ThalassemiaNCT00872833
Assessment of Pain in People With Thalassemia Who Are Treated With Regular Blood Transfusions
An observational study of Thalassemia, run by Carelon Research at 9 sites in 2 countries.
Trial summary
Thalassemia is an inherited blood disorder that can result in mild to severe anemia. Regular blood transfusions, which refresh the healthy red blood cell supply, are one treatment for thalassemia. People with thalassemia often experience pain, but the exact source of pain remains unknown. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thalassemia
- How many people
- 32 participants
- Where
- 9 sitesin 2 countries
- Study dates
- Jul 2009to Oct 2010
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: People with transfusion-dependent thalassemia who receive care at sites funded by the Thalassemia Clinical Research Network and their satellites.
Who may be able to join
- Participating in the Thalassemia Clinical Research Network Assessment of Pain study
- Has a transfusion dependence of at least eight transfusions per year
- Diagnosis of beta thalassemia or E-beta-thalassemia
- Experiences at least "mild" pain in the 1 month before study entry, as measured by the response to the Brief Pain Inventory (BPI) question #6 from the Assessment of Pain study
Who cannot join
- Unwillingness or inability to complete the Brief Pain Inventory Short Form (BPI-SF) on a daily basis
Trial locations and contacts
9 sites in 2 countries.
9 sites in 2 countries
United States8 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30342
Children's Memorial Hospital - Chicago
Chicago, Illinois, 60614
Baylor College of Medicine
Houston, Texas, 77030
Children's Hospital of Los Angeles
Los Angeles, California, 90027
Weill Medical College of Cornell
New York, New York, 10065
Children's Hospital Research Center Oakland
Oakland, California, 94609
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Stanford University - School of Medicine
Stanford, California, 94305
Canada1 site
University Health Network - Toronto General Hospital
Toronto, Ontario, M5G 2C4
About this study
Official title: Assessment of Pain in Transfusion Dependent Patients With Thalassemia During Transfusion Cycles
Thalassemia is an inherited blood disorder that can result in mild to severe anemia. Regular blood transfusions, which refresh the healthy red blood cell supply, are one treatment for thalassemia. People with thalassemia often experience pain, but the exact source of pain remains unknown. This study will examine how pain varies during the blood transfusion cycle in people with thalassemia who are treated with regular blood transfusions.
Read the detailed description
Thalassemia is an inherited blood disorder in which the body makes an abnormal form of hemoglobin-the protein in red blood cells that carries oxygen. People with thalassemia often experience fatigue, shortness of breath, and pain. There have been no previous research studies that have fully examined pain levels in people with thalassemia, and as a result, the sources of pain remain unknown. This study is a substudy of the Assessment of Pain study, which is a Thalassemia Clinical Research Network (TCRN) study that is examining the prevalence and severity of pain in people with transfusion-dependent thalassemia and non-transfusion-dependant thalassemia. This study will enroll a subset of participants from the Assessment of Pain study who have transfusion-dependant thalassemia. The purpose of this study is to examine whether pain varies during the blood transfusion cycle and whether the length of the transfusion cycle affects pain levels in people with transfusion-dependent thalassemia.
Participants will complete daily questionnaires through an automated telephone system to assess pain levels during three blood transfusion cycles. Each transfusion cycle will last between 2 to 4 weeks, depending on the individual needs of the participant, and the cycles will be separated by at least 3 months but no more than 4 months. Prior to each transfusion cycle, study researchers will review participants' medical records for certain blood level measurements.
- Conditions
- Thalassemia
- Keywords
- Transfusion-Dependant Thalassemia, Pain, Mild Pain
- Medical subject headings
- Thalassemia, Pain
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 |
|---|---|---|
| age 18-29 | People age groups 18-29 with transfusion-dependent thalassemia who have reported at least mild degrees of pain during the main Assessment of Pain study. | |
| age 30+ | People age groups 30+ years with transfusion-dependent thalassemia who have reported at least mild degrees of pain during the main Assessment of Pain study. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Report of Pain by Age Group
Subjects reported whether or not they had pain (yes/no) during each transfusion cycle (data on up to 3 transfusion cycles were collected for each subject). The percent of subject-cycles with pain was calculated for the quartiles of the transfusion cycle.
Measured: measured daily over the 3 transfusion cycles
Secondary outcomes (1)
Report of Pain by Length of the Transfusion Cycle
Subjects reported whether or not they had pain (yes/no) during each transfusion cycle (data on up to 3 transfusion cycles were collected for each subject). The percent of subject-cycles with pain was calculated for the quartiles of the transfusion cycle.
