Clinical trial NCT01503567: Epidemiological Study on Haemophilia Care and Orthopaedic Status in Developing Countries
Official title: Epidemiological Study on Haemophilia Care and Orthopaedic Status in Developing Countries
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Congenital Bleeding Disorder
Haemophilia A
Haemophilia A With Inhibitors
Haemophilia B
Haemophilia B With Inhibitors
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2012-01-04
Enrollment: 282
Sex: MALE
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: ECOLOGIC_OR_COMMUNITY
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Male patients at least 6 years old with severe congenital haemophilia A or B (FVIII or FIX activities below 1% or level below 1U dL\^-1) without inhibitor or with inhibitors against FVIII or FIX

Brief summary: This study is conducted in Africa and Asia. The aim of this study is to evaluate in the participating countries the orthopaedic status and the degree of arthropathy of severe haemophilia patients in general.


Eligibility criteria: Inclusion Criteria:

* Patient (and/or parents or the patient's legally acceptable representative, if applicable) must give signed and dated informed consent before enrolment in the study
* Male patients at least 6 years old with diagnosis of severe congenital haemophilia A or B with or without inhibitors
* Patients receiving on demand replacement factors/bypassing agents therapy

Exclusion Criteria:

* Clinically relevant coagulation disorders other than congenital haemophilia A or B
* Patients on currently active treatment for HCV (Hepatitis C Virus) or HIV (Human Immune Deficiency Virus) infections

Primary outcomes:
measure: Type of haemophilia and inhibitor characteristics: Against FVIII or FIX; high or low titre; anamnestic response (high or low responder)
timeFrame: After 6 months (recruitment and data collection)
measure: Clinical (using Haemophilia joint score) and radiological (using Pettersson score) orthopaedic status of defined joints: Elbow, knees and ankles in relation to haemophilia A or B
timeFrame: After 6 months (recruitment and data collection)
Secondary outcomes:
measure: Mean orthopaedic score in the 4 groups according to the Pettersson and Haemophilia joint scores
timeFrame: After 6 months (recruitment and data collection)
measure: Usage of anti haemophilic treatment in IU/kg
timeFrame: During the last year preceding patient recruitment
measure: Quality of Life - EQ-5D (Euro Quality - 5 Domains) questionnaire
timeFrame: After 6 months (recruitment and data collection)
measure: Economic aspects of the management of haemophiliacs and its burden on patient/family and community resources
timeFrame: After 6 months (recruitment and data collection)
measure: Living characteristics of the patient's household
timeFrame: After 6 months (recruitment and data collection)
Other outcomes:
Locations:
city: Bangalore
country: India
facility: Novo Nordisk Investigational Site
city: Casablanca
country: Morocco
facility: Novo Nordisk Investigational Site
city: Muscat
country: Oman
facility: Novo Nordisk Investigational Site
city: Sandton
country: South Africa
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References:
citation: Gupta N, Belhani M, Benbouzid A, Andaloussi M El, Maani K, Mahlangu J, Wali Y, Saad HA, Fegoun SB el. The Haemocare Protocol - A composite method to measure the disease burden from Haemophilia in developing countries. European Hematology Association 2013; Country: Sweden City: Stockholm