Brief Title
A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors
Official Title
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
Brief Summary
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.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Cohort A: Model-Based Annualized Bleed Rate (ABR) for Treated Bleeds in Treated Participants <12 Years of Age
Secondary Outcome
Cohorts B and C: Model-Based Annualized Bleed Rate (ABR) for Treated Bleeds in Treated Participants <12 Years of Age
Condition
Hemophilia A
Intervention
Emicizumab
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.
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
88
Start Date
July 22, 2016
Completion Date
November 11, 2020
Primary Completion Date
April 30, 2018
Eligibility Criteria
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
Gender
All
Ages
N/A - 17 Years
Accepts Healthy Volunteers
No
Contacts
Clinical Trials, ,
Location Countries
Costa Rica
Location Countries
Costa Rica
Administrative Informations
NCT ID
NCT02795767
Organization ID
BH29992
Secondary IDs
2016-000073-21
Responsible Party
Sponsor
Study Sponsor
Hoffmann-La Roche
Collaborators
Chugai Pharmaceutical
Study Sponsor
Clinical Trials, Study Director, Hoffmann-La Roche
Verification Date
May 2021