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

NCT02795767

CompletedPhase 3

Sponsor: Hoffmann-La Roche

Conditions: Hemophilia A

Interventions: Emicizumab

Countries: United States, Costa Rica, France, Germany, Italy, Japan, South Africa, Spain

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.

Eligibility overview

Sex: ALL

Age: — to 17 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

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
Locations (28)
  • Los Angeles, California, United States
  • Aurora, Colorado, United States
  • Atlanta, Georgia, United States
  • Chicago, Illinois, United States
  • Detroit, Michigan, United States
  • New Hyde Park, New York, United States
  • Portland, Oregon, United States
  • Seattle, Washington, United States
  • San José, Costa Rica
  • Bron, France
  • Le Kremlin-Bicêtre, France
  • Paris, France
  • Bonn, Germany
  • Milan, Lombardy, Italy
  • Aichi, Japan
  • Kitakyushu-shi, Japan
  • Nara, Japan
  • Shizuoka, Japan
  • Tokyo, Japan
  • Johannesburg, South Africa
  • Madrid, Spain
  • Seville, Spain
  • Valencia, Spain
  • Adana, Turkey (Türkiye)
  • Istanbul, Turkey (Türkiye)
  • Istanbul, Turkey (Türkiye)
  • Izmir, Turkey (Türkiye)
  • London, United Kingdom