Hydroxyurea in Young Children With Sickle Cell Anemia

Duke University

Status: Completed

Conditions: Sickle Cell Anemia

Treatments: Drug: hydroxyurea

Study type: Interventional

Funder types: Other

Identifiers

NCT00519701

3297

Details and patient eligibility

About

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.

Full 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

Enrollment: 14 patients

Sex: All

Ages: 18 months to 5 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:

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

Trial design

Primary purpose: Prevention

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

---

14 participants in 1 patient group

1

Experimental group

Description:

hydroxyurea

Treatment:

Drug: hydroxyurea