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

NCT07163260 | Enrolling By Invitation

• 2 other identifiers: DAS-8107U1111-1303-7372
• Study Type: observational
• Target: 244
• Locations: 4 countries
• Sites: 4

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.

Conditions

Haemophilia

Outcome Measures

Primary Outcomes (3)

• Estimated prevalence of haemophilia

  Proportion \[%\] of individuals in a population

  From start of data collection to end of data collection (approximately 5 months)

• Estimated incidence of haemophilia

  Number of new cases of haemophilia

  From start of data collection to end of data collection (approximately 5 months)

• Estimated proportion of patients with haemophilia (PWH) who develop inhibitors (%)

  proportion \[%\] of individuals in a population

  From start of data collection to end of data collection (approximately 5 months)

Study Arms (3)

Experienced/Senior Treating Physicians

This group includes haematologists, paediatric haematologist and physicians treating patients with haemophilia (PWH)

Other: No treatment given

Patients Organisations

This group includes the organisations working closely to support patient with haemophillia (PWH) at a national/regional level

Other: No treatment given

Patients/Caregivers

This group includes patients with haemophilia and caregivers managing patients with haemophilia

Other: No treatment given

Interventions

No treatment given OTHER

No treatment given

Experienced/Senior Treating Physicians; Patients Organisations; Patients/Caregivers

Eligibility Criteria

• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study will enroll 3 types of participant categories: 1. Experienced/senior treating physicians i.e., haematologists, paediatric haematologist and physicians treating patients with haemophilia (PWH) 2. Patient organisations (representatives of patient organisations), defined as the organisations working closely to support PWH at a national/regional level 3. Patients/caregivers.

You may qualify if:

• 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
• Patients of all age groups, diagnosed with haemophilia A or haemophilia B (with or without inhibitors) for greater than 2 years.
• 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.
• Able to communicate in English or country specific language.
• Agree/sign informed consent before data collection.

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (4)

Novo Nordisk Investigational Site

Bangalore, Karnataka, 560 066, India

Location

Novo Nordisk Investigational Site

Kuala Lumpur, 50470, Malaysia

Location

Novo Nordisk Investigational Site

Seoul, South Korea

Location

Novo Nordisk Investigational Site

Bangkok, 10500, Thailand

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 Officials

• Clinical Transparency (dept. 2834)

  Novo Nordisk A/S

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 6, 2025
• First Posted: September 9, 2025
• Study Start: January 31, 2025
• Primary Completion: December 19, 2025
• Study Completion: December 19, 2025
• Last Updated: October 24, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share

Locations

TH (1); MY (1); IN (1); KR (1)