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

Completed

• Conditions: Hemophilia A
• Interventions: Drug: FVIII Replacement; Drug: Bypassing Agents

• Registration Number: NCT02476942
• Lead Sponsor: 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2015-05-26
• Study First Submitted: 2015-06-03
• Study First Submitted QC: 2015-06-19
• Study First Posted: 2015-06-22
• Primary Completion: 2017-03-31
• Study Completion: 2017-03-31
• Status Verified: 2017-05
• Last Update Submitted: 2017-05-26
• Last Update Posted: 2017-05-30

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 221

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)

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Cohort C: Adults and Adolescents without FVIII Inhibitors	FVIII Replacement	Adults and adolescents with severe hemophilia A without the presence of FVIII inhibitors will be observed.
Cohort A: Adults and Adolescents with FVIII Inhibitors	Bypassing Agents	Adults and adolescents with hemophilia A of any severity with the presence of FVIII inhibitors will be observed.

Primary Outcome Measures

Name	Time	Method
Number of Bleeds	Approximately 6 months (from Baseline until study completion)

Secondary Outcome Measures

Name	Time	Method
European Quality of Life-5 Dimensions (EQ-5D-5L) Questionnaire Score Among Adult and Adolescent Patients	Approximately 6 months (every 4 weeks from Baseline until study completion and on days that bleeds are reported)
Hemophilia A-Specific Quality of Life (Haem-A-QoL) Questionnaire Score Among Adult Patients	Approximately 6 months (every 4 weeks from Baseline until study completion)
Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) Questionnaire Score	Approximately 6 months (every 4 weeks from Baseline until study completion)

Trial Locations

Locations (35)

Santa Monica Oncology Center; 🇺🇸 Santa Monica, California, United States

University of Colorado Denver, Children's Hospital; 🇺🇸 Aurora, Colorado, United States

Georgetown Uni Medical Center; Lombardi Cancer Center; 🇺🇸 Washington, D.C., District of Columbia, United States

Winship Cancer Institute; 🇺🇸 Atlanta, Georgia, United States

Tulane Uni Health Sciences Center; 🇺🇸 New Orleans, Louisiana, United States

Children's Hospital of Michigan; Pediatrics; 🇺🇸 Detroit, Michigan, United States

Cornell Univ Medical College; Hematology-Oncolog; 🇺🇸 New York, New York, United States

Oregon Health & Science Uni ; Dept of Pediatrics; 🇺🇸 Portland, Oregon, United States

University of Pittsburgh; 🇺🇸 Pittsburgh, Pennsylvania, United States

Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia; 🇺🇸 Seattle, Washington, United States

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