Recruiting

Observational Study

Sponsor:

PedNet Haemophilia Research Foundation

Code:

NCT02979119

Conditions

Factor VIII Deficiency

Factor IX Deficiency

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Study Details

Brief summary:

Rationale:

Haemophilia is a rare disease; to improve knowledge international collaboration is needed. Well-defined clinical data will be collected from complete cohorts in order to prevent selection bias.

Objective:

To collect data on bleeding during neonatal period, endogenous (genetic) and exogenous (treatment-related) determinants of inhibitor development and long term outcome.

Conditions

Factor VIII Deficiency

Factor IX Deficiency

Study ID

NCT02979119

Start date

Jun, 2014

Status verified date

Sep, 2025

Completion date

Dec, 2039

Anticipated

Primary completion date

Dec, 2039

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Diagnosed with Haemophilia A or B
  • Factor VIII/ IX activity of <1 to 25%
  • Complete records of Factor treatment and bleeds
  • Treated in one of the participating centres

Exclusion Criteria:

  • Patients referred because of an inhibitor\*
  • Informed consent not obtained

Study Design

Enrollment

4000 participants

Anticipated

Interventions and Outcome Measures

Arms

Cohort I

Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2000 until December 31st 2009 who have been or are to be treated with coagulation proteins in one of the participating centres

Cohort II

Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2010 until December 31st 2019 who have been or are to be treated with coagulation proteins in one of the participating centres

Cohort III

Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2020 until December 31st 2029 who have been or are to be treated with coagulation proteins in one of the participating centres

Cohort IV

Cohort IV Group/Cohort Description: Children with mild ( FVIII/IX 6 to 25%), moderate (FVIII/IX 1 to 5%) or severe (FVIII/IX <1%) haemophilia A or B, born from January 1st 2030 until December 31st 2039 who have been or are to be treated with coagulation proteins in one of the participating centres

Primary outcome measure

  • Number of patients with antibody development to exogenous clotting factors [ Time Frame: Until patient reaches age of 18 ]

Central Contacts and Locations

Central contacts

Angelique Hermeling

+31850299993a.hermeling@pednet.eu

Hermeling

info@pednet.eu

Locations

Division of Hematology/Oncology Hôpital St Justine

Recruiting

Montreal, Canada, H3T 1C5

Division of Haematology/Oncology Hospital for Sick Children

Recruiting

Toronto, Canada, M5G-1X8

More Information

Sponsor

PedNet Haemophilia Research Foundation

Last update posted

Sep 16, 2025

Last verified

Sep, 2025

Keywords

  • Hemophilia
  • Children
  • Inhibitors
  • Prophylaxis
  • Long-term outcome
  • Congenital

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by PedNet Haemophilia Research Foundation on 2025-09-16.