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Musculoskeletal Function in Hemophilia

NCT00324493 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 250

Last updated 2006-11-30

No results posted yet for this study

Summary

Hemophilia, which results from deficiency of factor VIII or IX, is a common hereditary X-linked bleeding disorder affecting up to 10/100,000 population. About 60-70% of them have severe disease (factor level \<1%). This group is characterized by the occurrence of frequent spontaneous bleeding into joints and soft tissues. If inadequately treated, it results in progressive damage to joints and muscles leading to crippling deformities. Close clinical observation of these patients over many years has shown that those with \>1% levels have much less bleeding compared to those with less than 1%. This observation has gained immense clinical importance in planning therapy for these patients.

To prevent progressive joint damage, the missing factor needs to be replaced. Much has evolved in this practice in the last 50 years. From administration of whole blood in the beginning, to plasma and cryoprecipitate, to purified plasma-derived concentrates and finally recombinant factor concentrates. The standard of therapy now is to replace factors frequently enough to maintain \>1% factor levels at all times ("prophylaxis") or administer immediately on premonition or earliest signs of bleeding ("on demand" therapy). This has greatly enhanced the quality of life of people with hemophilia. However, the optimal regimens of factor replacement remain to be defined. The definition of what is optimal management of this chronic condition, currently incurable for the vast majority of patients, varies significantly in different parts of the world, depending on practicality and social expectations. Models have care have been developed in Western countries based on careful documentation of outcome over many years. Such data is lacking from developing countries.

This multi-center study aims to systematically record the outcome of musculoskeletal function in people with hemophilia in developing countries for the first time and provide information that can help plan care for the 80% of all hemophiliacs in the world who live in these countries. Currently there is no well documented model of care at the range of factor replacement practiced in these countries nor is there any significant information on the long-term outcome of musculo-skeletal function among these patients.

Conditions

Sponsors & Collaborators

  • Christian Medical College, Vellore, India

    lead OTHER

Principal Investigators

  • Alok Srivastava, MD · Christian Medical College, Vellore, India

Eligibility

Min Age
5 Years
Max Age
15 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-06-30
Completion
2009-06-30

Countries

  • Argentina
  • Brazil
  • Egypt
  • India
  • Iran
  • Singapore
  • South Africa
  • Thailand
  • Venezuela

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00324493 on ClinicalTrials.gov