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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03191799




Registration number
NCT03191799
Ethics application status
Date submitted
15/06/2017
Date registered
19/06/2017
Date last updated
11/06/2021

Titles & IDs
Public title
A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors
Scientific title
A Single-Arm, Multicenter Phase IIIB Clinical Trial to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors
Secondary ID [1] 0 0
2016-004366-25
Secondary ID [2] 0 0
MO39129
Universal Trial Number (UTN)
Trial acronym
STASEY
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: 1.5 mg/kg Emicizumab QW - Participants will receive initial weekly doses of prophylactic emicizumab subcutaneously for 4 weeks, followed by maintenance doses consisting of half the initial dose, administered subcutaneously for the remainder of the 2-year treatment period


Treatment: Drugs: Emicizumab
Initial dosing will be 3 mg/kg/week subcutaneously for 4 weeks; Maintenance dosing will follow at 1.5 mg/kg/week subcutaneously for the remainder of the 2-year treatment period
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Overall Summary of the Number of Participants With Adverse Events, Severity Assessed According to the World Health Organization (WHO) Toxicity Grading Scale
Timepoint [1] 0 0
From Baseline until study completion (up to 2 years)
Primary outcome [2] 0 0
Adverse Events (AEs) Rates Per 100 Patient-Years for All-Grade AEs, Serious AEs, and Grade =3 AEs
Timepoint [2] 0 0
From Baseline until study completion (up to 2 years)
Primary outcome [3] 0 0
Number of Participants by Hematology and Biochemistry Laboratory Parameter Test Results as Shifts From the WHO Toxicity Grade at Baseline to the Worst WHO Toxicity Grade Post-Baseline
Timepoint [3] 0 0
Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Primary outcome [4] 0 0
Change From Baseline in Body Temperature at Specified Timepoints
Timepoint [4] 0 0
Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Primary outcome [5] 0 0
Change From Baseline in Systolic Blood Pressure at Specified Timepoints
Timepoint [5] 0 0
Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Primary outcome [6] 0 0
Change From Baseline in Diastolic Blood Pressure at Specified Timepoints
Timepoint [6] 0 0
Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Primary outcome [7] 0 0
Change From Baseline in Pulse Rate at Specified Timepoints
Timepoint [7] 0 0
Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Primary outcome [8] 0 0
Change From Baseline in Respiratory Rate at Specified Timepoints
Timepoint [8] 0 0
Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Primary outcome [9] 0 0
Change From Baseline in Body Weight at Specified Timepoints
Timepoint [9] 0 0
Baseline, Weeks 2, 3, and 5; Months 3, 6, 9, 12, and 18; and at Early Termination/Study Completion (up to 24 months)
Secondary outcome [1] 0 0
Model-Based Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds
Timepoint [1] 0 0
From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Secondary outcome [2] 0 0
Mean Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds
Timepoint [2] 0 0
From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Secondary outcome [3] 0 0
Median Calculated Annualized Bleed Rates (ABR) for Treated Bleeds, All Bleeds, Treated Joint Bleeds, Treated Target Joint Bleeds, and Treated Spontaneous Bleeds
Timepoint [3] 0 0
From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Secondary outcome [4] 0 0
Percentage of Participants by the Categorized Number of Bleeds for Treated Bleeds
Timepoint [4] 0 0
From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Secondary outcome [5] 0 0
Percentage of Participants by the Categorized Number of Bleeds for All Bleeds
Timepoint [5] 0 0
From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Secondary outcome [6] 0 0
Percentage of Participants by the Categorized Number of Bleeds for Treated Spontaneous Bleeds
Timepoint [6] 0 0
From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Secondary outcome [7] 0 0
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for Treated Bleeds
Timepoint [7] 0 0
From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Secondary outcome [8] 0 0
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for All Bleeds
Timepoint [8] 0 0
From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Secondary outcome [9] 0 0
Percentage of Participants by the Categorized Calculated Annualized Bleed Rates (ABR) for Treated Spontaneous Bleeds
Timepoint [9] 0 0
From first dose of emicizumab until dose up-titration or withdrawal from treatment (median [min-max] efficacy period: 103.14 [1.1-108.3] weeks)
Secondary outcome [10] 0 0
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints, Adult Participants
Timepoint [10] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [11] 0 0
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Total Score at Specified Timepoints
Timepoint [11] 0 0
At 3, 6, 12, and 18 months
Secondary outcome [12] 0 0
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints, Adult Participants
Timepoint [12] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [13] 0 0
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Physical Health Domain Score at Specified Timepoints
Timepoint [13] 0 0
At 3, 6, 12, and 18 months
Secondary outcome [14] 0 0
