ThalassemiaPainNCT00872339
Assessment of Pain in People With Thalassemia
An observational study of Thalassemia, run by Carelon Research at 8 sites in 2 countries.
Trial summary
Thalassemia is an inherited blood disorder that can result in mild to severe anemia. People with thalassemia often experience pain, but the exact sources and prevalence of pain remain unknown. Read more about this study
- Who is it for?
- Ages 12 to 90 years
- Conditions the study is for
- Thalassemia
- How many people
- 252 participants
- Where
- 8 sitesin 2 countries
- Study dates
- Mar 2009to Dec 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 12 to 90 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Thalassemia patients who receive regular blood transfusions and those who do not receive regular blood transfusions.
Who may be able to join
- Thalassemia, as documented by clinical diagnosis, including the following:
- B-thalassemia (intermedia or major)
- Hgb H disease
- Hgb H with non-deletional mutations (e.g., Hgb H Constant Spring)
- E-B-thalassemia
- Homozygous alpha thalassemia
Show all 9Show fewer
- Other thalassemic conditions not explicitly excluded
- Thalassemia intermedia due to heterozygous B mutation with an alpha excess
- Participants can be of any race, ethnicity, and either gender.
Who cannot join
- Thalassemia trait (i.e., single recessive B gene mutation, 2 gene alpha mutation) and thalassemia Hgb S, C, or D compound heterozygotes
- Unwillingness or inability to complete the Brief Pain Inventory (BPI) on a quarterly basis
- Has had a successful bone marrow transplant
Trial locations and contacts
8 sites in 2 countries.
8 sites in 2 countries
United States7 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30342
Children's Hospital of Boston
Boston, Massachusetts, 02115
Children's Memorial Hospital of Chicago
Chicago, Illinois, 60614
Baylor College of Medicine
Houston, Texas, 77030
Weill Medical College
New York, New York, 10065
Children's Hospital and Research Institute at Oakland
Oakland, California, 94609
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Thalassemia is an inherited blood disorder that can result in mild to severe anemia. People with thalassemia often experience pain, but the exact sources and prevalence of pain remain unknown. This study will examine the prevalence and severity of pain in people with thalassemia who are treated with regular blood transfusions and people with thalassemia who are not 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. Recent medical advances in treating people with thalassemia who receive regular blood transfusions-a standard procedure that refreshes the healthy red blood supply-have resulted in increased life spans for these people. However, with the extended life spans have come additional issues, including pain. There have been no previous research studies that have examined pain levels in people with thalassemia, and as a result, the sources and prevalence of pain remain unknown. The purpose of this study is to assess the prevalence and severity of pain, common pain sites, and the impact of pain on functioning and well-being in people with thalassemia who receive regular blood transfusions and people with thalassemia who do not receive regular blood transfusions.
This study will enroll people with transfusion-dependant thalassemia and people with non-transfusion-dependant thalassemia. At a baseline study visit, participants will complete a demographic questionnaire and a pain assessment questionnaire. At Months 3, 6, and 9, study researchers will telephone participants to go over the same pain assessment questionnaire again.
- Conditions
- Thalassemia
- Keywords
- Transfusion-Dependent Thalassemia, Non-Transfusion-Dependent 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 |
|---|---|---|
| transfusion-dependant | People with transfusion-dependant thalassemia who received at least 8 transfusions in the past year. | |
| non-transfusion-dependant | People with non-transfusion-dependant thalassemia who received no transfusions in the past year. | |
| intermittently transfused | Intermittently transfused patients- individuals who received at least one but fewer than eight transfusions in the last year |
What is being measured
The primary outcome is the main question the study is designed to answer.
Prevalence of Pain
Measured: Measured at Month 9
Secondary outcomes (3)
Common Sites of Pain
Measured: Measured at Month 9
Pain Occurrence by Age
Measured: Measured at Month 9
Impact of Pain on Functioning and Well-being
Measured: Measured at Month 9
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 startMar 2009
- Primary completionJun 2010
- Study completionDec 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.
