Factor Product Utilization and Health Outcomes in Patients With Hemophilia

NCT02796222 | Completed

• 1 other identifier: CAN-FAB-15-10911
• Study Type: observational
• Target: 61
• Locations: 1 country
• Sites: 1

Brief Summary

Recombinant factor VIII Fc (rFVIIIFc) and recombinant factor IX Fc (rFIXFc) are extended half-life coagulation factors approved by Health Canada in 2014 for the treatment of severe hemophilia A and B, respectively. The objectives of this observational study is to describe the change in annual factor consumption, clinical and patient-reported outcomes for patients who switch from recombinant factor VIII (rFVIII) and recombinant factor IX (rFIX) to rFVIIIFc/ rFIXFc in Canada, and to explore clinicians' and patients' reasons for switching or not switching.

Conditions

Hemophilia A, Congenital; Hemophilia B, Congenital

Keywords

Hemophilia; Recombinant factor VIII; Recombinant factor VIII Fc; Recombinant factor IX; Patient-reported outcomes; Recombinant factor IX Fc

Outcome Measures

Primary Outcomes (1)

• Change in the total annualized factor consumption (in units/kilogram/year)

  From baseline to 24-month period on rFVIIIFc or rFIXFc

Secondary Outcomes (22)

• Change in health-related quality of life (HRQoL) SF-36

  From baseline to 3 months, 12 months and 24 months

• Change in health-related quality of life (HRQoL) Haem-A-Qol

  From baseline to 3 months, 12 months and 24 months

• Change in health-related quality of life (HRQoL) CHO-KLAT

  From baseline to 3 months, 12 months and 24 months

• Change in the Work Productivity and Impairment Questionnaire (WPAI+CIQ: HS) score

  From baseline to 3 months, 12 months and 24 months

• Change in chronic pain Numeric Rating Scale (0-10)

  From baseline to 3 months, 12 months and 24 months

Study Arms (4)

Hemophilia A patients on rFVIIIFc

Patients with hemophilia A who switch from on-demand or prophylactic treatment with rFVIII to rFVIIIFc

Hemophilia A patients on rFVIII

Patients with hemophilia A who remain on on-demand or prophylactic treatment with rFVIII

Hemophilia B patients on rFIXFc

Patients with hemophilia B who switch from on-demand or prophylactic treatment with rFIX to rFIXFc

Hemophilia A patients on rFIX

Patients with hemophilia B who remain on on-demand or prophylactic treatment with rFIX

Eligibility Criteria

• Age: 12 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

This study will be conducted in male patients ≥12 years of age with severe and moderate hemophilia A or hemophilia B (baseline factor level \<5%) who are able to sign the informed consent or assent.

You may qualify if:

• Males ≥12 years of age with severe and moderate congenital hemophilia A or B (baseline factor activity\<5%)
• Ability to understand the purpose and risks of the study and provide signed and dated informed consent or assent and authorization to use protected health information (PHI) in accordance with national and local privacy regulations.

You may not qualify if:

• Unable or unwilling to provide informed consent
• Patients with an existing bleeding disorder other than hemophilia A or B
• History of hypersensitivity or severe allergic reactions to factor products
• Patients currently participating in a phase 1-3 study with another factor replacement product
• Unable to adhere to the study requirements based on the judgment of the Prescribing Physician (e.g. unable to enter accurate and timely infusion and bleeding records)

Sponsors & Collaborators

• University of British Columbia: lead
• Biogen: collaborator

Study Sites (1)

BC Hemophilia Adult Program

Vancouver, British Columbia, V6T 2G2, Canada

Location

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Officials

• Shannon Jackson, MD

  Division of Hematology, Department of Medicine, University of British Columbia

  PRINCIPAL INVESTIGATOR

• Robert Klaassen, MD

  Division of Hematology/Oncology, Department of Pediatrics, University of Ottawa

  STUDY CHAIR

• Man-Chiu Poon, MD

  Division of Hematology, Department of Medicine, University of Calgary

  STUDY CHAIR

• Sue Robinson, MD

  Division of Hematology, Department of Medicine, Dalhousie University

  STUDY CHAIR

• John Wu, MD

  BC Children's hospital, Division of Hematology, Department of Medicine, University of British Columbia

  STUDY CHAIR

• Alfonso Iorio, MD

  Hemophilia Program, Hamilton Health Services Program, McMaster University

  STUDY CHAIR

• Michelle Sholzberg, MD

  Hemophilia Program, St. Michael's Hospital, University of Toronto

  STUDY CHAIR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Associate Professor

Study Record Dates

• First Submitted: May 26, 2016
• First Posted: June 10, 2016
• Study Start: April 1, 2016
• Primary Completion: April 26, 2021
• Study Completion: April 26, 2021
• Last Updated: April 28, 2021

Record last verified: 2021-04

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)