Hydroxyurea in Young Children With Sickle Cell Anemia

• Sponsor: Duke University (Other)
• Overall Status: Completed
• CT.gov ID: NCT00519701
• Collaborator: (none)
• Enrollment: 14
• Location: 1
• Arm: 1
• Duration (Months): 58.1
• Patients Per Site Per Month: 0.2

Study Details

Study Description

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	N/A

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 Design

• Study Type: Interventional
• 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: Apr 1, 2002
• Actual Study Completion Date: Feb 1, 2007

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 1 hydroxyurea	Drug: hydroxyurea

Outcome Measures

Primary Outcome Measures

1. Transcranial doppler ultrasound velocity [2 years]

2. Magnetic resonance imaging/angiography [2 years]

3. Glomerular Filtration Rate [2 years]

4. Quality of Life [2 years]

5. Neurocognitive outcomes [2 years]

Secondary Outcome Measures

1. Growth parameters [2 years]

2. Hematological parameters [2 years]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Months to 5 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• ClinicalTrials.gov Identifier: NCT00519701
• Other Study ID Numbers: 3297
• First Posted: Aug 23, 2007
• Last Update Posted: Aug 23, 2007
• Last Verified: Aug 1, 2007

• Keywords provided by , ,: sickle cell anemia; hydroxyurea
• Additional relevant MeSH terms: Anemia; Anemia, Sickle Cell; Hydroxyurea

Study Results

No Results Posted as of Aug 23, 2007