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Assessment of Pain in People With Thalassemia

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
New England Research Institutes
ClinicalTrials.gov Identifier:
NCT00872339
First received: March 30, 2009
Last updated: May 27, 2014
Last verified: January 2014
  Purpose

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.


Condition
Thalassemia

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Assessment of Pain in People With Thalassemia

Resource links provided by NLM:


Further study details as provided by New England Research Institutes:

Primary Outcome Measures:
  • Prevalence of Pain [ Time Frame: Measured at Month 9 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Common Sites of Pain [ Time Frame: Measured at Month 9 ] [ Designated as safety issue: No ]
  • Pain Occurrence by Age [ Time Frame: Measured at Month 9 ] [ Designated as safety issue: No ]
  • Impact of Pain on Functioning and Well-being [ Time Frame: Measured at Month 9 ] [ Designated as safety issue: No ]

Enrollment: 252
Study Start Date: March 2009
Study Completion Date: December 2010
Primary Completion Date: June 2010 (Final data collection date for primary outcome measure)
Groups/Cohorts
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

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.

  Eligibility

Ages Eligible for Study:   12 Years to 90 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Thalassemia patients who receive regular blood transfusions and those who do not receive regular blood transfusions.

Criteria

Inclusion Criteria:

  • Thalassemia, as documented by clinical diagnosis, including the following:

    1. B-thalassemia (intermedia or major)
    2. Hgb H disease
    3. Hgb H with non-deletional mutations (e.g., Hgb H Constant Spring)
    4. E-B-thalassemia
    5. Homozygous alpha thalassemia
    6. Other thalassemic conditions not explicitly excluded
    7. Thalassemia intermedia due to heterozygous B mutation with an alpha excess
  • Participants can be of any race, ethnicity, and either gender.

Exclusion Criteria:

  • 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00872339

Locations
United States, California
Children's Hospital and Research Institute at Oakland
Oakland, California, United States, 94609
United States, Georgia
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30342
United States, Illinois
Children's Memorial Hospital of Chicago
Chicago, Illinois, United States, 60614
United States, Massachusetts
Children's Hospital of Boston
Boston, Massachusetts, United States, 02115
United States, New York
Weill Medical College
New York, New York, United States, 10065
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
Canada, Ontario
Toronto General Hospital
Toronto, Ontario, Canada, M5G 2C4
Sponsors and Collaborators
New England Research Institutes
Investigators
Principal Investigator: Jeanne Boudreeaux, MD Children's Healthcare of Atlanta
Principal Investigator: Ellis Neufeld, MD Children's Hospital Boston
Principal Investigator: Alexis Thompson, MD Children's Memorial Hospital of Chicago
Principal Investigator: Brigitta Mueller, MD Baylor College of Medicine at Houston
Study Chair: Dru Foote, RN, NP Children's Hospital and Research Institute of Oakland
Principal Investigator: Patricia Giardina, MD Weill Medical College of Cornell
Principal Investigator: Janet Kwiatkowski, MD Children's Hospital of Philadelphia
Principal Investigator: Nancy Olivieri, MD Toronto General Hospital
  More Information

Publications:
Responsible Party: New England Research Institutes
ClinicalTrials.gov Identifier: NCT00872339     History of Changes
Other Study ID Numbers: 639, U01HL065238
Study First Received: March 30, 2009
Results First Received: January 2, 2014
Last Updated: May 27, 2014
Health Authority: United States: Federal Government

Keywords provided by New England Research Institutes:
Transfusion-Dependent Thalassemia
Non-Transfusion-Dependent Thalassemia

Additional relevant MeSH terms:
Thalassemia
Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies

ClinicalTrials.gov processed this record on November 27, 2014