Status:
COMPLETED
Evaluating People With Thalassemia: The Thalassemia Longitudinal Cohort (TLC) Study
Lead Sponsor:
Carelon Research
Collaborating Sponsors:
National Heart, Lung, and Blood Institute (NHLBI)
Thalassemia Clinical Research Network
Conditions:
Thalassemia
Eligibility:
All Genders
5+ years
Brief Summary
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 ...
Detailed Description
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...
Eligibility Criteria
Inclusion
- Inclusion Criteria for People with Thalassemia:
- Thalassemia, as documented by clinical diagnosis, including the following types:
- Beta-thalassemia (intermedia or major)
- Hemoglobin H (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
- Requires at least annual monitoring for end-organ injury related to thalassemia, including all clinical measures specified in this study
- Inclusion Criteria for People who Have Received a Successful Stem Cell Transplant:
- Received a successful hematopoietic stem cell transplant, defined as engraftment of all three cell lines and transfusion independence by 100 days post-transplant, for any of the thalassemia disorders listed above
- Monitored for end-organ injury related to thalassemia before their successful stem cell transplant, including all clinical measures specified in this study
- Exclusion Criteria for People with Thalassemia:
- Has any of the following mild or mixed diagnoses:
- Thalassemia trait (i.e., single recessive beta-gene mutation, two-gene alpha-gene mutation)
- Thalassemia/Hb S, C, or D compound heterozygotes
- HbH with steady state hemoglobin above 9.0 g/dL and no history of significant thalassemia complications (e.g., endocrinopathies, cardiac dysfunction, growth impairment, pulmonary hypertension)
- Unable or unwilling to be followed annually
Exclusion
Key Trial Info
Start Date :
May 1 2007
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
June 1 2011
Estimated Enrollment :
416 Patients enrolled
Trial Details
Trial ID
NCT00661804
Start Date
May 1 2007
End Date
June 1 2011
Last Update
November 11 2011
Active Locations (14)
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1
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
2
Children's Hospital of Oakland
Oakland, California, United States, 94609
3
Stanford Hospital
Stanford, California, United States, 94305
4
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30342