (kg) at the time of screening - Diagnosis of severe congenital hemophilia A - Documentation of the details of prophylactic or episodic FVIII treatment and of number of bleeding episodes for at least the last 24 weeks - Adequate hematologic function - Adequate hepatic function - Adequate renal function - For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods that result in a failure rate of less than (<) 1 percent (%) per year during the treatment period and for at least 5 elimination half-lives (24 weeks) after the last dose of study drug Exclusion Criteria: - Inherited or acquired bleeding disorder other than hemophilia A - Previous or current treatment for thromboembolic disease or signs of thromboembolic disease - Conditions that may increase risk of bleeding or thrombosis - History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection - Known human immunodeficiency virus (HIV) infection with cluster of differentiation (CD) 4 count <200 cells per microliter (cells/mcL) within 24 weeks prior to screening. Participants with HIV infection who has CD4 greater than (>) 200 and meet all other criteria are eligible - Use of systemic immunomodulators at enrollment or planned use during the study, with the exception of anti-retroviral therapy - Participants who are at high risk for thrombotic microangiopathy (TMA) (for example, have a previous medical or family history of TMA), in the investigator's judgment - Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose additional risk, or would, in the opinion of the investigator, preclude the participant's safe participation in and completion of the study - Planned surgery (excluding minor procedures) during the study - Receipt of emicizumab in a prior investigational study; an investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration; a non-hemophilia-related investigational drug concurrently, within last 30 days or 5 half-lives, whichever is shorter - Pregnant or lactating, or intending to become pregnant during the study
Gender
All
Ages
12 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Clinical Trials, ,
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT02847637
Organization ID
BH30071
Secondary IDs
2016-000072-17
Responsible Party
Sponsor
Study Sponsor
Hoffmann-La Roche
Collaborators
Chugai Pharmaceutical
Study Sponsor
Clinical Trials, Study Director, Hoffmann-La Roche
Verification Date
June 2022