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A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors (HAVEN 3)

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02847637
First Posted: July 28, 2016
Last Update Posted: November 21, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Chugai Pharmaceutical
Information provided by (Responsible Party):
Hoffmann-La Roche
  Purpose
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.

Condition Intervention Phase
Hemophilia A Drug: Emicizumab Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Hoffmann-La Roche:

Primary Outcome Measures:
  • Number of Bleeds Over Time [ Time Frame: Baseline up to 24 weeks ]

Secondary Outcome Measures:
  • Reduction in Number of Bleeds Over Time [ Time Frame: Baseline up to 24 weeks ]
  • Reduction in Number of Joint Bleeds Over Time [ Time Frame: Baseline up to 24 weeks ]
  • Reduction in Number of Target Joint Bleeds Over Time [ Time Frame: Baseline up to 24 weeks ]
  • Hemophilia A Quality of Life (Haemo-A-QoL) Questionnaire Score in Participants Greater Than or Equal to (>/=) 18 Years of Age [ Time Frame: Week 24 ]
  • Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Score in Participants 12 to 17 Years of Age [ Time Frame: Week 24 ]
  • European Quality of Life 5-Dimensions-5 Levels Questionnaire (EQ-5D-5L) Score [ Time Frame: Week 24 ]
  • Trough Plasma Concentration (Ctrough) of Emicizumab [ Time Frame: Predose (Hour 0) on every week during Weeks 1-4, every 2 weeks during Weeks 5-8, every 4 weeks during Weeks 9-24, every 8 weeks during Weeks 25-48, every 12 weeks thereafter up to the end of the study (up to 2 years) ]
  • Percentage of Participants With Adverse Events (AEs) [ Time Frame: Baseline up to 2 years ]
  • Percentage of Participants With Anti-Emicizumab Antibodies [ Time Frame: Baseline up to 2 years ]
  • Percentage of Participants With De Novo Development of FVIII Inhibitors [ Time Frame: Baseline up to 2 years ]

Enrollment: 145
Actual Study Start Date: September 27, 2016
Estimated Study Completion Date: September 30, 2019
Primary Completion Date: September 15, 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Emicizumab 1.5 mg/kg/week
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).
Drug: Emicizumab
Participants will receive emicizumab prophylaxis at the specified dose subcutaneously until the end of the study (maximum up to 2 years).
Other Name: ACE910; RO5534262
Experimental: Emicizumab 3 mg/kg/2 weeks
Participants who received episodic treatment with FVIII prior to study entry will receive emicizumab prophylaxis at a dose of 3 mg/kg/week subcutaneously for 4 weeks, followed by 3 mg/kg/2 weeks emicizumab subcutaneously until the end of study (maximum up to 2 years).
Drug: Emicizumab
Participants will receive emicizumab prophylaxis at the specified dose subcutaneously until the end of the study (maximum up to 2 years).
Other Name: ACE910; RO5534262
Active Comparator: No Prophylaxis
Participants who received episodic treatment with FVIII prior to study entry will be randomized to continue episodic FVIII treatment when they start the trial; they will have the opportunity to switch to emicizumab prophylaxis after 24 weeks on-study.
Drug: Emicizumab
Participants will receive emicizumab prophylaxis at the specified dose subcutaneously until the end of the study (maximum up to 2 years).
Other Name: ACE910; RO5534262
Experimental: Emicizumab (Pre-study FVIII Prophylaxis)
Participants who received FVIII prophylaxis prior to study entry will receive emicizumab prophylaxis at a dose of 3 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).
Drug: Emicizumab
Participants will receive emicizumab prophylaxis at the specified dose subcutaneously until the end of the study (maximum up to 2 years).
Other Name: ACE910; RO5534262

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02847637


  Show 44 Study Locations
Sponsors and Collaborators
Hoffmann-La Roche
Chugai Pharmaceutical
Investigators
Study Director: Clinical Trials Hoffmann-La Roche
  More Information

Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT02847637     History of Changes
Other Study ID Numbers: BH30071
2016-000072-17 ( EudraCT Number )
First Submitted: July 26, 2016
First Posted: July 28, 2016
Last Update Posted: November 21, 2017
Last Verified: November 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Hoffmann-La Roche:
Hemophilia A
Emicizumab

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Antibodies, Bispecific
Coagulants
Immunologic Factors
Physiological Effects of Drugs