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NCT Number: NCT02476942

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

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.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Sex eligibility

All sexes

Study type

Observational

Primary location

Royal Prince Alfred Hospital; Haematology, Camperdown, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

Bypassing Agents

Drug

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.

FVIII Replacement

Drug

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 outcomes

  1. Number of Bleeds

    Time frame: Approximately 6 months (from Baseline until study completion)

Secondary outcomes

  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)

Sponsors and collaborators

Lead sponsor

Hoffmann-La Roche

Industry

Registry information

Official study 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

Important dates

Study start
2015
Primary completion
2017
Study completion
2017
First posted
Jun 22, 2015
Registry last updated
May 30, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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