A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors

• Status: Completed
• Trial end date: 2020-11-11
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hoffmann-La Roche
• Collaborator: Chugai Pharmaceutical
• Treatments: Factor VIII

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