A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors

Completed · Phase 3

Conditions studied: Hemophilia A

In brief

This is a randomized, global, multicenter, open-label, Phase 3 clinical study in participants with severe hemophilia A without inhibitors against Factor VIII (FVIII) who are 12 years or older. The study evaluates two prophylactic emicizumab regimens versus no prophylaxis in this population with emphasis on efficacy, safety, and pharmacokinetics.

Key facts

Study ID

NCT02847637

Run by

Hoffmann-La Roche

People needed

152

Starts

2016-09-27

Expected to finish

2022-05-12

Last updated by the study team

2022-11-16

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Body weight >/= 40 kilogram (kg) at the time of screening
• Diagnosis of severe congenital hemophilia A
• Documentation of the details of prophylactic or episodic FVIII treatment and of number of bleeding episodes for at least the last 24 weeks
• Adequate hematologic function
• Adequate hepatic function
• Adequate renal function
• For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods that result in a failure rate of less than (<) 1 percent (%) per year during the treatment period and for at least 5 elimination half-lives (24 weeks) after the last dose of study drug

You may not qualify if…

• Inherited or acquired bleeding disorder other than hemophilia A
• Previous or current treatment for thromboembolic disease or signs of thromboembolic disease
• Conditions that may increase risk of bleeding or thrombosis
• History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
• Known human immunodeficiency virus (HIV) infection with cluster of differentiation (CD) 4 count <200 cells per microliter (cells/mcL) within 24 weeks prior to screening. Participants with HIV infection who has CD4 greater than (>) 200 and meet all other criteria are eligible
• Use of systemic immunomodulators at enrollment or planned use during the study, with the exception of anti-retroviral therapy
• Participants who are at high risk for thrombotic microangiopathy (TMA) (for example, have a previous medical or family history of TMA), in the investigator's judgment
• Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose additional risk, or would, in the opinion of the investigator, preclude the participant's safe participation in and completion of the study
• Planned surgery (excluding minor procedures) during the study
• Receipt of emicizumab in a prior investigational study; an investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration; a non-hemophilia-related investigational drug concurrently, within last 30 days or 5 half-lives, whichever is shorter
• Pregnant or lactating, or intending to become pregnant during the study

Where it is running

• Santa Monica Oncology Center — Santa Monica, California, United States
• Georgetown Uni Medical Center; Lombardi Cancer Center — Washington D.C., District of Columbia, United States
• University of Florida — Gainesville, Florida, United States
• Winship Cancer Institute — Atlanta, Georgia, United States
• Dana-Farber Cancer Institute; Brigham and Women's Cancer Center — Boston, Massachusetts, United States
• Children's Hospital of Michigan; Pediatrics — Detroit, Michigan, United States
• Cornell Univ Medical College; Hematology-Oncolog — New York, New York, United States
• Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia — Seattle, Washington, United States
• Royal Adelaide Hospital — Adelaide, South Australia, Australia
• The Alfred Hospital, Melbourne; Thrombosis and Haemostasis Unit — Melbourne, Victoria, Australia
• The Perth Blood Institute — Nedlands, Western Australia, Australia
• 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
• St James's Hospital — Dublin, Ireland
• IRCCS Ca' Granda Ospedale Maggiore Policlinico; Centro Emofilia e Trombosi "Angelo Bianchi e Bonomi" — Milan, Lombardy, Italy
• AOU Careggi; SOD Malattie Emorragiche — Florence, Tuscany, Italy
• Nagoya University Hospital — Aichi, Japan
• St. Marianna University Hospital — Kanagawa, Japan
• Sendai Medical Center — Miyagi, Japan
• Nara Medical University Hospital — Nara, Japan
• NHO Osaka National Hospital — Osaka, Japan
• Tokyo Medical University Hospital — Tokyo, Japan

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.