Treatment of Hemoglobin SC Disease With Hydroxyurea (SCYTHE)
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|ClinicalTrials.gov Identifier: NCT02336373|
Recruitment Status : Terminated (low enrollment)
First Posted : January 13, 2015
Results First Posted : September 9, 2020
Last Update Posted : September 9, 2020
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.
|Condition or disease||Intervention/treatment||Phase|
|Hemoglobin SC Disease||Drug: hydroxyurea||Phase 2|
To be eligible to participate in this study, patients must have HbSC disease, have experienced a sickle cell disease related complication, or have a score of 80 or lower on the PedsQL™ Sickle Cell Disease Module version 3.0. This questionnaire will be offered to all patients with HbSC seen in our clinic that consent to this study. If the patient is sexually active, they will be offered birth control. If the patient chooses not to initiate effective birth control, they will be tested at their scheduled vist with a urine pregnancy test. If the patient becomes pregnant they will be removed from the study.
The maximum time patients will be on the study is 12 months after starting hydroxyurea therapy, with an option to participate in a 2 year observation study following the end of the study.
Patients will be assessed in the clinic every two months after starting treatment. Hydroxyurea will be started at 10 mg/kg/day, and increased by 5 mg/kg/day at eight week intervals if needed to reach a maximum tolerated dose (MTD). The most common side effect of the drug is a drop in infection fighting cells, or white blood cells, so the medication will be started at a low dose and the dose will be increased only if it is safe to do so.
Patients will be asked to allow the investigators to review information from their medical records at the start of the study, and throughout the study. If the patient would like to participate in the two year follow-up, their records will be reviewed during that period as well.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||32 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||SC Youth Treatment With Hydroxyurea Effects|
|Study Start Date :||December 2014|
|Actual Primary Completion Date :||February 2017|
|Actual Study Completion Date :||March 31, 2017|
Initiate hydroxyurea at 10 mg/kg daily and escalate hydroxyurea dose by 5 mg/kg/day every 8 weeks up to a maximum dose of 35 mg/kg/day if blood counts meet escalation criteria on at least 2 blood tests over eight weeks prior to dose increase.
Treat symptomatic HbSC patients to MTD on hydroxyurea, and assess for clinical improvement using the PedsQL™ Sickle Cell Disease Module version 3.0 after 6 months at MTD, compared to entrance scores
- Change in PedsQL SCDM [ Time Frame: 6 months ]
Mean change in PedsQL™ Sickle Cell Disease Module version 3.0 score after achieving MTD compared with baseline.
PedsQL™ Sickle Cell Disease Module version 3.0 scores are on a 100 point scale ranging from 0 to 100 with higher values indicating better quality of life.
PedsQL: pediatric qulaity of life
- Change in HVR at 45s-1 [ Time Frame: up to 7 months ]Change in hematocrit to viscosity ratio (HVR) at 45s-1 at follow-up versus baseline. This is a measure of oxygen carrying capacity, as a higher hematocrit with lower viscosity indicates ability to deliver oxygen without slowed blood flow due to high viscosity or blood thickness. Higher values are associated with improvement.
- Change in HVR at 225s-1 [ Time Frame: up to 7 months ]Change in hematocrit viscosity ratio at 225s
- DRBC [ Time Frame: up to 7 months ]Change in percent dense red blood cells
- Change in HbF [ Time Frame: up to 7 months ]Change in fetal hemaglobin
- Change in MCV [ Time Frame: up to 7 months ]Change in mean corpuscular volume
- Change in MCHC [ Time Frame: up to 7 months ]Change in mean corpuscular hemoglobin concentration
- Change in Hb [ Time Frame: up to 7 months ]Change in hemoglobin
- Change in ARC [ Time Frame: up to 7 months ]Change in absolute reticulocyte count
- Change in ANC [ Time Frame: up to 7 months ]Change in absolute neutrophil count
- Change in LDH [ Time Frame: up to 7 months ]Change in lactate dehydrogenase
- Change in UB Levels [ Time Frame: up to 7 months ]Change in unconjugated bilirubin levels
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02336373
|United States, Texas|
|Texas Children's Hospital|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Vivien Sheehan, MD||Baylor College of Medicine|