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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

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.
 
ClinicalTrials.gov Identifier: NCT02476942
Recruitment Status : Completed
First Posted : June 22, 2015
Last Update Posted : May 30, 2017
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

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.

Condition or disease Intervention/treatment
Hemophilia A Drug: Bypassing Agents Drug: FVIII Replacement

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Study Type : Observational
Actual Enrollment : 221 participants
Observational Model: Cohort
Time Perspective: Cross-Sectional
Official 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
Actual Study Start Date : May 26, 2015
Actual Primary Completion Date : March 31, 2017
Actual Study Completion Date : March 31, 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Bleeding Hemophilia

Group/Cohort Intervention/treatment
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.
Drug: 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.

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.
Drug: 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.




Primary Outcome Measures :
  1. Number of Bleeds [ Time Frame: Approximately 6 months (from Baseline until study completion) ]

Secondary Outcome Measures :
  1. European Quality of Life-5 Dimensions (EQ-5D-5L) Questionnaire Score Among Adult and Adolescent Patients [ Time Frame: Approximately 6 months (every 4 weeks from Baseline until study completion and on days that bleeds are reported) ]
  2. Hemophilia A-Specific Quality of Life (Haem-A-QoL) Questionnaire Score Among Adult Patients [ Time Frame: Approximately 6 months (every 4 weeks from Baseline until study completion) ]
  3. Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Score [ Time Frame: Approximately 6 months (every 4 weeks from Baseline until study completion) ]


Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with hemophilia A with or without inhibitors against FVIII under standard-of-care treatment are to be enrolled.
Criteria

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

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)

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): NCT02476942


Locations
Show Show 35 study locations
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
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Study Director: Clinical Trials Hoffmann-La Roche
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Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT02476942    
Other Study ID Numbers: BH29768
First Posted: June 22, 2015    Key Record Dates
Last Update Posted: May 30, 2017
Last Verified: May 2017
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn