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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02847637




Registration number
NCT02847637
Ethics application status
Date submitted
26/07/2016
Date registered
28/07/2016
Date last updated
19/04/2019

Titles & IDs
Public title
A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors
Scientific 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 ID [1] 0 0
2016-000072-17
Secondary ID [2] 0 0
BH30071
Universal Trial Number (UTN)
Trial acronym
HAVEN 3
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia A 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Emicizumab

Experimental: A: 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).

Experimental: B: 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).

Active Comparator: C: 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.

Experimental: D: Emicizumab 1.5 mg/kg/week (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).


Treatment: Drugs: Emicizumab
Participants will receive emicizumab prophylaxis at the specified dose subcutaneously until the end of the study (maximum up to 2 years).

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Annualized Bleeding Rate (ABR) for Treated Bleeds - The number of treated bleeds over the efficacy period is presented as an annualized bleeding rate (ABR) that was assessed using a negative binomial (NB) regression model, which accounts for different follow-up times, with the number of bleeds as a function of randomization and the time that each participant stays in the study (i.e., length of the efficacy period) included as an offset in the model. The model also includes the number of bleeds (<9 or =9) in the last 24 weeks prior to study entry as a stratification factor. A bleed is considered a "treated bleed" if it is directly followed (i.e., no intervening bleed) by a hemophilia medication reported to be a "treatment for bleed", irrespective of the time between treatment and the preceding bleed. Bleeds due to surgery/procedure are excluded.
Timepoint [1] 0 0
From Baseline to at least 24 weeks
Secondary outcome [1] 0 0
Annualized Bleeding Rate (ABR) for All Bleeds - The number of all bleeds over the efficacy period is presented as an annualized bleeding rate (ABR) that was assessed using a NB regression model, which accounts for different follow-up times, with the patient's number of bleeds as a function of randomization and the time that each patient stays in the study (i.e., length of the efficacy period) included as an offset in the model. The model also includes the number of bleeds (<9 or =9) in the last 24 weeks prior to study entry as a stratification factor. "All bleeds" comprises both treated and non-treated bleeds. In this definition, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
Timepoint [1] 0 0
From Baseline to at least 24 weeks
Secondary outcome [2] 0 0
Annualized Bleeding Rate (ABR) for Treated Joint Bleeds - The number of treated joint bleeds over the efficacy period is presented as an annualized bleeding rate (ABR) that was assessed using a NB regression model, which accounts for different follow-up times, with the patient's number of bleeds as a function of randomization and the time that each patient stays in the study (i.e., length of the efficacy period) included as an offset in the model. The model also includes the number of bleeds (<9 or =9) in the last 24 weeks prior to study entry as a stratification factor. A "joint bleed" is defined as a bleed reported as "joint" and with at least one of the following symptoms: increasing swelling or warmth of the skin over the joint; and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline. It is considered a "treated joint bleed" if it is directly followed (i.e., no intervening bleed) by a hemophilia medication reported to be a "treatment for bleed". Bleeds due to surgery/procedure are excluded.
Timepoint [2] 0 0
From Baseline to at least 24 weeks
Secondary outcome [3] 0 0
Annualized Bleeding Rate (ABR) for Treated Spontaneous Bleeds - The number of treated spontaneous bleeds over the efficacy period is presented as an annualized bleeding rate (ABR) that was assessed using a NB regression model, which accounts for different follow-up times, with the patient's number of bleeds as a function of randomization and the time that each patient stays in the study (i.e., length of the efficacy period) included as an offset in the model. The model also includes the number of bleeds (<9 or =9) in the last 24 weeks prior to study entry as a stratification factor. A bleed is classified as "spontaneous" if there is no other known contributing factor such as trauma or procedure/surgery. A "treated spontaneous bleed" is a spontaneous bleed that is directly followed (i.e., no intervening bleed) by a hemophilia medication reported to be a "treatment for bleed". Bleeds due to surgery/procedure are excluded.
Timepoint [3] 0 0
From baseline to at least 24 weeks
Secondary outcome [4] 0 0
Annualized Bleeding Rate (ABR) for Treated Target Joint Bleeds - The number of treated target joint bleeds over the efficacy period is presented as an annualized bleeding rate (ABR) that was assessed using a NB regression model, which accounts for different follow-up times, with the patient's number of bleeds as a function of randomization and the time that each patient stays in the study (i.e., length of the efficacy period) included as an offset in the model. The model also includes the number of bleeds (<9 or =9) in the last 24 weeks prior to study entry as a stratification factor. A "target joint bleed" is defined as a bleed reported as a joint bleed into a target joint, defined as at least 3 bleeds into the same joint during the last 24 weeks prior to study entry. It is considered a "treated target joint bleed" if it is directly followed (i.e., no intervening bleed) by a hemophilia medication reported to be a "treatment for bleed". Bleeds due to surgery/procedure are excluded.
