Treatment of Hemoglobin SC Disease With Hydroxyurea

• Status: Terminated
• Trial end date: 2017-03-31
• Target enrollment: 0
• Participant gender: All

Summary

Sickle cell disease (SCD), specifically hemoglobin SC disease (HbSC), is a subtype of sickle cell disease with typically higher hemoglobin and milder or later disease complications. Sickle cell disease is a disorder in which red blood cells (RBCs) are abnormally shaped. This can result in painful episodes, serious infections, and damage to body organs. One medication used to treat sickle cell disease is hydroxyurea.

Hydroxyurea therapy offers significant benefits for infants, children, and adolescents with sickle cell anemia. These include a reduction in the frequency of pain crises and acute chest syndrome (inflammation of the lungs). Hydroxyurea has been given to many HbSC patients but HbSC patients were not included in the large clinical trials used to test hydroxyurea in SCD, so less is known about how HbSC patients respond to hydroxyurea.

The purpose of this research study is to see if hydroxyurea, a medication given to many children with the most common type of sickle cell, those who are homozygous for the sickle mutation (HbSS), helps children who have HbSC. The investigators will see if it helps by giving a questionaire when the medication is started, and then every two months at a clinic visit. The questionaire, called the Pediatric Quality of Life Inventory (PedsQL™) Sickle Cell Disease Module version 3.0, measures quality of life. The investigators will also see how hydroxyurea changes laboratory test numbers, and blood thickness.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Baylor College of Medicine
• Collaborator: Texas Children's Hospital
• Treatments: Hydroxyurea

Criteria

Inclusion Criteria:

1. Diagnosis of HbSC disease

2. Score equal or lower than 80 on the PedsQL™ Sickle Cell Disease Module version 3.0

3. Have experienced a sickle cell disease related complication

Exclusion Criteria:

1. Failure to meet inclusion criteria.

2. Hydroxyurea usage in the last 3 months.

3. Chronic RBC transfusion therapy.

4. Packed red blood cell transfusion in the last 3 months (temporary exclusion).

5. Pregnancy, or refusal to use medically effective birth control if female and sexually active.