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Transcranial Doppler (TCD) Ultrasound of Subjects Enrolled in BABY HUG - Ancillary to BABY HUG

This study has been completed.
Sponsor:
Information provided by:
National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier:
NCT00059293
First received: April 22, 2003
Last updated: July 23, 2008
Last verified: July 2008
  Purpose

To use transcranial Doppler (TCD) ultrasound to detect stroke risk in children with sickle cell disease.


Condition
Blood Disease
Anemia, Sickle Cell
Cerebrovascular Disorders
Cerebral Embolism and Thrombosis

Study Type: Observational

Resource links provided by NLM:


Further study details as provided by National Heart, Lung, and Blood Institute (NHLBI):

Study Start Date: September 2002
Study Completion Date: July 2008
Primary Completion Date: July 2008 (Final data collection date for primary outcome measure)
Detailed Description:

BACKGROUND:

Cerebral vasculopathy and stroke are among the most serious complications of sickle cell disease. Not every patient will develop central nervous system disease but most children have some evidence of cognitive impairment and about 10% will have overt stroke if this is not forestalled by transcranial Doppler (TCD) screening, identification of individuals at greatest risk by estimating maximal cerebral blood flow and commencing prophylactic transfusion. The basis of these recommendations is studies in children over age 2-years where stroke is most prevalent. Transfusion is not without its own serious and considerable risks so that other measures for delaying or preventing cerebral vasculopathy would be valuable. Hydroxyurea (HU) can reduce both morbidity and mortality of sickle cell anemia in adults; most likely the same pertains in children. Unknown are the effects of HU in infants and the youngest children and whether its successful use can reduce the risks of sickle cerebrovascular disease. If HU is associated with reduced TCD velocity, and if it is safe in infants, the medical implications would be high.

Baby TCD is an ancillary study to the initial phase of BABY HUG, a multicenter clinical trial funded by the NHLBI. In BABY HUG, infants (12-17 months old) with sickle cell disease (SCD) will be randomized to either hydroxyurea (HU) or placebo and followed for 24 months. There is broad interest in the effect of hydroxyurea on the central nervous system in the children being studied. Stroke occurs after age 1 year and there is evidence that the process of cerebrovascular disease starts very early in some patients. The Cooperative Study of Sickle Cell Disease documented a high risk of incident stroke in the 2-6 year age group. The study is in response to an initiative on Ancillary Studies in Heart, Lung, and Blood Disease Trials released in June, 2000.

DESIGN NARRATIVE:

The study will perform TCD on the 40 children in BABY HUG at baseline and at the end of study. TCD has proven very useful in estimation of stroke risk in children with SCD ages 2-16 years, and is at the center of a paradigm of primary stroke prevention recommended by the National Heart, Lung, and Blood Institute based on the STOP study. If stroke risk could be detected at an earlier stage (i.e., younger than the 2-16 year-old subjects in STOP), then physicians would be able to provide preventive treatment at an earlier stage of the disease. TCD will be performed before and after 24 months of treatment. TCD performance and reading will be centrally controlled and STOP-consistent. The investigators have experience in TCD of infants, the age targeted by the parent study. TCD will be compared between HU and control groups, before and after treatment. In addition, the investigators have access to data from over 400 children in the 2-4 year age range from the STOP screening program performed in the mid- to late 1990s. Since these patients were not on HU or transfusion this dataset will be used to give an estimate of the prevalence of high stroke risk without HU treatment, and can be compared to the HU group in Baby HUG, adding another important comparative dimension to the analyses. Although the sample size is purposely small as designed by the parent study, large differences in the prevalence of high-risk patients at the end of treatment could be detected by Baby TCD.

  Eligibility

Ages Eligible for Study:   1 Year to 2 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

No eligibility criteria

  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: NCT00059293

Sponsors and Collaborators
Investigators
Investigator: Robert Adams Georgia Regents University
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00059293     History of Changes
Other Study ID Numbers: 1208
Study First Received: April 22, 2003
Last Updated: July 23, 2008
Health Authority: United States: Federal Government

Additional relevant MeSH terms:
Anemia, Sickle Cell
Cerebrovascular Disorders
Embolism and Thrombosis
Hematologic Diseases
Intracranial Embolism and Thrombosis
Thrombosis
Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Brain Diseases
Cardiovascular Diseases
Central Nervous System Diseases
Genetic Diseases, Inborn
Hemoglobinopathies
Nervous System Diseases
Thromboembolism
Vascular Diseases

ClinicalTrials.gov processed this record on November 25, 2014