A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors (HAVEN2)

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

  • Hemophilia A
Trial Status:

Completed

This trial runs in
Countries
  • Costa Rica
  • France
  • Germany
  • Italy
  • Japan
  • South Africa
  • Spain
  • Turkey
  • United Kingdom
  • United States
Trial Identifier:

NCT02795767 2016-000073-21, HAVEN2 BH29992

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      The source of the below information is the publicly available website ClinicalTrials.gov. It has been summarised and edited into simpler language.

      The below information is taken directly from the publicly available website ClinicalTrials.gov within a week of any updates, and has not been edited.

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      Trial 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.

      Hoffmann-La Roche Sponsor
      Phase 3 Phase
      NCT02795767 , BH29992 , 2016-000073-21, HAVEN2 Trial Identifier
      Emicizumab Treatments
      Hemophilia A Condition
      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

      Eligibility Criteria

      All Gender
      ≤ 17 Years Age
      No Healthy Volunteers
      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

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