Ignite Creation Date:
2025-12-24 @ 9:14 PM
Ignite Modification Date:
2026-02-22 @ 6:47 PM
Study NCT ID:
NCT00661804
Status:
COMPLETED
Last Update Posted:
2011-11-11
First Post:
2008-04-16
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Evaluating People With Thalassemia: The Thalassemia Longitudinal Cohort (TLC) Study
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D013789', 'term': 'Thalassemia'}], 'ancestors': [{'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'DNA and frozen plasma genetic biorepository'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 416}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2007-05'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2011-06', 'completionDateStruct': {'date': '2011-06', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2011-11-09', 'studyFirstSubmitDate': '2008-04-16', 'studyFirstSubmitQcDate': '2008-04-16', 'lastUpdatePostDateStruct': {'date': '2011-11-11', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2008-04-18', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2010-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'The prevalence and incidence of complications specific to thalassemia and its treatment among participants', 'timeFrame': 'Measured throughout the duration of the study'}], 'secondaryOutcomes': [{'measure': 'Fertility and pregnancy outcomes; causes of mortality and changes in mortality risk; genotypic and phenotypic variation; and body iron burden', 'timeFrame': 'Measured throughout the duration of the study'}, {'measure': 'Relationships among adherence, quality of life, and complications of thalassemia', 'timeFrame': 'Measured throughout the duration of the study'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Thalassemia']}, 'referencesModule': {'references': [{'pmid': '23216870', 'type': 'DERIVED', 'citation': 'Trachtenberg FL, Mednick L, Kwiatkowski JL, Neufeld EJ, Haines D, Pakbaz Z, Thompson AA, Quinn CT, Grady R, Sobota A, Olivieri N, Horne R, Yamashita R; Thalassemia Clinical Research Network. Beliefs about chelation among thalassemia patients. Health Qual Life Outcomes. 2012 Dec 7;10:148. doi: 10.1186/1477-7525-10-148.'}, {'pmid': '21772051', 'type': 'DERIVED', 'citation': 'Morris CR, Kim HY, Trachtenberg F, Wood J, Quinn CT, Sweeters N, Kwiatkowski JL, Thompson AA, Giardina PJ, Boudreaux J, Olivieri NF, Porter JB, Neufeld EJ, Vichinsky EP; Thalassemia Clinical Research Network. Risk factors and mortality associated with an elevated tricuspid regurgitant jet velocity measured by Doppler-echocardiography in thalassemia: a Thalassemia Clinical Research Network report. Blood. 2011 Oct 6;118(14):3794-802. doi: 10.1182/blood-2010-11-319152. Epub 2011 Jul 19.'}]}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': "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.\n\nThis study has enrolled 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."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '5 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'All patients with thalassemia, as documented by clinical diagnosis, seen at sites funded by the Thalassemia Clinical Research Network (TCRN) and their satellites.', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria for People with Thalassemia:\n\n* Thalassemia, as documented by clinical diagnosis, including the following types:\n\n 1. Beta-thalassemia (intermedia or major)\n 2. Hemoglobin H (HbH) disease\n 3. HbH with non-deletional mutations (e.g., HbH Constant Spring)\n 4. E-beta-thalassemia\n 5. Homozygous alpha-thalassemia (i.e., 4-gene alpha deletion or equivalent null alpha mutation)\n 6. Other thalassemic conditions not explicitly excluded\n 7. Thalassemia intermedia due to heterozygous beta mutation with alpha-gene excess\n* Requires at least annual monitoring for end-organ injury related to thalassemia, including all clinical measures specified in this study\n\nInclusion Criteria for People who Have Received a Successful Stem Cell Transplant:\n\n* 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\n* Monitored for end-organ injury related to thalassemia before their successful stem cell transplant, including all clinical measures specified in this study\n\nExclusion Criteria for People with Thalassemia:\n\n* Has any of the following mild or mixed diagnoses:\n\n 1. Thalassemia trait (i.e., single recessive beta-gene mutation, two-gene alpha-gene mutation)\n 2. Thalassemia/Hb S, C, or D compound heterozygotes\n 3. 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)\n* Unable or unwilling to be followed annually'}, 'identificationModule': {'nctId': 'NCT00661804', 'briefTitle': 'Evaluating People With Thalassemia: The Thalassemia Longitudinal Cohort (TLC) Study', 'organization': {'class': 'OTHER', 'fullName': 'Carelon Research'}, 'officialTitle': 'A Longitudinal Cohort Study of Patients With Thalassemia in the Thalassemia Clinical Research Network', 'orgStudyIdInfo': {'id': '568'}, 'secondaryIdInfos': [{'id': 'U01HL065238', 'link': 'https://reporter.nih.gov/quickSearch/U01HL065238', 'type': 'NIH'}]}, 'armsInterventionsModule': {'armGroups': [{'label': 'Thalassemia cohort', 'description': 'Thalassemia as documented by clinical diagnosis, including:\n\nthalassemia (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.'}, {'label': 'Successful SCT cohort', 'description': '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.'}]}, 'contactsLocationsModule': {'locations': [{'zip': '90027', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital of Los Angeles", 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94609', 'city': 'Oakland', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital of Oakland", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '94305', 'city': 'Stanford', 'state': 'California', 'country': 'United States', 'facility': 'Stanford Hospital', 'geoPoint': {'lat': 37.42411, 'lon': -122.16608}}, {'zip': '30342', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': "Children's Healthcare of Atlanta", 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '60614', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Children's Memorial Hospital Chicago", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Children's Hospital Boston", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '10021', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Weill Medical College of Cornell University', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '75390', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'Southwestern Medical Center at Dallas', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Baylor College of Medicine', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': 'V6H 3V4', 'city': 'Vancouver', 'state': 'British Columbia', 'country': 'Canada', 'facility': "British Columbia Children's Hospital", 'geoPoint': {'lat': 49.24966, 'lon': -123.11934}}, {'zip': 'M5G 1X8', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Toronto Sick Kids', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'zip': 'M5G 2C4', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Toronto General Hospital', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'zip': 'WC1E 6BT', 'city': 'London', 'state': 'England', 'country': 'United Kingdom', 'facility': 'Royal Free and University College London Medical School', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Ellis Neufeld, MD, PhD', 'role': 'STUDY_CHAIR', 'affiliation': "Boston Children's Hospital"}, {'name': 'Janet Kwiatkowski, MD', 'role': 'STUDY_CHAIR', 'affiliation': "Children's Hospital of Philadelphia"}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Carelon Research', 'class': 'OTHER'}, 'collaborators': [{'name': 'National Heart, Lung, and Blood Institute (NHLBI)', 'class': 'NIH'}, {'name': 'Thalassemia Clinical Research Network', 'class': 'NETWORK'}], 'responsibleParty': {'oldNameTitle': 'National Heart, Lung and Blood Institute', 'oldOrganization': 'National Institutes of Health'}}}}