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Hydroxyurea in Young Children With Sickle Cell Anemia

This study has been completed.
Sponsor:
Information provided by:
Duke University
ClinicalTrials.gov Identifier:
NCT00519701
First received: August 21, 2007
Last updated: August 22, 2007
Last verified: August 2007
  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

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Prevention
Official Title: Effects of Hydroxyurea on the Prevention of Chronic Organ Damage in Young Children With Sickle Cell Anemia

Resource links provided by NLM:


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
Experimental: 1
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

No publications provided

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

Keywords provided by Duke University:
sickle cell anemia
hydroxyurea

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

ClinicalTrials.gov processed this record on November 25, 2014