Treatment of Adult Patients With Hemoglobin SC Disease (SCYTHE)

• Status: Terminated
• Trial end date: 2017-11-16
• 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 patients with the most common type of sickle cell, those who are homozygous for the sickle mutation (HbSS), helps individuals 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 AdultsQLTM 3.0 Sickle Cell Disease Module, 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
• Collaborators: Texas Children's Hospital; The University of Texas Health Science Center, Houston
• Treatments: Hydroxyurea

Criteria

Inclusion Criteria:

• Diagnosis of HbSC disease

• Score of 80 or lower on the AdultQLTM 3.0 Sickle Cell Disease Module, or any disease related complication, including, but not limited to, one or more pain events per year, proliferative sickle retinopathy, avascular necrosis, cholelithiasis, or any thrombotic event. If the subject has a score >80, they may still enroll on the trial, and be analyzed for secondary endpoints. They will be excluded from analysis of the primary endpoint.

Exclusion Criteria:

• Failure to meet inclusion criteria

• Hydroxyurea usage in the last 3 months.

• Chronic RBC transfusion therapy

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

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

• Current phlebotomy therapy