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Sponsored by: |
Christian Medical College, Vellore, India |
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Information provided by: | Christian Medical College, Vellore, India |
ClinicalTrials.gov Identifier: | NCT00324493 |
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 |
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Hemophilia |
Study Type: | Observational |
Study Design: | Natural History, Longitudinal, Defined Population, Prospective Study |
Official Title: | Musculoskeletal Function in Hemophilia in Developing Countries |
Estimated Enrollment: | 250 |
Study Start Date: | June 2004 |
Estimated Study Completion Date: | June 2009 |
Ages Eligible for Study: | 5 Years to 15 Years |
Genders Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Argentina, Ciudad de Buenos Aires | |
Raul Perez Bianco | |
Buenos Aires, Ciudad de Buenos Aires, Argentina | |
Brazil | |
Elbio A.D' Amico / Jorge | |
Sao Paulo, Brazil, 01246 903 | |
Brazil, Cidade Univrsitaria Zeferino Vaz-Campinas-Sp | |
Margareth Castro Ozelo / | |
Chagas, Cidade Univrsitaria Zeferino Vaz-Campinas-Sp, Brazil, 13 083 970 | |
Egypt | |
Magdy EI Ekiaby | |
Cairo, Egypt | |
India, Tamil Nadu | |
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 |
Principal Investigator: | Alok Srivastava, MD | Christian Medical College, Vellore, India |
Study ID Numbers: | MUSFIH2004 |
Study First Received: | May 9, 2006 |
Last Updated: | November 29, 2006 |
ClinicalTrials.gov Identifier: | NCT00324493 History of Changes |
Health Authority: | India: Institutional Review Board |
Severe Hemophilia Musculoskeletal Function Factor Replacement |
Hemorrhagic Disorders Genetic Diseases, Inborn Hematologic Diseases |
Blood Coagulation Disorders Hemophilia A Hemostatic Disorders |
Hemorrhagic Disorders Blood Coagulation Disorders, Inherited Genetic Diseases, Inborn Coagulation Protein Disorders |
Hematologic Diseases Blood Coagulation Disorders Hemophilia A |