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Treatment Domain Score at Specified Timepoints, Adult Participants
Timepoint [14] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [15] 0 0
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Work and School Domain Score at Specified Timepoints, Adult Participants
Timepoint [15] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [16] 0 0
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adult Participants
Timepoint [16] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [17] 0 0
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Feelings Domain Score at Specified Timepoints, Adult Participants
Timepoint [17] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [18] 0 0
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Family Planning Domain Score at Specified Timepoints, Adult Participants
Timepoint [18] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [19] 0 0
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Future Domain Score at Specified Timepoints, Adult Participants
Timepoint [19] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [20] 0 0
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Partnership and Sexuality Domain Score at Specified Timepoints, Adult Participants
Timepoint [20] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [21] 0 0
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire Sports and Leisure Domain Score at Specified Timepoints, Adult Participants
Timepoint [21] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [22] 0 0
Change From Baseline in the Hemophilia Adult Quality of Life (Haem-A-QoL) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adult Participants
Timepoint [22] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [23] 0 0
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints, Adolescent Participants
Timepoint [23] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [24] 0 0
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Total Score at Specified Timepoints
Timepoint [24] 0 0
At 3, 6, 12, and 18 months
Secondary outcome [25] 0 0
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints, Adolescent Participants
Timepoint [25] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [26] 0 0
Percentage of Participants With an Improvement From Baseline Greater Than the Responder Threshold for the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Physical Health Domain Score at Specified Timepoints
Timepoint [26] 0 0
At 3, 6, 12, and 18 months
Secondary outcome [27] 0 0
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Treatment Domain Score at Specified Timepoints, Adolescent Participants
Timepoint [27] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [28] 0 0
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Sports and School Domain Score at Specified Timepoints, Adolescent Participants
Timepoint [28] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [29] 0 0
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Dealing With Hemophilia Domain Score at Specified Timepoints, Adolescent Participants
Timepoint [29] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [30] 0 0
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Feelings Domain Score at Specified Timepoints, Adolescent Participants
Timepoint [30] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [31] 0 0
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Family Domain Score at Specified Timepoints, Adolescent Participants
Timepoint [31] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [32] 0 0
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Friends Domain Score at Specified Timepoints, Adolescent Participants
Timepoint [32] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [33] 0 0
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Other People Domain Score at Specified Timepoints, Adolescent Participants
Timepoint [33] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [34] 0 0
Change From Baseline in the Hemophilia Quality of Life Short Form (Haemo-QoL-SF) Questionnaire View of Yourself Domain Score at Specified Timepoints, Adolescent Participants
Timepoint [34] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [35] 0 0
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Index Utility Score at Specified Timepoints
Timepoint [35] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [36] 0 0
Change From Baseline in the EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L) Quality-of-Life Visual Analogue Scale (VAS) Score at Specified Timepoints
Timepoint [36] 0 0
Baseline (Week 1), 3, 6, 12, and 18 months, and at Early Termination or Study Completion (up to 24 months)
Secondary outcome [37] 0 0
Percentage of Participants Who Preferred the New Emicizumab Treatment or the Old Hemophilia Treatment, or Without Treatment Preference, as Assessed by the EmiPref Questionnaire
Timepoint [37] 0 0
Month 3
Secondary outcome [38] 0 0
Number and Percentage of Participants With Anti-Drug Antibodies (ADAs) Against Emicizumab at Anytime Post-Baseline
Timepoint [38] 0 0
Baseline (Week 1), Week 5, and at 3, 6, 9, 12, and 18 months, and at early termination/study completion (up to 24 months)
Secondary outcome [39] 0 0
Mean Trough Plasma Concentrations of Emicizumab at Specified Timepoints
Timepoint [39] 0 0
Pre-dose at Weeks 2, 3, and 5, Months 3, 6, 12, and 18, and at treatment discontinuation (up to 2 years)

