EPOCH-Economics and Patient Outcomes in China in Haemophilia

NCT07454239 | Active Not Recruiting

• 2 other identifiers: QTJC2024006EPOCH Study, Ltd.
• Study Type: observational
• Target: 500
• Locations: 1 country
• Sites: 1

Brief Summary

The overarching goal of the EPOCH project is to:

• Support a broad and robust needs assessment in the haemophilia space in China
• Measure the variability in the needs and care provision
• Generate outcome data (health economic outcomes and supporting clinical and PRO data) to enable measuring the impact of different treatment modalities and different levels of treatment access PRIMARY OBJECTIVES
• Determine the burden of haemophilia in China, and the determinants of its variability
• Determine the costs of haemophilia care in China, and the determinants of its variability SECONDARY OBJECTIVES
• Measure treatment patterns and their variability
• Measure levels of access to care
• Estimate the impact of haemophilia and its treatment of patients reported outcomes
• Understand consistency of care by centres/geography/demographics

Conditions

Hemophilia

Outcome Measures

Primary Outcomes (7)

• Successfully registered research centers

  Successfully registered research centers in Trial Phase

  Baseline until week 52

• Number of enrolled patients

  Number of enrolled patients in Trial Phase

  Baseline until week 52

• Proportion of completed questionnaires

  Proportion of completed questionnaires for the Trial Phase

  Baseline until week 52

• Proportion of missing data

  Proportion of missing data for the Trial Phase

  Baseline until week 52

• Qualitative information on study barriers and facilitators

  Qualitative information on study barriers and facilitators for the Trial Phase

  Baseline until week 52

• Diagnostic and treatment burden for hemophilia patients

  Diagnostic and treatment burden for hemophilia patients for the Implementation Phase

  Baseline until week 52

• Costs of hemophilia diagnosis and treatment

  Costs of hemophilia diagnosis and treatment for the Implementation Phase

  Baseline until week 52

Secondary Outcomes (3)

• Measure treatment patterns and their variability

  Baseline until week 52

• Measure levels of access to care

  Baseline until week 52

• Estimate the impact of haemophilia and its treatment on patients reported outcomes

  Baseline until week 52

Eligibility Criteria

• Age: 12 Minutes+
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Male patients, aged ≥12 years at the index date

You may qualify if:

• Male patients, aged ≥12 years at the index date
• Patients must be diagnosed with non-acquired severe haemophilia A or B prior to the previous 12 months
• Patients who are able to understand and assent / consent to the study

You may not qualify if:

• Patients who cannot, for any reasons (language barriers, mental incapacity, etc.) understand and answer the questions of the study questionnaires
• Patients that cannot access internet connection or download the application

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead
• EPOCH Study, Ltd.: collaborator

Study Sites (1)

Instiute of Hematology and Blood Diseases Hospital CAMS＆PUMC

Tianjin, Tianjin Municipality, 300020, China

Location

MeSH Terms

Conditions

Hemophilia A

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

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: CROSS SECTIONAL
• Target Duration: 1 Year
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 5, 2025
• First Posted: March 6, 2026
• Study Start: November 14, 2024
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): December 31, 2028
• Last Updated: March 6, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share

Locations

CN (1)