A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors
Completed · Phase 3
Conditions studied: Hemophilia A
In brief
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.
Key facts
- Study ID
- NCT02795767
- Run by
- Hoffmann-La Roche
- People needed
- 88
- Starts
- 2016-07-22
- Expected to finish
- 2020-11-11
- Last updated by the study team
- 2021-06-02
Who can join
Age: any, up to 17. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
Where it is running
- Children's Hospital Los Angeles — Los Angeles, California, United States
- University of Colorado Denver, Children's Hospital — Aurora, Colorado, United States
- Children'S Healthcare of Atlanta — Atlanta, Georgia, United States
- Rush Medical Center — Chicago, Illinois, United States
- Children's Hospital of Michigan; Pediatrics — Detroit, Michigan, United States
- North Shore/Long Island Jewish PRIME; Pediatric Hematology/Oncology & Stem Cell Transplantation — New Hyde Park, New York, United States
- Oregon Health & Science Uni ; Dept of Pediatrics — Portland, Oregon, United States
- Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia — Seattle, Washington, United States
- ICIC — San José, Costa Rica
- Hopital Cardio-vasculaire Louis Pradel; Hemostase clinique — Bron, France
- CH de Bicetre; Centre de Traitement d' Hemophilie — Le Kremlin-Bicêtre, France
- Groupe Hospitalier Necker Enfants Malades — Paris, France
- Universitätsklinikum Bonn; Institut für Experimentelle Hämatologie und Transfusionsmedizin — Bonn, Germany
- IRCCS Ca' Granda Ospedale Maggiore Policlinico; Centro Emofilia e Trombosi "Angelo Bianchi e Bonomi" — Milan, Lombardy, Italy
- Nagoya University Hospital — Aichi, Japan
- Hospital of the University of Occupational and Environmental Health,Japan — Kitakyushu-shi, Japan
- Nara Medical University Hospital — Nara, Japan
- Shizuoka Children's Hospital — Shizuoka, Japan
- Ogikubo Hospital — Tokyo, Japan
- Charlotte Maxeke Johannesburg Hospital; Haemophilia Comprehensive Care Center — Johannesburg, South Africa
- Hospital Universitario la Paz; Servicio de Hematologia — Madrid, Spain
- Hospital Universitario Virgen del Rocio; Servicio de Hematologia — Seville, Spain
- Hospital Universitario la Fe; Servicio de Hematologia — Valencia, Spain
- Adana Acibadem Hospital; Pediatric Hematology — Adana, Turkey (Türkiye)
- Istanbul Uni Istanbul Medical Faculty — Istanbul, Turkey (Türkiye)
Full record on ClinicalTrials.gov
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