Aspirin Prophylaxis in Sickle Cell Disease
Information source: University of Rochester
ClinicalTrials.gov processed this data on August 20, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Sickle Cell Disease
Intervention: aspirin (Drug)
Phase: Phase 1/Phase 2
Status: Completed
Sponsored by: University of Rochester Official(s) and/or principal investigator(s): Norma B. Lerner, MD, Principal Investigator, Affiliation: University of Rochester
Summary
Neurologic complications secondary to cerebrovascular damage are prevalent in children with
sickle cell disease. These patients experience both clinically overt cerebrovascular
accidents and "silent infarctions" demonstrated by magnetic resonance imaging (MRI). They
are also at risk for neurocognitive abnormalities. We hypothesize that daily, low-dose
aspirin therapy will safely diminish the incidence and progression of cognitive deficits as
well as the predisposition to overt and silent stroke in children with homozygous sickle
cell disease (Hgb SS) or hemoglobin S Beta Zero Thalassemia (Hgb SB-0 Thal). In order to
optimize the design of a future trial to test this hypothesis, we propose a pilot study to
test the safety and tolerability of aspirin in young children with sickle cell disease.
Clinical Details
Official title: Aspirin Prophylaxis in Sickle Cell Disease
Study design: Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Number of Serious Adverse EventsNumber of Adverse Events
Secondary outcome: # of Subjects Recruited Over Time, Screening Failures, Withdrawal Rates;Compliance (Pill Counts & Labs);Changes in Performance on Neurocognitive Tests; Changes in MRI/MRA; Changes in TCD;Incidences of Stroke, Acute Chest Crises, and Pain Crises
Detailed description:
The trial's primary objective is to evaluate the safety and tolerability of daily low-dose
aspirin in children with sickle cell disease. The secondary objectives are to assess (1)
The feasibility of recruiting children with Hgb SS and Hgb S Beta-0 Thalassemia to an
aspirin trial, (2) The level of compliance with aspirin administration in the proposed
patient population, (3) The most useful assessments in a battery of age-appropriate
neurocognitive tests, (4) The feasibility of magnetic resonance imaging (MRI) and magnetic
resonance angiography (MRA) studies and the utility of classification systems for use in
group comparisons, (5) Preliminary data regarding trends in transcranial Doppler (TCD)
ultrasound velocities over time and the validity of using trends for group comparisons, (6)
Preliminary data regarding the effect of aspirin therapy on the incidence of cognitive
deficit, imaging changes, overt stroke, painful crises, and acute chest syndrome. Subjects
will include children between the ages of 2 and 7. 99 years with documented Hgb SS or Hgb S
Beta-0 Thalassemia who are followed at Golisano Children's Hospital at Strong and the
University of Miami. All subjects will receive daily aspirin (about 2. 5 - 5. 1 mg/kg daily).
Subjects will receive therapy for 12 months. There will be careful laboratory and clinical
monitoring every 3-6 months and more frequently if needed. Pre and post treatment clinical
complications, neurocognitive testing, MRI, MRA, and TCD studies will be assessed.
Eligibility
Minimum age: 2 Years.
Maximum age: 7 Years.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- 1. Children ages 2 - 7. 99 years with a diagnosis of Hb SS or Hb Sß0 thalassemia,
documented by hemoglobin electrophoresis and a complete blood count (CBC). 2.
Influenza vaccination during the previous year or intended before the upcoming flu
season. 3. Evidence of past infection with, or immunization against, varicella. 4.
Negative pregnancy tests in girls of childbearing potential. 5. Informed consent
signed by the parent or legal guardian.
Exclusion Criteria:
- 1. Prior history of overt stroke or cerebral hemorrhage. 2. Known history of
allergic reaction to aspirin. 3. History of Reye's syndrome 4. Diagnosis of G-6-PD
deficiency or von Willebrand's disease 5. Prolongation of the bleeding time or
abnormal closure time, prothrombin time (PT), or partial thromboplastin time (PTT).
6. Active gastrointestinal (GI) bleeding or a history of GI bleeding. 7. Hepatic
disease (AST or ALT >2x upper limit of normal, Direct bilirubin > 1. 5 mg/dL) or renal
disease (creatinine >2x upper limit of normal or 2 mg/dl, whichever is smaller). The
exclusion criteria laboratory study ranges have been specified as greater than 2
times the upper limit of normal. 8. Hypertension (BP >95% for age and height). 9.
Current treatment with chronic transfusion therapy. 10. Evidence of hemorrhage on
MRI. 11. A mean TCD velocity > 200 cm/sec. in the middle cerebral artery (MCA) or
internal carotid artery (ICA). 12. Evidence of Moyamoya syndrome on MRA. 13. Evidence
of pregnancy. 14. Evidence of an inability to comply with testing procedures. 15.
Inability to provide informed consent.
Locations and Contacts
Additional Information
Starting date: March 2005
Last updated: November 29, 2011
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