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

Brief Title

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

Official Title

A Randomized, Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Patients Without Inhibitors

Brief Summary

      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.

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Annualized Bleeding Rate (ABR) for Treated Bleeds

Secondary Outcome

 Annualized Bleeding Rate (ABR) for All Bleeds

Condition

Hemophilia A

Intervention

Emicizumab

Study Arms / Comparison Groups

 A: Emicizumab 1.5 mg/kg/week

Description:  Participants who received episodic treatment with FVIII prior to study entry will receive emicizumab prophylaxis at a dose of 3 milligrams per kilogram per week (mg/kg/week) subcutaneously for 4 weeks, followed by 1.5 mg/kg/week emicizumab subcutaneously until the end of study (maximum up to 2 years).

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Drug

Estimated Enrollment

Start Date

September 27, 2016

Completion Date

May 10, 2022

Primary Completion Date

September 15, 2017

Eligibility Criteria

        Inclusion Criteria:

          -  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

        Exclusion Criteria:

          -  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

Gender

All

Ages

12 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Clinical Trials, ,

Location Countries

Australia

Location Countries

Australia

Administrative Informations

NCT ID

NCT02847637

Organization ID

BH30071

Secondary IDs

2016-000072-17

Responsible Party

Sponsor

Study Sponsor

Hoffmann-La Roche

Collaborators

 Chugai Pharmaceutical

Study Sponsor

Clinical Trials, Study Director, Hoffmann-La Roche

Verification Date

July 2020