Timepoint [4] 0 0
From Baseline to at least 24 weeks
Secondary outcome [5] 0 0
Intra-Participant Comparison of ABR for Treated Bleeds on Study Versus Pre-Study in Participants From the Non-Interventional Study Population Previously Treated With Factor VIII (FVIII) Prophylaxis (NISP) - This is an intra-participant comparison of the annualized bleeding rate (ABR) for treated bleeds on study versus pre-study in the NIS population previously treated with FVIII prophylaxis in NIS BH29768. The number of treated bleeds over the efficacy period is presented as an ABR that was assessed using a NB regression model, which accounts for different follow-up times, with the number of bleeds as a function of treatment and the time that each participant stays in the study (i.e., length of the efficacy period) included as an offset in the model. The model also includes a repeated statement to account for intra-participant comparison. A bleed is considered a "treated bleed" if it is directly followed (i.e., no intervening bleed) by a hemophilia medication reported to be a "treatment for bleed", irrespective of the time between treatment and the preceding bleed. Bleeds due to surgery/procedure are excluded.
Timepoint [5] 0 0
24 weeks prior to study entry, Baseline, through at least 24 weeks
Secondary outcome [6] 0 0
Intra-Participant Comparison of ABR for All Bleeds on Study Versus Pre-Study in Participants From the Non-Interventional Study Population Previously Treated With FVIII Prophylaxis (NISP) - This is an intra-participant comparison of the annualized bleeding rate (ABR) for all bleeds on study versus pre-study in the NIS population previously treated with FVIII prophylaxis in NIS BH29768. The number of all bleeds over the efficacy period is presented as an ABR that was assessed using a NB regression model, which accounts for different follow-up times, with the participant's number of bleeds as a function of treatment and the time that each participant stays in the study (i.e., length of the efficacy period) included as an offset in the model. The model also includes a repeated statement to account for intra-participant comparison. "All bleeds" comprises both treated and non-treated bleeds. In this definition, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
Timepoint [6] 0 0
24 weeks prior to study entry, Baseline, through at least 24 weeks
Secondary outcome [7] 0 0
Intra-Participant Comparison of ABR for Treated Bleeds on Study Versus Pre-Study in Participants From the NIS Population Previously Treated With Episodic FVIII (NISE) - This is an intra-participant comparison of the annualized bleeding rate (ABR) for treated bleeds on study versus pre-study in the NIS population previously treated with episodic FVIII in NIS BH29768. The number of treated bleeds over the efficacy period is presented as an ABR that was assessed using a NB regression model, which accounts for different follow-up times, with the number of bleeds as a function of treatment and the time that each participant stays in the study (i.e., length of the efficacy period) included as an offset in the model. The model also includes a repeated statement to account for intra-participant comparison. A bleed is considered a "treated bleed" if it is directly followed (i.e., no intervening bleed) by a hemophilia medication reported to be a "treatment for bleed", irrespective of the time between treatment and the preceding bleed. Bleeds due to surgery/procedure are excluded.
Timepoint [7] 0 0
24 weeks prior to study entry, Baseline, through at least 24 weeks
Secondary outcome [8] 0 0
Intra-Participant Comparison of ABR for All Bleeds on Study Versus Pre-Study in Participants From the NIS Population Previously Treated With Episodic FVIII (NISE) - This is an intra-participant comparison of the annualized bleeding rate (ABR) for all bleeds on study versus pre-study in the NIS population previously treated with episodic FVIII in NIS BH29768. The number of all bleeds over the efficacy period is presented as an ABR that was assessed using a NB regression model, which accounts for different follow-up times, with the participant's number of bleeds as a function of treatment and the time that each participant stays in the study (i.e., length of the efficacy period) included as an offset in the model. The model also includes a repeated statement to account for intra-participant comparison. "All bleeds" comprises both treated and non-treated bleeds. In this definition, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
Timepoint [8] 0 0
24 weeks prior to study entry, Baseline, through at least 24 weeks
Secondary outcome [9] 0 0