- Publications (1)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
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 252 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
- Jeanne Boudreeaux, MD, Principal investigator, Children's Healthcare of AtlantaEllis Neufeld, MD, Principal investigator, Boston Children's HospitalAlexis Thompson, MD, Principal investigator, Children's Memorial Hospital of ChicagoBrigitta Mueller, MD, Principal investigator, Baylor College of Medicine at HoustonDru Foote, RN, NP, Study chair, Children's Hospital and Research Institute of OaklandPatricia Giardina, MD, Principal investigator, Weill Medical College of CornellJanet Kwiatkowski, MD, Principal investigator, Children's Hospital of PhiladelphiaNancy Olivieri, MD, Principal investigator, Toronto General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00872339
- Sponsor's protocol ID
- 639
- Other IDs
- U01HL065238
- Acronym
- Pain
- Status last verified
- Jan 2014
- First submitted
- 30 Mar 2009
- Last updated
- 5 Jun 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Haines D, Martin M, Carson S, Oliveros O, Green S, Coates T, Eile J, Schilling L, Dinu B, Mendoza T, Gerstenberger E, Trachtenberg F, Vichinsky E; Thalassemia Clinical Research Network. Pain in thalassaemia: the effects of age on pain frequency and severity. Br J Haematol. 2013 Mar;160(5):680-7. doi: 10.1111/bjh.12177. Epub 2012 Dec 30.
PubMed 23278768
Study at a glance
From the registry record, last updated by the sponsor on 5 Jun 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Pain in People With Thalassemia
- ClinicalTrials.gov ID
- NCT00872339
- Acronym / sponsor ID
- Pain / 639
- Conditions
- Thalassemia
- Keywords
- Transfusion-Dependent Thalassemia, Non-Transfusion-Dependent Thalassemia
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 252 participants (actual)
- Lead sponsor
- Carelon ResearchUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- Mar 2009
- Primary completion
- Jun 2010 (actual)
- Study completion
- Dec 2010 (actual)
- First posted
- 31 Mar 2009 (estimated)
- Results
- Posted 5 Jun 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. People with thalassemia often experience pain, but the exact sources and prevalence of pain remain unknown. This study will examine the prevalence and severity of pain in people with thalassemia who are treated with regular blood transfusions and people with thalassemia who are not 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. Recent medical advances in treating people with thalassemia who receive regular blood transfusions-a standard procedure that refreshes the healthy red blood supply-have resulted in increased life spans for these people. However, with the extended life spans have come additional issues, including pain. There have been no previous research studies that have examined pain levels in people with thalassemia, and as a result, the sources and prevalence of pain remain unknown. The purpose of this study is to assess the prevalence and severity of pain, common pain sites, and the impact of pain on functioning and well-being in people with thalassemia who receive regular blood transfusions and people with thalassemia who do not receive regular blood transfusions.
This study will enroll people with transfusion-dependant thalassemia and people with non-transfusion-dependant thalassemia. At a baseline study visit, participants will complete a demographic questionnaire and a pain assessment questionnaire. At Months 3, 6, and 9, study researchers will telephone participants to go over the same pain assessment questionnaire again.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| transfusion-dependant | – | –People with transfusion-dependant thalassemia who received at least 8 transfusions in the past year. |
| non-transfusion-dependant | – | –People with non-transfusion-dependant thalassemia who received no transfusions in the past year. |
| intermittently transfused | – | –Intermittently transfused patients- individuals who received at least one but fewer than eight transfusions in the last year |
Who may qualify
- Ages
- 12 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Thalassemia patients who receive regular blood transfusions and those who do not receive regular blood transfusions.
Key inclusion criteria (9 listed)
- Thalassemia, as documented by clinical diagnosis, including the following:
- Participants can be of any race, ethnicity, and either gender.
Key exclusion criteria (3 listed)
- Thalassemia trait (i.e., single recessive B gene mutation, 2 gene alpha mutation) and thalassemia Hgb S, C, or D compound heterozygotes
- Unwillingness or inability to complete the Brief Pain Inventory (BPI) on a quarterly basis
- Has had a successful bone marrow transplant
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
Prevalence of Pain
Time frame: Measured at Month 9
3 secondary outcomes
Common Sites of Pain
Time frame: Measured at Month 9
Pain Occurrence by Age
Time frame: Measured at Month 9
Impact of Pain on Functioning and Well-being
Time frame: Measured at Month 9
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jun 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 23278768Haines D, Martin M, Carson S, Oliveros O, Green S, Coates T, Eile J, Schilling L, Dinu B, Mendoza T, Gerstenberger E, Trachtenberg F, Vichinsky E; Thalassemia Clinical Research Network. Pain in thalassaemia: the effects of age on pain frequency and severity. Br J Haematol. 2013 Mar;160(5):680-7. doi: 10.1111/bjh.12177. Epub 2012 Dec 30.
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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.
Jeanne Boudreeaux, MD principal investigator
Children's Healthcare of Atlanta
Ellis Neufeld, MD principal investigator
Boston Children's Hospital
Alexis Thompson, MD principal investigator
Children's Memorial Hospital of Chicago
8 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 8 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 9 inclusion and 3 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 5 Jun 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 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 5 Jun 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?
Prevalence of Pain (time frame: Measured at Month 9). 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 NCT00872339, which also lists 1 linked publication.
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 5 Jun 2014.