Prevention of Cerebral Infarction in Sickle Cell Anemia - Comprehensive Sickle Cell Center

Sponsor

University of Southern California (Other)

Overall Status

Completed

CT.gov ID

NCT00005327

Collaborator

National Heart, Lung, and Blood Institute (NHLBI) (NIH)

119

Actual Duration (Months)

Study Details

Study Description

Brief Summary

To conduct a prospective study aimed at the early detection and treatment of cerebral vascular disease prior to irreversible brain injury in young children with sickle cell anemia (SCA).

Condition or Disease	Intervention/Treatment	Phase
Anemia, Sickle Cell Blood Disease Cerebrovascular Disorders Cerebrovascular Accident

Detailed Description

BACKGROUND:

Cerebral infarction is a major contributor to childhood morbidity and mortality in sickle cell anemia (SCA)

DESIGN NARRATIVE:

The investigators tested the hypotheses that young children with SCA experienced a variable period of asymptomatic progressive central nervous system (CNS) vasculopathy prior to cerebral infarction; that pre-infarct CNS vasculopathy could be identified by non-invasive imaging techniques:MRI, magnetic resonance angiography (MRA), and transcranial Doppler (TCD); and that therapeutic intervention at this stage of the disease could significantly reduce the subsequent occurrence of cerebral infarction. MRI, MRA, TCD, and standardized neurologic and psychometric examinations were performed yearly in a cohort of homozygous Hb SS children enrolled at 2-4 years of age. Subjects without MRI evidence of cerebral infarction who had significant cerebral vasculopathy (cerebral arterial stenosis on MRA and/or elevated blood flow velocity on TCD) were randomized to receive either no therapy or chronic transfusion therapy, in order to determine the risk of subsequent cerebral infarction in untreated subjects with these abnormalities, and the extent to which transfusion therapy could significantly reduce the risk. Subjects with evidence of prior cerebral infarction on MRI, whether symptomatic or asymptomatic, were randomized to receive either chronic transfusion therapy alone ('standard therapy') or chronic transfusion therapy plus ticlopidine, in order to determine whether ticlopidine could significantly increase the efficacy of standard therapy in preventing recurrent cerebral infarction in SCA. Subjects with prior cerebral infarction were also offered the option of bone marrow transplantation if an HLA-identical non-SS sibling donor was available.

Study Design

Study Type:

Observational

Actual Study Start Date :

Apr 1, 1993

Actual Primary Completion Date :

Mar 1, 1998

Actual Study Completion Date :

Mar 1, 2003

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 100 Years

Sexes Eligible for Study:

Male

Accepts Healthy Volunteers:

No eligibility criteria

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

University of Southern California
National Heart, Lung, and Blood Institute (NHLBI)

Investigators

: Darleen Powars, University of Southern California

Study Documents (Full-Text)

None provided.

More Information

Publications

Responsible Party:

University of Southern California

ClinicalTrials.gov Identifier:

NCT00005327

Other Study ID Numbers:

4117
P60HL048484

First Posted:

May 26, 2000

Last Update Posted:

Jul 5, 2019

Last Verified:

Mar 1, 2017

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Stroke

Cerebrovascular Disorders

Anemia

Anemia, Sickle Cell

Hematologic Diseases

Study Results

No Results Posted as of Jul 5, 2019