Musculoskeletal Function in Hemophilia

The recruitment status of this study is unknown because the information has not been verified recently.
Verified June 2005 by Christian Medical College, Vellore, India.
Recruitment status was  Active, not recruiting
Sponsor:
Information provided by:
Christian Medical College, Vellore, India
ClinicalTrials.gov Identifier:
NCT00324493
First received: May 9, 2006
Last updated: November 29, 2006
Last verified: June 2005
  Purpose

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.


Condition
Hemophilia

Study Type: Observational
Study Design: Observational Model: Defined Population
Time Perspective: Longitudinal
Official Title: Musculoskeletal Function in Hemophilia in Developing Countries

Resource links provided by NLM:


Further study details as provided by Christian Medical College, Vellore, India:

Estimated Enrollment: 250
Study Start Date: June 2004
Estimated Study Completion Date: June 2009
  Show Detailed Description

  Eligibility

Ages Eligible for Study:   5 Years to 15 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Severe hemophilia, defined as factor assay showing <1% activity (assay to be done using standard reagents), between 5-15 years of age
  • Be willing to come for evaluation at least once in 6-12 months for 5 years

Exclusion Criteria:

  • Detectable inhibitors by screening tests at recruitment
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00324493

Locations
Argentina
Raul Perez Bianco
Buenos Aires, Ciudad de Buenos Aires, Argentina
Brazil
Margareth Castro Ozelo /
Chagas, Cidade Univrsitaria Zeferino Vaz-Campinas-Sp, Brazil, 13 083 970
Elbio A.D' Amico / Jorge
Sao Paulo, Brazil, 01246 903
Egypt
Magdy EI Ekiaby
Cairo, Egypt
India
Christian Medical College
Vellore, Tamil Nadu, India, 632004
Iran, Islamic Republic of
Mohammad Reza Baghaipour
Teheran, Iran, Islamic Republic of, 14158 63675
Singapore
Tien Sim Leng
Singapore, Singapore, 169608
South Africa
Prof. Glynn Wessels
Tygerberg, South Africa
Thailand
Prof. Ampaiwan Chuansumrit
Bangkok, Thailand
Venezuela
Norma De Bosch
Caracas, Venezuela
Sponsors and Collaborators
Christian Medical College, Vellore, India
Investigators
Principal Investigator: Alok Srivastava, MD Christian Medical College, Vellore, India
  More Information

Additional Information:
No publications provided

ClinicalTrials.gov Identifier: NCT00324493     History of Changes
Other Study ID Numbers: MUSFIH2004
Study First Received: May 9, 2006
Last Updated: November 29, 2006
Health Authority: India: Institutional Review Board

Keywords provided by Christian Medical College, Vellore, India:
Severe Hemophilia
Musculoskeletal Function
Factor Replacement

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 23, 2014