Stroke Prevention in Sickle Cell Anemia (STOP 1)

December 21, 2015 updated by: Augusta University
To reduce episodes of first time stroke by 75 percent in children with sickle cell anemia by the administration of prophylactic transfusion therapy.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

BACKGROUND:

Stroke, occurring in about 10 percent of pediatric patients with sickle cell disease, is one of the most devastating complications, with a high recurrence rate after the first episode. Several non-randomized studies have shown reduction in stroke recurrence when periodic blood transfusions are administered to maintain hemoglobin S under 30 percent. Periodic blood transfusions are associated with significant risks of iron overload and other complications and must be accompanied by parenteral iron chelation therapy. However, this has become a standard of care for prevention of recurrent stroke in SS children. Thus, a randomized trial of blood transfusion for secondary prevention would not be feasible because it would be considered unethical. Based on various studies, the recurrence rate is reduced from 46 to 67 percent to approximately 7 percent on transfusion therapy. Because most stroke patients are left with some neurological deficit, and face a lifetime of disability, primary prevention would have a significant impact on the management of patients. However, because of complications of blood transfusions, the hypothesis should be proven by a randomized clinical trial.

A primary prevention trial had not been possible because an acceptable means of detecting those children at risk of stroke was not available. The advent of TCD to identify arterial abnormalities for the prediction of stroke has provided a means of detection. TCD abnormalities have a high specificity (100 percent) and high sensitivity (90 percent) for detecting angiographically proven narrowing of arterial diameter. Thus, TCD examination of the basal cerebral arteries is predictive of who will develop a stroke.

DESIGN NARRATIVE:

Randomized, Phase III, multicenter. Approximately 3,000 children from 12 clinics were screened with transcranial Doppler (TCD). A total of 130 were randomized to receive either standard supportive care or periodic blood transfusions if they were found to be at high risk of stroke on the basis of elevated cerebral blood flow as measured by TCD screening tests. Primary endpoints included clinically evident symptoms of cerebral infarction with consistent findings on magnetic resonance imaging (MRI), and/or symptomatic intracranial hemorrhage. Secondary endpoints included asymptomatic brain lesions detected by MRI in brain areas not involved in primary endpoints. Hematologic characteristics of the high risk group were analyzed and serum and DNA samples frozen for future analysis. Recruitment ended in October 1997 with the accrual of 130 subjects. The clinical phase ended in 1999.

Study Type

Interventional

Phase

  • Phase 3

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

7 months to 14 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Pediatric patients, ages 24 months to 16 years, with sickle cell anemia or S-beta zero thalassemia.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Randomized

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Augusta University

Collaborators

National Heart, Lung, and Blood Institute (NHLBI)

Investigators

  • Donald Brambilla, New England Research Institute Inc.

Publications and helpful links

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General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

July 1, 1994

Study Completion

August 1, 2000

Study Registration Dates

First Submitted

October 27, 1999

First Submitted That Met QC Criteria

October 27, 1999

First Posted (Estimate)

October 28, 1999

Study Record Updates

Last Update Posted (Estimate)

December 23, 2015

Last Update Submitted That Met QC Criteria

December 21, 2015

Last Verified

November 1, 2005

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 312 (CTEP)
  • U01HL052193 (U.S. NIH Grant/Contract)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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