Eligibility
Key inclusion criteria
* As per investigator's judgement, a willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures, including the patient-reported outcome (PRO) questionnaires and bleed diaries through the use of an electronic device or paper
* Aged 12 years or older at the time of informed consent
* Diagnosis of congenital hemophilia A with persistent inhibitors against FVIII
* Documented treatment with bypassing agents or FVIII concentrates in the last 6 months (on-demand or prophylaxis). Prophylaxis needs to be discontinued the latest by a day before starting emicizumab
* Adequate hematologic, hepatic, and renal function
* For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use a highly effective contraceptive method with a failure rate of <1% per year during the treatment period and for at least five elimination half-lives (24 weeks) after the last dose of emicizumab
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Inherited or acquired bleeding disorder other than hemophilia A
* Ongoing (or plan to receive during the study) immune tolerance induction (ITI) therapy (prophylaxis regimens with FVIII and/or bypassing agents must be discontinued prior to enrollment). Patients receiving ITI therapy will be eligible following the completion of a 72-hour washout period prior to the first emicizumab administration
* History of illicit drug or alcohol abuse within 12 months prior to screening, as per the investigator's judgment
* High risk for thrombotic microangiopathy (TMA) (e.g., have a previous medical or family history of TMA), as per the investigator's judgment
* Previous (in the past 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which antithrombotic treatment is not currently ongoing) or current signs of thromboembolic disease
* Other conditions (e.g., certain autoimmune diseases) that may increase the risk of bleeding or thrombosis
* History of clinically significant hypersensitivity reaction associated with monoclonal antibody therapies or components of the emicizumab injection
* Known human immunodeficiency virus (HIV) infection with CD4 count <200 cells/µL within 6 months prior to screening
* Use of systemic immunomodulators (e.g., interferon or rituximab) at enrollment or planned use during the study, with the exception of antiretroviral therapy
* Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study or that would, in the opinion of the investigator or Sponsor, preclude the patient's safe participation in and completion of the study or interpretation of the study results
* Receipt of: Emicizumab in a prior investigational study; An investigational drug to treat or reduce the risk of hemophilic bleeds within five half-lives of last drug administration; A non-hemophilia-related investigational drug within last 30 days or five half-lives, whichever is shorter; or, Any concurrent investigational drug.
* Pregnancy or lactation, or intent to become pregnant during the study
* Positive serum pregnancy test result within 7 days prior to initiation of emicizumab (females only)

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
5/09/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
19/11/2020
Sample size
Target
Accrual to date
Final
195
Recruitment in Australia
Recruitment state(s)
NSW,VIC,WA
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital; Haematology - Camperdown
Recruitment hospital [2] 0 0
Alfred Hospital - Melbourne
Recruitment hospital [3] 0 0
Fiona Stanley Hospital - Murdoch
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
3004 - Melbourne
Recruitment postcode(s) [3] 0 0
6150 - Murdoch
Recruitment outside Australia
Country [1] 0 0
Belgium
State/province [1] 0 0
Leuven
Country [2] 0 0
Brazil
State/province [2] 0 0
PR
Country [3] 0 0
Brazil
State/province [3] 0 0
RJ
Country [4] 0 0
Brazil
State/province [4] 0 0
SP
Country [5] 0 0
Canada
State/province [5] 0 0
Alberta
Country [6] 0 0
Canada
State/province [6] 0 0
Manitoba
Country [7] 0 0
Canada
State/province [7] 0 0
Ontario
Country [8] 0 0
Colombia
State/province [8] 0 0
Localidad Puente Aranda
Country [9] 0 0
Colombia
State/province [9] 0 0
Medellin
Country [10] 0 0
Finland
State/province [10] 0 0
Helsinki
Country [11] 0 0
Germany
State/province [11] 0 0
Berlin
Country [12] 0 0
Germany
State/province [12] 0 0
Bonn
Country [13] 0 0
Germany
State/province [13] 0 0
Frankfurt/M.
Country [14] 0 0
Germany
State/province [14] 0 0
Leipzig
Country [15] 0 0
Germany
State/province [15] 0 0
Mörfelden-Walldorf
Country [16] 0 0
Germany
State/province [16] 0 0
München
Country [17] 0 0
Guatemala
State/province [17] 0 0
Guatemala
Country [18] 0 0
Hungary
State/province [18] 0 0
Budapest
Country [19] 0 0
Hungary
State/province [19] 0 0
Gyor
Country [20] 0 0
Hungary
State/province [20] 0 0
Pecs
Country [21] 0 0
India
State/province [21] 0 0
Karnataka
Country [22] 0 0
India
State/province [22] 0 0
Maharashtra
Country [23] 0 0
India
State/province [23] 0 0
WEST Bengal
Country [24] 0 0
India
State/province [24] 0 0
Pune
Country [25] 0 0
Israel
State/province [25] 0 0
Tel Hashomer
Country [26] 0 0
Italy
State/province [26] 0 0
Campania
Country [27] 0 0
Italy
State/province [27] 0 0
Emilia-Romagna
Country [28] 0 0
Italy
State/province [28] 0 0
Lazio
Country [29] 0 0
Italy
State/province [29] 0 0
Lombardia
Country [30] 0 0
Italy
State/province [30] 0 0
Piemonte
Country [31] 0 0
Italy
State/province [31] 0 0
Sicilia
Country [32] 0 0
Italy
State/province [32] 0 0
Toscana
Country [33] 0 0
Italy
State/province [33] 0 0
Veneto
Country [34] 0 0
Mexico
State/province [34] 0 0
Mexico CITY (federal District)
Country [35] 0 0
Mexico
State/province [35] 0 0
Mexico City
Country [36] 0 0
Netherlands
State/province [36] 0 0
Rotterdam
Country [37] 0 0
Netherlands
State/province [37] 0 0
Utrecht
Country [38] 0 0
Panama
State/province [38] 0 0
Panama City
Country [39] 0 0
Poland
State/province [39] 0 0
Warsaw
Country [40] 0 0
Portugal
State/province [40] 0 0
Coimbra
Country [41] 0 0
Portugal
State/province [41] 0 0
Lisboa
Country [42] 0 0
Portugal
State/province [42] 0 0
Porto
Country [43] 0 0
Romania
State/province [43] 0 0
Timisoara
Country [44] 0 0
Russian Federation
State/province [44] 0 0
Altaj
Country [45] 0 0
Russian Federation
State/province [45] 0 0
Moscow
Country [46] 0 0
Russian Federation
State/province [46] 0 0
Saint Petersburg
Country [47] 0 0
Russian Federation
State/province [47] 0 0
Samara
Country [48] 0 0
Saudi Arabia
State/province [48] 0 0
Riyadh
Country [49] 0 0
Spain
State/province [49] 0 0
Barcelona
Country [50] 0 0
Spain
State/province [50] 0 0
Madrid
Country [51] 0 0
Spain
State/province [51] 0 0
Malaga
Country [52] 0 0
Spain
State/province [52] 0 0
Sevilla
Country [53] 0 0
Spain
State/province [53] 0 0
Valencia
Country [54] 0 0
Sweden
State/province [54] 0 0
Göteborg
Country [55] 0 0
Sweden
State/province [55] 0 0
Solna
Country [56] 0 0
Switzerland
State/province [56] 0 0
Bern
Country [57] 0 0
United Kingdom
State/province [57] 0 0
Birmingham
Country [58] 0 0
United Kingdom
State/province [58] 0 0
London
Country [59] 0 0
United Kingdom
State/province [59] 0 0
Newcastle upon Tyne
Country [60] 0 0
United Kingdom
State/province [60] 0 0
Sheffield

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Hoffmann-La Roche
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a phase IIIb, single arm, open-label, multi-center study to evaluate the safety and tolerability of emicizumab in participants with congenital hemophilia A who have documented inhibitors against Factor VIII (FVIII) at enrollment. Approximately 200 participants, aged 12 or older, will be enrolled in this study and are expected to be enrolled at approximately 85 sites globally. Participants will receive an initial weekly dose of prophylactic emicizumab subcutaneously for 4 weeks, followed by a weekly maintenance dose subcutaneously for the remainder of the 2-year treatment period.
Trial website
https://clinicaltrials.gov/study/NCT03191799
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

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03191799