A Study of Emicizumab Administered Subcutaneously (SC) in Pediatric Participants With Hemophilia A and Factor VIII (FVIII) Inhibitors
NCT ID: NCT02795767
Last Updated: 2021-06-02
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE3
88 participants
INTERVENTIONAL
2016-07-22
2020-11-11
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks in Participants With Hemophilia A
NCT03020160
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors
NCT04431726
A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors
NCT02847637
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors
NCT04158648
Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants
NCT03315455
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Cohort A: 1.5 mg/kg Emicizumab QW
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.
Emicizumab
Emicizumab will be administered as per the schedule specified in the respective arm.
Cohort B: 3 mg/kg Emicizumab Q2W
Participants will receive emicizumab at a loading dose of 3 mg/kg QW SC for the first 4 weeks followed by a maintenance dose of 3 mg/kg every 2 weeks (Q2W) 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.
Emicizumab
Emicizumab will be administered as per the schedule specified in the respective arm.
Cohort C: 6 mg/kg Emicizumab Q4W
Participants will receive emicizumab at a loading dose of 3 mg/kg QW SC for the first 4 weeks followed by a maintenance dose of 6 mg/kg every 4 weeks (Q4W) 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.
Emicizumab
Emicizumab will be administered as per the schedule specified in the respective arm.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Emicizumab
Emicizumab will be administered as per the schedule specified in the respective arm.
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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
* 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
17 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Chugai Pharmaceutical
INDUSTRY
Hoffmann-La Roche
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Clinical Trials
Role: STUDY_DIRECTOR
Hoffmann-La Roche
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
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)
Istanbul University, Cerrahpasa Medical Faculty; Pediatrics Department
Istanbul, , Turkey (Türkiye)
Ege University, School of Medicine; Pediatrics Department
Izmir, , Turkey (Türkiye)
Great Ormond street Hospital for Children NHS Foundation Trust; Haemophilia Centre
London, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Kruse-Jarres R, Peyvandi F, Oldenburg J, Chang T, Chebon S, Doral MY, Croteau SE, Lambert T, Kempton CL, Pipe SW, Ko RH, Trzaskoma B, Dhalluin C, Bienz NS, Niggli M, Lehle M, Paz-Priel I, Young G, Jimenez-Yuste V. Surgical outcomes in people with hemophilia A taking emicizumab prophylaxis: experience from the HAVEN 1-4 studies. Blood Adv. 2022 Dec 27;6(24):6140-6150. doi: 10.1182/bloodadvances.2022007458.
Young G, Liesner R, Chang T, Sidonio R, Oldenburg J, Jimenez-Yuste V, Mahlangu J, Kruse-Jarres R, Wang M, Uguen M, Doral MY, Wright LY, Schmitt C, Levy GG, Shima M, Mancuso ME. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019 Dec 12;134(24):2127-2138. doi: 10.1182/blood.2019001869.
Provided Documents
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2016-000073-21
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
BH29992
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.