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Hydroxyurea in Young Children With Sickle Cell Anemia

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ClinicalTrials.gov Identifier: NCT00519701
Recruitment Status : Completed
First Posted : August 23, 2007
Last Update Posted : August 23, 2007
Sponsor:
Information provided by:
Duke University

Brief Summary:
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 or disease Intervention/treatment Phase
Sickle Cell Anemia Drug: hydroxyurea Not Applicable

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

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 14 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: Effects of Hydroxyurea on the Prevention of Chronic Organ Damage in Young Children With Sickle Cell Anemia
Study Start Date : April 2002
Actual Study Completion Date : February 2007

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia
Drug Information available for: Hydroxyurea

Arm Intervention/treatment
Experimental: 1
hydroxyurea
Drug: hydroxyurea



Primary Outcome Measures :
  1. Transcranial doppler ultrasound velocity [ Time Frame: 2 years ]
  2. Magnetic resonance imaging/angiography [ Time Frame: 2 years ]
  3. Glomerular Filtration Rate [ Time Frame: 2 years ]
  4. Quality of Life [ Time Frame: 2 years ]
  5. Neurocognitive outcomes [ Time Frame: 2 years ]

Secondary Outcome Measures :
  1. Growth parameters [ Time Frame: 2 years ]
  2. Hematological parameters [ Time Frame: 2 years ]


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Ages Eligible for Study:   18 Months to 5 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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

ClinicalTrials.gov Identifier: NCT00519701     History of Changes
Other Study ID Numbers: 3297
First Posted: August 23, 2007    Key Record Dates
Last Update Posted: August 23, 2007
Last Verified: August 2007

Keywords provided by Duke University:
sickle cell anemia
hydroxyurea

Additional relevant MeSH terms:
Anemia
Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Hydroxyurea
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors