Home
Search
Study Topics
Glossary
|
|
|
|
|
Sponsors and Collaborators: |
National Heart, Lung, and Blood Institute (NHLBI) Thalassemia Clinical Research Network |
---|---|
Information provided by: | National Heart, Lung, and Blood Institute (NHLBI) |
ClinicalTrials.gov Identifier: | NCT00661804 |
Thalassemias are inherited blood disorders that can cause anemia and other health problems. The goal of this study is to collect information on complications of the disease among people who currently have or previously had thalassemia.
Condition |
---|
Thalassemia |
Study Type: | Observational |
Study Design: | Prospective |
Official Title: | A Longitudinal Cohort Study of Patients With Thalassemia in the Thalassemia Clinical Research Network |
Blood and plasma
Estimated Enrollment: | 400 |
Study Start Date: | May 2007 |
Estimated Study Completion Date: | June 2010 |
Estimated Primary Completion Date: | April 2010 (Final data collection date for primary outcome measure) |
Groups/Cohorts |
---|
Thalassemia cohort
Thalassemia as documented by clinical diagnosis, including: thalassemia (intermedia or major); HbH disease; HbH with non-deletional mutations, e.g., HbH Constant Spring E beta-thalassemia; Homozygous alpha-thalassemia (i.e., 4-gene alpha deletion or equivalent null alpha mutation); Other thalassemic conditions not explicitly excluded; Thalassemia intermedia due to heterozygous beta mutation with alpha-gene excess. |
Successful SCT cohort
Individuals who have received a successful hematopoietic SCT, defined as engraftment of all three cell lines and transfusion independence by 100 days post-transplant, for any of the disorders listed above;Monitored for end-organ injury related to thalassemia prior to their successful SCT;Participants who were enrolled in TCRN Registry or had a successful SCT after 01 Jan 2002.
|
Thalassemias are inherited blood disorders that are characterized by low levels of hemoglobin and healthy red blood cells. The two major types of thalassemia are alpha thalassemia and beta thalassemia, and there are several forms of each type. Symptoms can range from mild to severe and may include anemia, delayed growth, bone problems, and an enlarged spleen. People with mild forms of the disease may not need any treatment, while people with moderate to severe thalassemia may be treated with blood transfusions to refresh the healthy red blood cell supply, iron chelation therapy to remove excess iron from the body, and folic acid supplements to help build healthy red blood cells. Stem cell transplants can cure the disease, but they are not widely used because of the difficulty of finding donors. This study will establish a database of people with thalassemia and people who used to have thalassemia to examine the prevalence and incidence of complications related to the disease.
Participants' DNA will be analyzed and plasma will be collected for use in future studies. Participants in this study may also be asked if they are interested in enrolling in other Thalassemia Clinical Research Network studies.
This study will enroll people with thalassemia or people whose thalassemia was cured after undergoing a stem cell transplant. At a baseline study visit, participants with thalassemia will undergo a medical history interview; a medical record review; blood collection; and questionnaires on quality of life, nutritional status, and medication adherence. Follow-up visits will occur once a year for at least 3 years or for the duration of the study and will include repeat baseline testing. Participants who have undergone a successful stem cell transplant will attend only one study visit that will include a medical history interview, a medical record review, and quality of life questionnaires.
Ages Eligible for Study: | 5 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | Yes |
Sampling Method: | Non-Probability Sample |
All patients with thalassemia, as documented by clinical diagnosis, seen at sites funded by the Thalassemia Clinical Research Network (TCRN) and their satellites.
Inclusion Criteria for People with Thalassemia:
Thalassemia, as documented by clinical diagnosis, including the following types:
Inclusion Criteria for People who Have Received a Successful Stem Cell Transplant:
Exclusion Criteria for People with Thalassemia:
Has any of the following mild or mixed diagnoses:
United States, California | |
Children's Hospital of Los Angeles | |
Los Angeles, California, United States, 90027 | |
Children's Hospital of Oakland | |
Oakland, California, United States, 94609 | |
Stanford Hospital | |
Stanford, California, United States, 94305 | |
United States, Georgia | |
Children's Healthcare of Atlanta | |
Atlanta, Georgia, United States, 30342 | |
United States, Illinois | |
Children's Memorial Hospital Chicago | |
Chicago, Illinois, United States, 60614 | |
United States, Massachusetts | |
Children's Hospital Boston | |
Boston, Massachusetts, United States, 02115 | |
United States, New York | |
Weill Medical College of Cornell University | |
New York, New York, United States, 10021 | |
United States, Pennsylvania | |
Children's Hospital of Philadelphia | |
Philadelphia, Pennsylvania, United States, 19104 | |
United States, Texas | |
Southwestern Medical Center at Dallas | |
Dallas, Texas, United States, 75390 | |
Baylor College of Medicine | |
Houston, Texas, United States, 77030 | |
Canada, British Columbia | |
British Columbia Children's Hospital | |
Vancouver, British Columbia, Canada, V6H 3V4 | |
Canada, Ontario | |
Toronto General Hospital | |
Toronto, Ontario, Canada, M5G 2C4 | |
Toronto Sick Kids | |
Toronto, Ontario, Canada, M5G 1X8 | |
United Kingdom, England | |
Royal Free and University College London Medical School | |
London, England, United Kingdom, WC1E 6BT |
Study Chair: | Ellis Neufeld, MD, PhD | Children's Hospital Boston |
Study Chair: | Janet Kwiatkowski, MD | Children's Hospital of Philadelphia |
Responsible Party: | National Institutes of Health ( National Heart, Lung and Blood Institute ) |
Study ID Numbers: | 568, U01 HL065238 |
Study First Received: | April 16, 2008 |
Last Updated: | July 10, 2009 |
ClinicalTrials.gov Identifier: | NCT00661804 History of Changes |
Health Authority: | United States: Federal Government |
Anemia, Hemolytic, Congenital Genetic Diseases, Inborn Hematologic Diseases Hemoglobinopathies |
Anemia Anemia, Hemolytic Hemoglobinopathy Thalassemia |
Anemia, Hemolytic, Congenital Genetic Diseases, Inborn Hematologic Diseases Hemoglobinopathies |
Anemia Anemia, Hemolytic Thalassemia |