Hemophilia Clinical Trial
Official title:
Musculoskeletal Function in Hemophilia in Developing Countries
Verified date | June 2005 |
Source | Christian Medical College, Vellore, India |
Contact | n/a |
Is FDA regulated | No |
Health authority | India: Institutional Review Board |
Study type | Observational |
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.
Status | Active, not recruiting |
Enrollment | 250 |
Est. completion date | June 2009 |
Est. primary completion date | |
Accepts healthy volunteers | No |
Gender | Male |
Age group | 5 Years to 15 Years |
Eligibility |
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 |
Observational Model: Defined Population, Time Perspective: Longitudinal
Country | Name | City | State |
---|---|---|---|
Argentina | Raul Perez Bianco | Buenos Aires | Ciudad de Buenos Aires |
Brazil | Margareth Castro Ozelo / | Chagas | Cidade Univrsitaria Zeferino Vaz-Campinas-Sp |
Brazil | Elbio A.D' Amico / Jorge | Sao Paulo | |
Egypt | Magdy EI Ekiaby | Cairo | |
India | Christian Medical College | Vellore | Tamil Nadu |
Iran, Islamic Republic of | Mohammad Reza Baghaipour | Teheran | |
Singapore | Tien Sim Leng | Singapore | |
South Africa | Prof. Glynn Wessels | Tygerberg | |
Thailand | Prof. Ampaiwan Chuansumrit | Bangkok | |
Venezuela | Norma De Bosch | Caracas |
Lead Sponsor | Collaborator |
---|---|
Christian Medical College, Vellore, India |
Argentina, Brazil, Egypt, India, Iran, Islamic Republic of, Singapore, South Africa, Thailand, Venezuela,
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT05617209 -
In Vitro Correction of Thrombin Generation by Concizumab (Anti-TFPI) for Severe Hemophilia Patients
|
||
Completed |
NCT05039008 -
Restricting Blood Flow in Improving Muscle Strength in Patients With Hemophilic Arthropathy
|
N/A | |
Recruiting |
NCT04398628 -
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
|
||
Unknown status |
NCT02433782 -
Myofascial Therapy in Patients With Hemophilic Arthropathy
|
N/A | |
Completed |
NCT02165462 -
Bilateral Deficit Phenomenon in Patients With Haemophilic Arthropathy
|
N/A | |
Terminated |
NCT02586012 -
Weight-based Dosing in Hemophilia A
|
Phase 2 | |
Unknown status |
NCT02165592 -
Assessment of Proprioceptive and Functional Characteristics in Patients With Hemophilia
|
N/A | |
Completed |
NCT02546622 -
ATHN 2: Factor Switching Study
|
||
Completed |
NCT01232634 -
Validation of Ultrasound as a Diagnostic Tool for Assessment of Hemophilic Arthropathy of Knees and Ankles
|
Phase 2 | |
Completed |
NCT05104164 -
Self-myofascial Release in Hemophilic Ankle Arthropathy
|
N/A | |
Terminated |
NCT01191372 -
First-in-Human and Proof-of-Mechanism Study of ARC19499 Administered to Hemophilia Patients
|
Phase 1 | |
Completed |
NCT05173129 -
Posture Analysis for Patients With Haemophilia
|
N/A | |
Completed |
NCT03818529 -
ATHN 8: Previously Untreated Patients (PUPs) Matter Study
|
||
Withdrawn |
NCT03996486 -
Study to Test the Safety of an Investigational Drug Given Repeatedly to Adult Men With Severe Hemophilia
|
Phase 1 | |
Completed |
NCT01708564 -
A Phase I Safety, Pharmacokinetics and Pharmacodynamics Study of Recombinant Factor VIIa in Adult Patients With Hemophilia A or B
|
Phase 1 | |
Completed |
NCT03842605 -
Efficacy of Strength Training in Improving Elbow Range of Motion and Function in Adults With Hemophilia
|
N/A | |
Completed |
NCT05549843 -
Manual Therapy in the Treatment of Hemophilic Arthropathy of the Ankle
|
N/A | |
Recruiting |
NCT06010953 -
SS109 and NovoSeven ® PK / PD Profile, and Preliminary Efficacy and Safety of SS109 on Demand Treatment
|
Phase 1/Phase 2 | |
Completed |
NCT05027230 -
A Safety and Efficacy Study of STSP-0601 in Adult Patients With Hemophilia A or B With Inhibitor
|
Phase 1/Phase 2 | |
Not yet recruiting |
NCT06014320 -
Alterations in Coagulation Factor Levels in Patients With End Stage Liver Disease
|