Aspirin Prophylaxis in Sickle Cell Disease - Full Text View

Sponsors and Collaborators:	University of Rochester University of Miami Bayer National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by:	University of Rochester
ClinicalTrials.gov Identifier:	NCT00178464

Purpose

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.

Condition	Intervention	Phase
Sickle Cell Disease	Drug: aspirin	Phase I Phase II

Genetics Home Reference related topics: beta thalassemia sickle cell disease

MedlinePlus related topics: Sickle Cell Anemia Thalassemia

Drug Information available for: Acetylsalicylic acid

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Active Control, Single Group Assignment, Safety Study
Official Title:	Aspirin Prophylaxis in Sickle Cell Disease

Further study details as provided by University of Rochester:

Primary Outcome Measures:

Ability to complete the trial on the originally assigned dosage of study medication with or without dosage modification or suspension; Occurrence of individual adverse events, intracranial hemorrhage &/or Reyes syndrome

Secondary Outcome Measures:

# of subjects recruited over time, screening failures, withdrawl 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

Estimated Enrollment:	40
Study Start Date:	March 2005

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

Ages Eligible for Study:	2 Years to 7 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00178464

Contacts

Contact: Norma B. Lerner, MD, MPH

585-275-2981

Norma_Lerner@urmc.rochester.edu

Locations

United States, New York
University of Rochester Medical Center	Recruiting
Rochester, New York, United States, 14642
Contact: Norma B Lerner, MD, MPH 585-275-2981 Norma_Lerner@urmc.rochester.edu
Principal Investigator: Norma B. Lerner, MD

Sponsors and Collaborators

University of Rochester

University of Miami

Bayer

National Institute of Neurological Disorders and Stroke (NINDS)

Investigators

Principal Investigator:

Norma B. Lerner, MD

University of Rochester

More Information

Responsible Party:	University of Rochester Medical Center ( Norma Lerner, MD )
Study ID Numbers:	09661, 1 RO1 NS045948-01
Study First Received:	September 13, 2005
Last Updated:	July 31, 2008
ClinicalTrials.gov Identifier:	NCT00178464
Health Authority:	United States: Food and Drug Administration; United States: Institutional Review Board

Keywords provided by University of Rochester:

sickle cell disease
hemoglobin SS disease
hemoglobin S Beta-0 Thalassemia
silent infarction in sickle cell disease

overt stroke in sickle cell disease
aspirin
transcranial Doppler ultrasound
neurocognitive testing

Study placed in the following topic categories:

Cerebral Infarction
Hematologic Diseases
Stroke
Anemia
Anemia, Hemolytic
Thalassemia
Sickle cell anemia

Anemia, Hemolytic, Congenital
Aspirin
Genetic Diseases, Inborn
Hemoglobinopathies
Infarction
Hemoglobinopathy
Anemia, Sickle Cell

Additional relevant MeSH terms:

Anti-Inflammatory Agents
Molecular Mechanisms of Pharmacological Action
Cyclooxygenase Inhibitors
Hematologic Agents
Physiological Effects of Drugs
Enzyme Inhibitors
Fibrinolytic Agents
Cardiovascular Agents
Pharmacologic Actions
Fibrin Modulating Agents

Analgesics, Non-Narcotic
Sensory System Agents
Therapeutic Uses
Platelet Aggregation Inhibitors
Anti-Inflammatory Agents, Non-Steroidal
Analgesics
Peripheral Nervous System Agents
Antirheumatic Agents
Central Nervous System Agents

ClinicalTrials.gov processed this record on January 14, 2009