Hemophilia A Quality of Life (Haem-A-QoL) Questionnaire Physical Health Subscore for Adult Participants (=18 Years of Age) in the Randomized Population at Week 25 - The Haem-A-QoL questionnaire has been developed and used in hemophilia A participants, assessing very specific aspects of dealing with hemophilia. The questionnaire consists of items pertaining to 10 domains: physical health, sports and leisure, school and work, dealing with hemophilia, family planning, feeling, relationships, treatment, view of yourself, and outlook for the future. The total score for each domain ranges from 0 to 100 with lower scores reflective of better quality of life. Physical Health domain score is reported (range 0 to 100, with lower scores reflective of better physical health). The means were derived via an analysis of covariance (ANCOVA) model and have been adjusted for the following co-variates: baseline score, treatment group, and treatment by baseline interaction term.
Timepoint [9] 0 0
Baseline, Week 25
Secondary outcome [10] 0 0
Haem-A-QoL Questionnaire Total Score for Adult Participants (=18 Years of Age) in the Randomized Population at Week 25 - The Haem-A-QoL questionnaire has been developed and used in hemophilia A participants, assessing very specific aspects of dealing with hemophilia. The questionnaire consists of items pertaining to 10 domains: physical health, sports and leisure, school and work, dealing with hemophilia, family planning, feeling, relationships, treatment, view of yourself, and outlook for the future. The total score for each domain ranges from 0 to 100 with lower scores reflective of better quality of life. Haem-A-QoL Total Score is the average of all domain scores and it ranges from 0 to 100, with lower scores reflective of better quality of life. The means were derived via an analysis of covariance (ANCOVA) model and have been adjusted for the following co-variates: baseline score, treatment group, and treatment by baseline interaction term.
Timepoint [10] 0 0
Baseline, Week 25
Secondary outcome [11] 0 0
European Quality of Life 5-Dimensions-5 Levels (EQ-5D-5L) Questionnaire Visual Analogue Scale (VAS) Score in the Randomized Population at Week 25 - EQ-5D-5L is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D-5L includes 2 components: the EQ-5D-5L health state profile (descriptive system) and the EQ-5D-5L VAS. The VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. The means were derived via an analysis of covariance (ANCOVA) model and have been adjusted for the following co-variates: baseline score, treatment group, and treatment by baseline interaction term.
Timepoint [11] 0 0
Baseline, Week 25
Secondary outcome [12] 0 0
EQ-5D-5L Questionnaire Index Utility Score in the Randomized Population at Week 25 - EQ-5D-5L is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D-5L includes 2 components: the EQ-5D-5L health state profile (descriptive system) and the EQ-5D-5L VAS. The EQ-5D-5L health state profile is designed to record the participant's current health state in 5 domains: mobility, selfcare, usual activities, pain/discomfort, and anxiety/depression. Responses from the five domains are used to calculate a single index utility score on a scale of 0 to 1, with higher scores reflective of better quality of life. The means were derived via an analysis of covariance (ANCOVA) model and have been adjusted for the following co-variates: baseline score, treatment group, and treatment by baseline interaction term.
Timepoint [12] 0 0
Baseline, Week 25
Secondary outcome [13] 0 0
Hemophilia-Specific Quality of Life - Short Form (Haemo-QoL-SF) Questionnaire Score in Adolescent Participants (12 to 17 Years of Age) in the Randomized Population at Week 25 - The Haemo-QoL-SF contains 35 items, which cover nine domains considered relevant for the children's health-related quality of life (physical health, feelings, view of yourself, family, friends, other people, sports and school, dealing with hemophilia and treatment). Items are rated with five respective response options: never, seldom, sometimes, often, and always. Haemo-QoL-SF total score range from 0 to 100, where lower scores reflect better health-related quality of life.
Timepoint [13] 0 0
Week 25
Secondary outcome [14] 0 0
Percentage of Participants With at Least One Adverse Event - The percentage of participants experiencing at least one adverse event, including all non-serious and serious adverse events, is reported here. At the clinical cut-off date for primary analysis (15 Sep 2017), data was collected over a period of approximately 1 year.
Timepoint [14] 0 0
From Baseline up to 24 weeks after last dose of study drug (up to 2.5 years)
Secondary outcome [15] 0 0
Percentage of Participants With Grade =3 Adverse Events - The World Health Organization (WHO) toxicity grading scale will be used for assessing adverse event severity. For adverse events that are not specifically listed in the WHO toxicity grading scale, a grade 3 adverse event is defined as: severe, marked limitation in activity, some assistance usually required, medical intervention or therapy required, hospitalization possible; and a grade 4 adverse event is defined as: life-threatening, extreme limitation in activity, significant assistance required, significant medical intervention or therapy required, hospitalization or hospice care probable. At the clinical cut-off date for primary analysis (15 Sep 2017), data was collected over a period of approximately 1 year.
Timepoint [15] 0 0
From Baseline up to 24 weeks after last dose of study drug (up to 2.5 years)
Secondary outcome [16] 0 0
Percentage of Participants With Adverse Events Leading to Withdrawal From Treatment - At the clinical cut-off date for primary analysis (15 Sep 2017), data was collected over a period of approximately 1 year.
Timepoint [16] 0 0
From Baseline up to 24 weeks after last dose of study drug (up to 2.5 years)
Secondary outcome [17] 0 0