Measured: measured daily over the 3 transfusion cycles
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Mar 2009 (estimated)
- Study startJul 2009
- Primary completionOct 2010
- Study completionOct 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Carelon Research
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Dru Foote, NP, Principal investigator, Children's Hospital and Research Institute OaklandJeanne Boudreaux, MD, Study chair, Children's Healthcare of AtlantaThomas Coates, MD, Study chair, Children's Hospital Los AngelesElliott Vichinsky, MD, Study chair, UCSF Benioff Children's Hospital OaklandMichael Jeng, MD, Study chair, Stanford UniversityJanet Kwiatkowski, MD, Study chair, Children's Hospital of PhiladelphiaNancy Olivieri, MD, Study chair, University Health Network - Toronto General HospitalPatricia J. Giardina, MD, Study chair, Weill Medical College of Cornell
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00872833
- Sponsor's protocol ID
- 640
- Other IDs
- U01HL065238
- Status last verified
- Apr 2012
- First submitted
- 30 Mar 2009
- Last updated
- 7 Oct 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 7 Oct 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Pain in Transfusion Dependent Patients With Thalassemia During Transfusion Cycles
- ClinicalTrials.gov ID
- NCT00872833
- Acronym / sponsor ID
- 640
- Conditions
- Thalassemia
- Keywords
- Transfusion-Dependant Thalassemia, Pain, Mild Pain
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Carelon ResearchUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- Jul 2009
- Primary completion
- Oct 2010 (actual)
- Study completion
- Oct 2010 (actual)
- First posted
- 31 Mar 2009 (estimated)
- Results
- Posted 27 Aug 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Thalassemia is an inherited blood disorder that can result in mild to severe anemia. Regular blood transfusions, which refresh the healthy red blood cell supply, are one treatment for thalassemia. People with thalassemia often experience pain, but the exact source of pain remains unknown. This study will examine how pain varies during the blood transfusion cycle in people with thalassemia who are treated with regular blood transfusions.
Detailed description
Thalassemia is an inherited blood disorder in which the body makes an abnormal form of hemoglobin-the protein in red blood cells that carries oxygen. People with thalassemia often experience fatigue, shortness of breath, and pain. There have been no previous research studies that have fully examined pain levels in people with thalassemia, and as a result, the sources of pain remain unknown. This study is a substudy of the Assessment of Pain study, which is a Thalassemia Clinical Research Network (TCRN) study that is examining the prevalence and severity of pain in people with transfusion-dependent thalassemia and non-transfusion-dependant thalassemia. This study will enroll a subset of participants from the Assessment of Pain study who have transfusion-dependant thalassemia. The purpose of this study is to examine whether pain varies during the blood transfusion cycle and whether the length of the transfusion cycle affects pain levels in people with transfusion-dependent thalassemia.
Participants will complete daily questionnaires through an automated telephone system to assess pain levels during three blood transfusion cycles. Each transfusion cycle will last between 2 to 4 weeks, depending on the individual needs of the participant, and the cycles will be separated by at least 3 months but no more than 4 months. Prior to each transfusion cycle, study researchers will review participants' medical records for certain blood level measurements.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| age 18-29 | – | –People age groups 18-29 with transfusion-dependent thalassemia who have reported at least mild degrees of pain during the main Assessment of Pain study. |
| age 30+ | – | –People age groups 30+ years with transfusion-dependent thalassemia who have reported at least mild degrees of pain during the main Assessment of Pain study. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- People with transfusion-dependent thalassemia who receive care at sites funded by the Thalassemia Clinical Research Network and their satellites.
Key inclusion criteria (4 listed)
- Participating in the Thalassemia Clinical Research Network Assessment of Pain study
- Has a transfusion dependence of at least eight transfusions per year
- Diagnosis of beta thalassemia or E-beta-thalassemia
- Experiences at least "mild" pain in the 1 month before study entry, as measured by the response to the Brief Pain Inventory (BPI) question #6 from the Assessment of Pain study
Key exclusion criteria (1 listed)
- Unwillingness or inability to complete the Brief Pain Inventory Short Form (BPI-SF) on a daily basis
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
Report of Pain by Age Group
Time frame: measured daily over the 3 transfusion cycles
Subjects reported whether or not they had pain (yes/no) during each transfusion cycle (data on up to 3 transfusion cycles were collected for each subject). The percent of subject-cycles with pain was calculated for the quartiles of the transfusion cycle.
1 secondary outcome
Report of Pain by Length of the Transfusion Cycle
Time frame: measured daily over the 3 transfusion cycles
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Oct 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Thalassemia
Active, not recruiting
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
Transfusion-dependent Beta-Thalassemia
RecruitingObservational study
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
Not yet recruiting
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Beta-Thalassemia
RecruitingPhase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
Transfusion-dependent Beta-Thalassemia
Active, not recruitingPhase 3
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.
Dru Foote, NP principal investigator
Children's Hospital and Research Institute Oakland
Jeanne Boudreaux, MD study chair
Children's Healthcare of Atlanta
Thomas Coates, MD study chair
Children's Hospital Los Angeles
9 sites in 2 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 9 sites in 2 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 4 inclusion and 1 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 7 Oct 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Canada. 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?
Yes. Results are posted in the registry (first posted 27 Aug 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Report of Pain by Age Group (time frame: measured daily over the 3 transfusion cycles). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 7 Oct 2015.