Hydroxyurea in Young Children With Sickle Cell Anemia - Full Text View

Sponsored by:	Duke University
Information provided by:	Duke University
ClinicalTrials.gov Identifier:	NCT00519701

Purpose

The purpose of this study is to asses prospectively the safety and efficacy of hydroxyurea therapy in children with Sickle cell Anemia between ages 18 months and 5 years, with special emphasis on the ability of hydroxyurea to prevent or reverse chronic organ damage.

Condition	Intervention
Sickle Cell Anemia	Drug: hydroxyurea

Genetics Home Reference related topics: sickle cell disease

MedlinePlus related topics: Anemia Sickle Cell Anemia

Drug Information available for: Hydroxyurea

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Prevention, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	Effects of Hydroxyurea on the Prevention of Chronic Organ Damage in Young Children With Sickle Cell Anemia

Further study details as provided by Duke University:

Primary Outcome Measures:

Transcranial doppler ultrasound velocity [ Time Frame: 2 years ]
Magnetic resonance imaging/angiography [ Time Frame: 2 years ]
Glomerular Filtration Rate [ Time Frame: 2 years ]
Quality of Life [ Time Frame: 2 years ]
Neurocognitive outcomes [ Time Frame: 2 years ]

Secondary Outcome Measures:

Growth parameters [ Time Frame: 2 years ]
Hematological parameters [ Time Frame: 2 years ]

Enrollment:	14
Study Start Date:	April 2002
Study Completion Date:	February 2007

Arms	Assigned Interventions
1: Experimental hydroxyurea	Drug: hydroxyurea

Detailed Description:

Previous studies have shown that hydroxyurea therapy in adults and older children with SCA improves laboratory parameters and ameliorates the clinical severity of disease. Little is known, however, about the effects of hydroxyurea on the chronic organ damage that occurs in patients with SCA and leads to significant morbidity and mortality in young adults. The objectives of this study are to assess the safety and efficacy of HU in young children with SCA and to determine whether HU preserves renal function, reduces transcranial doppler ultrasound (TCD) values, and prevents development of brain ischemia as evidenced by MRI/MRA imaging. In addition, we will evaluate the effects of hydroxyurea on quality of life

Eligibility

Ages Eligible for Study:	18 Months to 5 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Clinical diagnosis of Sickle Cell Anemia (Hb SS or Hb S beta zero-thalassemia)

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00519701

Locations

United States, North Carolina
Duke University Medican Center
Durham, North Carolina, United States, 27710

Sponsors and Collaborators

Duke University

Investigators

Principal Investigator:

Courtney D Thornburg, MD mS

Duke University

More Information

Study ID Numbers:	3297
Study First Received:	August 21, 2007
Last Updated:	August 22, 2007
ClinicalTrials.gov Identifier:	NCT00519701
Health Authority:	United States: Institutional Review Board

Keywords provided by Duke University:

sickle cell anemia
hydroxyurea

Study placed in the following topic categories:

Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hydroxyurea
Hematologic Diseases
Hemoglobinopathies

Anemia
Anemia, Hemolytic
Hemoglobinopathy
Anemia, Sickle Cell
Sickle cell anemia

Additional relevant MeSH terms:

Antisickling Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Therapeutic Uses

Hematologic Agents
Enzyme Inhibitors
Nucleic Acid Synthesis Inhibitors
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 15, 2009