Percentage of Participants With Adverse Events of Changes From Baseline in Vital Signs - The percentage of participants with adverse events of changes from baseline in vital signs is reported here. Vital signs measurements consisted of heart and respiratory rate, temperature, and systolic and diastolic blood pressures, with an abnormal vital sign value being outside of the normal range. An abnormal vital sign result is reported as an adverse event if it meets any of the following criteria: is accompanied by clinical symptoms; results in a change in study treatment (e.g., dosage modification, treatment interruption or discontinuation); results in a medical intervention or a change in concomitant therapy; or is clinically significant in the investigator's judgment. At the clinical cut-off date for primary analysis (15 Sep 2017), data was collected over a period of approximately 1 year.
Timepoint [17] 0 0
From Baseline up to 24 weeks after last dose of study drug (up to 2.5 years)
Secondary outcome [18] 0 0
Percentage of Participants With Adverse Events of Changes From Baseline in Physical Examination Findings - Post-baseline physical examination abnormalities that were not present at baseline or worsened were reported as adverse events. At the clinical cut-off date for primary analysis (15 Sep 2017), data was collected over a period of approximately 1 year.
Timepoint [18] 0 0
From Baseline up to 24 weeks after last dose of study drug (up to 2.5 years)
Secondary outcome [19] 0 0
Percentage of Participants With Adverse Events of Abnormal Laboratory Values - The percentage of participants with adverse events of abnormal laboratory values is reported here. An abnormal laboratory value is defined as a laboratory test result outside of the normal range for hematology or serum chemistries. It is reported as an adverse event if it meets any of the following criteria: is accompanied by clinical symptoms; results in a change in study treatment (e.g., dosage modification, treatment interruption or discontinuation); results in a medical intervention or a change in concomitant therapy; or is clinically significant in the investigator's judgment. At the clinical cut-off date for primary analysis (15 Sep 2017), data was collected over a period of approximately 1 year.
Timepoint [19] 0 0
From Baseline up to 24 weeks after last dose of study drug (up to 2.5 years)
Secondary outcome [20] 0 0
Percentage of Participants With Local Injection-Site Reactions - Local adverse events that occurred within 24 hours after study drug administration and, in the investigator's opinion, were judged to be related to study drug injection, were captured as an "injection-site reaction" on the Adverse Event electronic Case Report Form (eCRF). An injection-related reaction that was localized was marked as a "local injection-site reaction." At the clinical cut-off date for primary analysis (15 Sep 2017), data was collected over a period of approximately 1 year.
Timepoint [20] 0 0
From Baseline up to 24 weeks after last dose of study drug (up to 2.5 years)
Secondary outcome [21] 0 0
Percentage of Participants With Thromboembolic Events - At the clinical cut-off date for primary analysis (15 Sep 2017), data was collected over a period of approximately 1 year.
Timepoint [21] 0 0
From Baseline up to 24 weeks after last dose of study drug (up to 2.5 years)
Secondary outcome [22] 0 0
Percentage of Participants With Thrombotic Microangiopathy - At the clinical cut-off date for primary analysis (15 Sep 2017), data was collected over a period of approximately 1 year.
Timepoint [22] 0 0
From Baseline up to 24 weeks after last dose of study drug (up to 2.5 years)
Secondary outcome [23] 0 0
Percentage of Participants With Systemic Hypersensitivity, Anaphylaxis, or Anaphylactoid Reactions - At the clinical cut-off date for primary analysis (15 Sep 2017), data was collected over a period of approximately 1 year.
Timepoint [23] 0 0
From Baseline up to 24 weeks after last dose of study drug (up to 2.5 years)
Secondary outcome [24] 0 0
Percentage of Participants With Anti-Emicizumab Antibodies - A validated ELISA method was used to analyze the levels of anti-emicizumab antibodies in plasma. A sample was considered positive for anti-emicizumab antibodies if the test result reached or exceeded a pre-determined threshold. At the clinical cut-off date for primary analysis (15 Sep 2017), data was collected over a period of approximately 1 year.
Timepoint [24] 0 0
From Baseline up to 24 weeks after last dose of study drug (up to 2.5 years)
Secondary outcome [25] 0 0
Percentage of Participants With De Novo Development of Factor VIII (FVIII) Inhibitors - Levels of anti-FVIII antibodies (inhibitors) were analyzed using a validated FVIII activity assay. A participant was considered to have developed de novo FVIII inhibitors if the inhibitor levels detected in a post-baseline sample reached or exceeded a pre-determined threshold. At the clinical cut-off date for primary analysis (15 Sep 2017), data was collected over a period of approximately 1 year.
Timepoint [25] 0 0
From Baseline up to 24 weeks after last dose of study drug (up to 2.5 years)
Secondary outcome [26] 0 0
Trough Plasma Concentration (Ctrough) of Emicizumab - Plasma concentrations of emicizumab were analyzed using a validated Enzyme Linked Immunosorbent Assay (ELISA). The lower limit of quantitation was 0.1 micrograms per milliliter (µg/mL). At the clinical cut-off date for primary analysis (15 Sep 2017), data was collected over a period of approximately 1 year.
Timepoint [26] 0 0
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)

