Defeat Rare Disease in Asia and Pacific (APAC) DRDA - Haemophilia

• Conditions: Haemophilia

• Registration Number: NCT07163260
• Lead Sponsor: Novo Nordisk A/S

Brief Summary

This study will help to better understand the course of disease in people with haemophilia (A or B \[with or without inhibitors\]) and the haemophilia care scenario in the selected countries and the experience so far in dealing with the condition. This is a survey-based study; hence no medications or other treatments will be provided to participants as a part of this study.

Detailed Description

Not available

Study Timeline

• Study Start: 2025-01-31
• Study First Submitted: 2025-08-06
• Status Verified: 2025-09
• Study First Submitted QC: 2025-09-02
• Last Update Submitted: 2025-09-02
• Study First Posted: 2025-09-09
• Last Update Posted: 2025-09-09
• Primary Completion: 2025-09-30
• Study Completion: 2025-09-30

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 244

Inclusion Criteria

For Experienced/Senior Treating Physicians

• Experienced/senior haematologist treating haemophilia (adult or paediatric) or physician treating patients with haemophilia. (PWH), with greater than 5 years of experience.

• Able to communicate in English or country specific language.

• Agree/sign informed consent before data collection. For Patients Organisations

• Patient organisation supporting haemophilia.

• Expert having greater than 3 years of work experience with the patient organisation.

• Expert level understanding of haemophilia landscape and care pathway in the relevant country.

• Able to communicate in English or country specific language.

• Agree/sign informed consent before data collection. For Patients/Caregivers

  1. Patients of all age groups, diagnosed with haemophilia A or haemophilia B (with or without inhibitors) for greater than 2 years.

  2. For patients less than 18 years of age (*less than 19 years for South Korea), their caregivers aged greater than or equal to 18 years (*greater than or equal to19 years for South Korea) should sign the informed consent.

     *For South Korea, the adult age is 19 years.

  3. Able to communicate in English or country specific language.

  4. Agree/sign informed consent before data collection.

Exclusion Criteria

• There is no explicit exclusion criterion in this study, thus any participants group who answers "no" to any of the inclusion criteria will be excluded from the study.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Estimated prevalence of haemophilia	From start of data collection to end of data collection (approximately 5 months)	Proportion \[%\] of individuals in a population
Estimated incidence of haemophilia	From start of data collection to end of data collection (approximately 5 months)	Number of new cases of haemophilia
Estimated proportion of patients with haemophilia (PWH) who develop inhibitors (%)	From start of data collection to end of data collection (approximately 5 months)	proportion \[%\] of individuals in a population

Trial Locations

Locations (1)

Novo Nordisk Investigational Site; 🇹🇭 Bangkok, Thailand