Ignite Creation Date:
2024-05-05 @ 10:00 AM
Last Modification Date:
2024-10-26 @ 9:05 AM
Study NCT ID:
NCT00005327
Status:
COMPLETED
Last Update Posted:
2019-07-05
First Post:
2000-05-25
Brief Title:
Prevention of Cerebral Infarction in Sickle Cell Anemia - Comprehensive Sickle Cell Center
Sponsor:
University of Southern California
Organization:
University of Southern California
Study Overview
Official Title:
None
Status:
COMPLETED
Status Verified Date:
2017-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
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 techniquesMRI 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 andor 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
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 techniquesMRI 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 andor 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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| P60HL048484 | NIH | None | httpsreporternihgovquickSearchP60HL048484 |