ThalassemiaNCT00661804
Evaluating People With Thalassemia: The Thalassemia Longitudinal Cohort (TLC) Study
An observational study of Thalassemia, run by Carelon Research at 14 sites in 3 countries.
Trial 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 have or previously had thalassemia. Read more about this study
- Who is it for?
- Ages 5 years and olderHealthy volunteers can take part
- Conditions the study is for
- Thalassemia
- How many people
- 416 participants
- Where
- 14 sitesin 3 countries
- Study dates
- May 2007to Jun 2011
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: All patients with thalassemia, as documented by clinical diagnosis, seen at sites funded by the Thalassemia Clinical Research Network (TCRN) and their satellites.
Who may be able to join
- 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)
Show all 12Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
14 sites in 3 countries.
14 sites in 3 countries
United States10 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30342
Children's Hospital Boston
Boston, Massachusetts, 02115
Children's Memorial Hospital Chicago
Chicago, Illinois, 60614
Southwestern Medical Center at Dallas
Dallas, Texas, 75390
Baylor College of Medicine
Houston, Texas, 77030
Children's Hospital of Los Angeles
Los Angeles, California, 90027
Weill Medical College of Cornell University
New York, New York, 10021
Children's Hospital of Oakland
Oakland, California, 94609
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Stanford Hospital
Stanford, California, 94305
Canada3 sites
Toronto Sick Kids
Toronto, Ontario, M5G 1X8
Toronto General Hospital
Toronto, Ontario, M5G 2C4
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3V4
United Kingdom1 site
Royal Free and University College London Medical School
London, England, WC1E 6BT
About this study
Official title: A Longitudinal Cohort Study of Patients With Thalassemia in the Thalassemia Clinical Research Network
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.
Read the 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, 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 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.
- Conditions
- Thalassemia
- Medical subject headings
- Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
The prevalence and incidence of complications specific to thalassemia and its treatment among participants
Measured: Measured throughout the duration of the study
Secondary outcomes (2)
Fertility and pregnancy outcomes; causes of mortality and changes in mortality risk; genotypic and phenotypic variation; and body iron burden
Measured: Measured throughout the duration of the study
Relationships among adherence, quality of life, and complications of thalassemia
Measured: Measured throughout the duration of the study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2008 (estimated)
- Study startMay 2007
- Primary completionDec 2010
- Study completionJun 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for ThalassemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Time perspective
- Prospective
- Enrolment
- 416 participants (actual)
Sponsor and investigators
- Lead sponsor
- Carelon Research
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Thalassemia Clinical Research Network
- Investigators
- Ellis Neufeld, MD, PhD, Study chair, Boston Children's HospitalJanet Kwiatkowski, MD, Study chair, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00661804
- Sponsor's protocol ID
- 568
- Other IDs
- U01HL065238
- Status last verified
- Jun 2011
- First submitted
- 16 Apr 2008
- Last updated
- 11 Nov 2011
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 23216870Morris 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.
PubMed 21772051
Study at a glance
From the registry record, last updated by the sponsor on 11 Nov 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Longitudinal Cohort Study of Patients With Thalassemia in the Thalassemia Clinical Research Network
- ClinicalTrials.gov ID
- NCT00661804
- Acronym / sponsor ID
- 568
- Conditions
- Thalassemia
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 416 participants (actual)
- Lead sponsor
- Carelon ResearchUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Thalassemia Clinical Research Network
- Study start
- May 2007
- Primary completion
- Dec 2010 (actual)
- Study completion
- Jun 2011 (actual)
- First posted
- 18 Apr 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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, 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 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.
Study design
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All patients with thalassemia, as documented by clinical diagnosis, seen at sites funded by the Thalassemia Clinical Research Network (TCRN) and their satellites.
Key inclusion criteria (12 listed)
- Thalassemia, as documented by clinical diagnosis, including the following types:
- Requires at least annual monitoring for end-organ injury related to thalassemia, including all clinical measures specified in this study
- 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
Key exclusion criteria (5 listed)
- Has any of the following mild or mixed diagnoses:
- Unable or unwilling to be followed annually
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
The prevalence and incidence of complications specific to thalassemia and its treatment among participants
Time frame: Measured throughout the duration of the study
2 secondary outcomes
Fertility and pregnancy outcomes; causes of mortality and changes in mortality risk; genotypic and phenotypic variation; and body iron burden
Time frame: Measured throughout the duration of the study
Relationships among adherence, quality of life, and complications of thalassemia
Time frame: Measured throughout the duration of the study
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Nov 2011.
- Derived from this study: PubMed 23216870Trachtenberg 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.
- Derived from this study: PubMed 21772051Morris 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.
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Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
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Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
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A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ellis Neufeld, MD, PhD study chair
Boston Children's Hospital
Janet Kwiatkowski, MD study chair
Children's Hospital of Philadelphia
14 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Thalassemia, run by Carelon Research at 14 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Nov 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Canada, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The prevalence and incidence of complications specific to thalassemia and its treatment among participants (time frame: Measured throughout the duration of the study). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00661804, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Nov 2011.