A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors
A Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Subcutaneous Administration of Emicizumab in Hemophilia A Pediatric Patients With Inhibitors
This non-randomized, multicenter, open-label, Phase III clinical study will evaluate the efficacy, safety, and pharmacokinetics of emicizumab administered subcutaneously initially once weekly (QW) in pediatric participants with hemophilia A with FVIII inhibitors. This study will open two additional non-randomized cohorts to investigate once every 2 weeks (Q2W) and once every 4 weeks (Q4W) regimens in pediatric participants.
Cohort A: Model-Based Annualized Bleed Rate (ABR) for Treated Bleeds in Treated Participants <12 Years of Age
Cohorts B and C: Model-Based Annualized Bleed Rate (ABR) for Treated Bleeds in Treated Participants <12 Years of Age
Study Arms / Comparison Groups
Cohort A: 1.5 mg/kg Emicizumab QW
Description: Participants will receive emicizumab at a loading dose of 3 milligrams per kilogram (mg/kg) QW SC for the first 4 weeks followed by a maintenance dose of 1.5 mg/kg QW SC for a minimum of 52 weeks, or until unacceptable toxicity, discontinuation from the study due to any cause, or other criteria set forth in the protocol, whichever occurs first.
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
July 22, 2016
November 11, 2020
Primary Completion Date
April 30, 2018
Inclusion Criteria: - Children less than (<) 12 years of age, with allowance for participants 12 to 17 years of age who weigh <40 kilograms (kg) (Cohort A only); and participants <2 years of age will be allowed to participate only after the protocol-defined interim data review criteria are met (Cohort A only) - 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 [BU]) - Requires treatment with bypassing agents - Adequate hematologic, hepatic, and renal function Exclusion Criteria: - Inherited or acquired bleeding disorder other than hemophilia A - Ongoing (or planning to receive during the study) immune tolerance induction (ITI) therapy or prophylaxis treatment with FVIII - Previous (in the past 12 months) or current treatment for thromboembolic disease or signs of thromboembolic disease - Other disease that may increase risk of bleeding or thrombosis - History of clinically significant hypersensitivity associated with monoclonal antibody therapy or components of the emicizumab injection - Known infection with human immunodeficiency virus (HIV) or hepatitis B or C virus - Use of systemic immunomodulators at enrollment or planned use during the study period - Planned surgery (excluding minor procedures such as tooth extraction or incision and drainage) during the study - Inability (or unwillingness by caregiver) to receive (allow receipt of) blood or blood products (or any standard-of-care treatment for a life-threatening condition) - Participants who are at high risk for thrombotic microangiopathy (TMA) (e.g., have a previous medical or family history of TMA), in the investigator's judgement
N/A - 17 Years
Accepts Healthy Volunteers
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Clinical Trials, Study Director, Hoffmann-La Roche