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History of Changes for Study: NCT02847637
A Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors
Latest version (submitted June 1, 2022) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 July 26, 2016 None (earliest Version on record)
2 August 1, 2016 Study Status
3 September 1, 2016 Contacts/Locations and Study Status
4 October 3, 2016 Study Status, Contacts/Locations and Study Design
5 November 1, 2016 Contacts/Locations and Study Status
6 November 4, 2016 Study Identification and Study Status
7 March 30, 2017 Study Status, Contacts/Locations and Study Identification
8 May 31, 2017 Recruitment Status, Study Status, Outcome Measures, Contacts/Locations, Arms and Interventions, Oversight, Study Identification, Eligibility, Study Design and Study Description
9 August 18, 2017 Study Status and Contacts/Locations
10 November 17, 2017 Study Status and Contacts/Locations
11 January 16, 2018 Contacts/Locations, Study Status and Study Design
12 April 16, 2018 Contacts/Locations and Study Status
13 July 13, 2018 Study Status and Contacts/Locations
Show
Results Submission Events
14 October 19, 2018 Outcome Measures, Study Status, Arms and Interventions, Document Section and Results
15 January 17, 2019 Study Status
16 April 17, 2019 Study Status and Contacts/Locations
17 July 16, 2019 Study Status
18 October 11, 2019 Study Status
19 January 9, 2020 Study Status and Contacts/Locations
20 April 8, 2020 Study Status
21 July 7, 2020 Study Status
22 September 8, 2020 Arms and Interventions and Study Status
23 August 6, 2021 Study Status and Contacts/Locations
24 February 2, 2022 Contacts/Locations and Study Status
25 May 2, 2022 Study Status and Contacts/Locations
26 June 1, 2022 Recruitment Status and Study Status
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Study NCT02847637
Submitted Date:  July 26, 2016 (v1)

Open or close this module Study Identification
Unique Protocol ID: BH30071
Brief Title: A Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of 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
Secondary IDs: 2016-000072-17 [EudraCT Number]
Open or close this module Study Status
Record Verification: July 2016
Overall Status: Recruiting
Study Start: June 2016
Primary Completion: March 2018 [Anticipated]
Study Completion: March 2018 [Anticipated]
First Submitted: July 26, 2016
First Submitted that
Met QC Criteria:
July 26, 2016
First Posted: July 28, 2016 [Estimate]
Last Update Submitted that
Met QC Criteria:
July 26, 2016
Last Update Posted: July 28, 2016 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: Hoffmann-La Roche
Responsible Party: Sponsor
Collaborators: Chugai Pharmaceutical
Open or close this module Oversight
U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring:
Open or close this module Study Description
Brief Summary: This is a randomized, multicenter, open-label, Phase 3 clinical study in participants aged 12 years or older to evaluate the efficacy, safety, and pharmacokinetics of prophylactic emicizumab versus no prophylaxis in participants with severe hemophilia A without inhibitors against FVIII.
Detailed Description:
Open or close this module Conditions
Conditions: Hemophilia A
Keywords: Hemophilia A
Emicizumab
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 3
Interventional Study Model: Parallel Assignment
Number of Arms: 4
Masking: None (Open Label)
Allocation: Randomized
Enrollment: 135 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
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 milligram per kilogram per week (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 Names:
  • ACE910; RO5534262
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 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 Names:
  • 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 Names:
  • 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 Names:
  • ACE910; RO5534262
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Number of bleeds over time
[ Time Frame: 24 weeks ]

Secondary Outcome Measures:
1. Reduction in number of bleeds over time
[ Time Frame: Baseline, 24 weeks ]

2. Reduction in number of joint bleeds over time
[ Time Frame: Baseline, 24 weeks ]

3. Reduction in number of target joint bleeds over time
[ Time Frame: Baseline, 24 weeks ]

4. Health related quality of life scores
[ Time Frame: 24 weeks ]

5. Trough plasma concentration (Ctrough) of emicizumab
[ Time Frame: (Pre-dose) 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 (maximum up to 2 years) ]

Open or close this module Eligibility
Minimum Age: 12 Years
Maximum Age:
Sex: All
Gender Based:
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 <1% 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 CD4 count <200 cells/mcL within 24 weeks prior to screening. Participants with HIV infection who has CD4 >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
  • 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
Open or close this module Contacts/Locations
Central Contact Person: Reference Study ID Number: BH30071 www.roche.com/about_roche/roche_worldwide.htm
Telephone: 888-662-6728 (U.S. and Canada)
Email: global.rochegenentechtrials@roche.com
Study Officials: Clinical Trials
Study Director
Hoffmann-La Roche
Locations: United States, California
[Not yet recruiting]
Santa Monica, California, United States, 90403
United States, District of Columbia
[Not yet recruiting]
Washington, District of Columbia, United States, 20007
United States, Florida
[Not yet recruiting]
Gainesville, Florida, United States, 32610
United States, Georgia
[Not yet recruiting]
Atlanta, Georgia, United States, 30322
United States, Massachusetts
[Not yet recruiting]
Boston, Massachusetts, United States, 02115
United States, Michigan
[Not yet recruiting]
Detroit, Michigan, United States, 48201
United States, New York
[Not yet recruiting]
New York, New York, United States, 10021
[Not yet recruiting]
New York, New York, United States, 10029
United States, Oregon
[Not yet recruiting]
Portland, Oregon, United States, 97201
United States, Washington
[Not yet recruiting]
Seattle, Washington, United States, 98104
Australia, South Australia
[Not yet recruiting]
Adelaide, South Australia, Australia, 5000
Australia, Victoria
[Not yet recruiting]
Melbourne, Victoria, Australia, 3004
Australia, Western Australia
[Not yet recruiting]
Nedlands, Western Australia, Australia
Costa Rica
[Not yet recruiting]
San Jose, Costa Rica, 10904
Germany
[Not yet recruiting]
Bonn, Germany, 53127
Italy, Lombardia
[Not yet recruiting]
Milano, Lombardia, Italy, 20122
Italy, Toscana
[Not yet recruiting]
Firenze, Toscana, Italy, 50134
Japan
[Not yet recruiting]
Aichi, Japan, 466-8560
[Not yet recruiting]
Kanagawa, Japan, 216-8511
[Not yet recruiting]
Miyagi, Japan, 983-8520
[Not yet recruiting]
Nara, Japan, 634-8521
[Not yet recruiting]
Osaka, Japan, 540-0006
[Not yet recruiting]
Saitama, Japan, 339-8551
[Recruiting]
Tokyo, Japan, 167-0035
Poland
[Not yet recruiting]
Gdansk, Poland, 80-952
[Not yet recruiting]
Lublin, Poland, 20-081
[Not yet recruiting]
Poznan, Poland, 61-828
[Not yet recruiting]
Warsaw, Poland, 02-776
South Africa
[Not yet recruiting]
Johannesburg, South Africa, 2193
Spain
[Not yet recruiting]
Madrid, Spain, 28046
[Not yet recruiting]
Sevilla, Spain, 41013
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Links:
Available IPD/Information:

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