NCT00661804

Brief 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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
416

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started May 2007

Longer than P75 for all trials

Geographic Reach
3 countries

14 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2007

Completed
12 months until next milestone

First Submitted

Initial submission to the registry

April 16, 2008

Completed
2 days until next milestone

First Posted

Study publicly available on registry

April 18, 2008

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2010

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2011

Completed
Last Updated

November 11, 2011

Status Verified

June 1, 2011

Enrollment Period

3.6 years

First QC Date

April 16, 2008

Last Update Submit

November 9, 2011

Conditions

Thalassemia

Outcome Measures

Primary Outcomes (1)

  • The prevalence and incidence of complications specific to thalassemia and its treatment among participants

    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 throughout the duration of the study

  • Relationships among adherence, quality of life, and complications of thalassemia

    Measured throughout the duration of the study

Study Arms (2)

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.

Eligibility Criteria

Age5 Years+
Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
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.

You may qualify if:

  • 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)
  • 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
  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (14)

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

Location

Children's Hospital of Oakland

Oakland, California, 94609, United States

Location

Stanford Hospital

Stanford, California, 94305, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30342, United States

Location

Children's Memorial Hospital Chicago

Chicago, Illinois, 60614, United States

Location

Children's Hospital Boston

Boston, Massachusetts, 02115, United States

Location

Weill Medical College of Cornell University

New York, New York, 10021, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Southwestern Medical Center at Dallas

Dallas, Texas, 75390, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

British Columbia Children's Hospital

Vancouver, British Columbia, V6H 3V4, Canada

Location

Toronto Sick Kids

Toronto, Ontario, M5G 1X8, Canada

Location

Toronto General Hospital

Toronto, Ontario, M5G 2C4, Canada

Location

Royal Free and University College London Medical School

London, England, WC1E 6BT, United Kingdom

Location

Related Publications (2)

  • 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: 23216870DERIVED

  • 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.

    PMID: 21772051DERIVED

Biospecimen

Retention: SAMPLES WITH DNA

DNA and frozen plasma genetic biorepository

MeSH Terms

Conditions

Thalassemia

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Ellis Neufeld, MD, PhD

    Boston Children's Hospital

    STUDY CHAIR

  • Janet Kwiatkowski, MD

    Children's Hospital of Philadelphia

    STUDY CHAIR

Study Design

Study Type
observational
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER

Study Record Dates

First Submitted

April 16, 2008

First Posted

April 18, 2008

Study Start

May 1, 2007

Primary Completion

December 1, 2010

Study Completion

June 1, 2011

Last Updated

November 11, 2011

Record last verified: 2011-06

Locations

US(10)CA(3)GB(1)