Eligibility
Key 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
Minimum age
12 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA,VIC,WA
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 0 0
The Alfred Hospital, Melbourne; Thrombosis and Haemostasis Unit - Melbourne
Recruitment hospital [3] 0 0
The Perth Blood Institute - Nedlands
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
3004 - Melbourne
Recruitment postcode(s) [3] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
District of Columbia
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
Washington
Country [9] 0 0
Costa Rica
State/province [9] 0 0
San Jose
Country [10] 0 0
France
State/province [10] 0 0
Bron
Country [11] 0 0
France
State/province [11] 0 0
Le Kremlin Bicetre
Country [12] 0 0
France
State/province [12] 0 0
Paris
Country [13] 0 0
Germany
State/province [13] 0 0
Bonn
Country [14] 0 0
Ireland
State/province [14] 0 0
Dublin
Country [15] 0 0
Italy
State/province [15] 0 0
Lombardia
Country [16] 0 0
Italy
State/province [16] 0 0
Toscana
Country [17] 0 0
Japan
State/province [17] 0 0
Aichi
Country [18] 0 0
Japan
State/province [18] 0 0
Hokkaido
Country [19] 0 0
Japan
State/province [19] 0 0
Kanagawa
Country [20] 0 0
Japan
State/province [20] 0 0
Miyagi
Country [21] 0 0
Japan
State/province [21] 0 0
Nara
Country [22] 0 0
Japan
State/province [22] 0 0
Osaka
Country [23] 0 0
Japan
State/province [23] 0 0
Saitama
Country [24] 0 0
Japan
State/province [24] 0 0
Tokyo
Country [25] 0 0
Korea, Republic of
State/province [25] 0 0
Seoul
Country [26] 0 0
Poland
State/province [26] 0 0
Gdansk
Country [27] 0 0
Poland
State/province [27] 0 0
Lublin
Country [28] 0 0
Poland
State/province [28] 0 0
Poznan
Country [29] 0 0
Poland
State/province [29] 0 0
Warsaw
Country [30] 0 0
South Africa
State/province [30] 0 0
Johannesburg
Country [31] 0 0
Spain
State/province [31] 0 0
Madrid
Country [32] 0 0
Spain
State/province [32] 0 0
Sevilla
Country [33] 0 0
Taiwan
State/province [33] 0 0
Chang Hua
Country [34] 0 0
Taiwan
State/province [34] 0 0
Taichung
Country [35] 0 0
Taiwan
State/province [35] 0 0
Taipei
Country [36] 0 0
United Kingdom
State/province [36] 0 0
Cardiff
Country [37] 0 0
United Kingdom
State/province [37] 0 0
London
Country [38] 0 0
United Kingdom
State/province [38] 0 0
Manchester
Country [39] 0 0
United Kingdom
State/province [39] 0 0
Oxford

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Hoffmann-La Roche
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
Chugai Pharmaceutical
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT02847637
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Trials
Address 0 0
Hoffmann-La Roche
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications