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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02476942




Registration number
NCT02476942
Ethics application status
Date submitted
3/06/2015
Date registered
22/06/2015
Date last updated
30/05/2017

Titles & IDs
Public title
A Prospective Study to Collect High-Quality Documentation of Bleeds, Health-Related Quality of Life (HRQoL), and Safety Outcomes in Patients With Hemophilia A Treated With Standard-of-Care Treatment
Scientific title
A Multicenter, Non-Interventional Study Evaluating Bleeding Incidence, Health-Related Quality of Life, and Safety in Patients With Hemophilia A Under Standard-of-Care Treatment
Secondary ID [1] 0 0
BH29768
Universal Trial Number (UTN)
Trial acronym
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
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Treatment: Drugs - Bypassing Agents
Treatment: Drugs - FVIII Replacement

Cohort A: Adults and Adolescents with FVIII Inhibitors - Adults and adolescents with hemophilia A of any severity with the presence of FVIII inhibitors will be observed.

Cohort B: Children with FVIII Inhibitors - Children with hemophilia A of any severity with the presence of FVIII inhibitors will be observed.

Cohort C: Adults and Adolescents without FVIII Inhibitors - Adults and adolescents with severe hemophilia A without the presence of FVIII inhibitors will be observed.


Treatment: Drugs: Bypassing Agents
Episodic or prophylactic treatment with the use of bypassing agents must be documented for at least the last 6 months prior to the study. During the study, treatment for bleeds will be documented. The choice of coagulation product is at the discretion of the investigator according to local practice standards in this non-interventional study and there are no specific protocol-defined interventions.

Treatment: Drugs: FVIII Replacement
Episodic or prophylactic treatment with the use of FVIII replacement must be documented for at least the last 6 months prior to the study. During the study, treatment for bleeds will be documented. The choice of coagulation product is at the discretion of the investigator according to local practice standards in this non-interventional study and there are no specific protocol-defined interventions.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Bleeds
Timepoint [1] 0 0
Approximately 6 months (from Baseline until study completion)
Secondary outcome [1] 0 0
European Quality of Life-5 Dimensions (EQ-5D-5L) Questionnaire Score Among Adult and Adolescent Patients
Timepoint [1] 0 0
Approximately 6 months (every 4 weeks from Baseline until study completion and on days that bleeds are reported)
Secondary outcome [2] 0 0
Hemophilia A-Specific Quality of Life (Haem-A-QoL) Questionnaire Score Among Adult Patients
Timepoint [2] 0 0
Approximately 6 months (every 4 weeks from Baseline until study completion)
Secondary outcome [3] 0 0
Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Score
Timepoint [3] 0 0
Approximately 6 months (every 4 weeks from Baseline until study completion)

Eligibility
Key inclusion criteria
* Cohort A: Patients greater than or equal to (>/=) 12 years of age at time of informed consent
* Cohort A: Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (that is, >/= 5 Bethesda units [BU])
* Cohort B: Pediatric patients less than (<) 12 years of age
* Cohort B: Diagnosis of congenital hemophilia A of any severity and documented history of high-titer inhibitor (that is, >/=5 BU)
* Cohort C: Patients >/=12 years of age
* Cohort C: Diagnosis of congenital hemophilia A and FVIII activity <1 percent (%)
* Cohort C: No prior history of a positive inhibitor against FVIII
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Prior RO5534262 (not applicable if patient agrees that prior RO5534262 will preclude participation in a future investigational RO5534262 study)
* Bleeding disorder other than congenital hemophilia A
* Ongoing (or planned during the study) immune tolerance induction therapy with FVIII or FVIII prophylaxis if currently/previously exposed to an inhibitor
* Previous or concomitant thromboembolic disease
* Known human immunodeficiency virus (HIV) infection with cluster of differentiation (CD) 4 count <200 cells per microliter (cells/mcL)

Study design
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
26/05/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
31/03/2017
Sample size
Target
Accrual to date
Final
221
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital; Haematology - Camperdown
Recruitment hospital [2] 0 0
The Alfred Hospital, Melbourne; Thrombosis and Haemostasis Unit - Melbourne
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
3004 - Melbourne
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
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
District of Columbia
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
Louisiana
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
Oregon
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
Washington
Country [11] 0 0
China
State/province [11] 0 0
Beijing
Country [12] 0 0
China
State/province [12] 0 0
Guangzhou
Country [13] 0 0
China
State/province [13] 0 0
Tianjin
Country [14] 0 0
Costa Rica
State/province [14] 0 0
San Jose
Country [15] 0 0
Germany
State/province [15] 0 0
Bonn
Country [16] 0 0
Italy
State/province [16] 0 0
Lombardia
Country [17] 0 0
Italy
State/province [17] 0 0
Toscana
Country [18] 0 0
Japan
State/province [18] 0 0
Aichi
Country [19] 0 0
Japan
State/province [19] 0 0
Hyogo
Country [20] 0 0
Japan
State/province [20] 0 0
Kanagawa
Country [21] 0 0
Japan
State/province [21] 0 0
Kitakyushu-shi
Country [22] 0 0
Japan
State/province [22] 0 0
Nara
Country [23] 0 0
Japan
State/province [23] 0 0
Tokyo
Country [24] 0 0
Korea, Republic of
State/province [24] 0 0
Seoul
Country [25] 0 0
Poland
State/province [25] 0 0
Gdansk
Country [26] 0 0
Poland
State/province [26] 0 0
Lublin
Country [27] 0 0
Poland
State/province [27] 0 0
Poznan
Country [28] 0 0
Poland
State/province [28] 0 0
Warsaw
Country [29] 0 0
South Africa
State/province [29] 0 0
Johannesburg
Country [30] 0 0
Spain
State/province [30] 0 0
Madrid
Country [31] 0 0
Spain
State/province [31] 0 0
Sevilla
Country [32] 0 0
Spain
State/province [32] 0 0
Valencia
Country [33] 0 0
Taiwan
State/province [33] 0 0
Taipei

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

Ethics approval
Ethics application status

Summary
Brief summary
This non-interventional study will prospectively collect detailed, high-quality documentation of bleeds, HRQoL, and safety in patients with hemophilia A with or without FVIII inhibitors treated according to local routine clinical practice (receiving FVIII replacement or bypassing agents as either episodic or prophylactic treatment). Actual patients will be enrolled from routine clinical practice in this observational study.
Trial website
https://clinicaltrials.gov/study/NCT02476942
Trial related presentations / publications
Oldenburg J, Shima M, Kruse-Jarres R, Santagostino E, Mahlangu J, Lehle M, Selak Bienz N, Chebon S, Asikanius E, Trask P, Mancuso ME, Jimenez-Yuste V, von Mackensen S, Levy GG. Outcomes in children with hemophilia A with inhibitors: Results from a noninterventional study. Pediatr Blood Cancer. 2020 Oct;67(10):e28474. doi: 10.1002/pbc.28474. Epub 2020 Aug 9.
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/NCT02476942