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Treatment of Hemoglobin SC Disease With Hydroxyurea (SCYTHE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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
Sponsor:
Information provided by (Responsible Party):
Vivien Sheehan, Baylor College of Medicine

Brief 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.


Condition or disease Intervention/treatment Phase
Hemoglobin SC Disease Drug: hydroxyurea Phase 2

Detailed Description:

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 32 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

Drug Information available for: Hydroxyurea

Arm Intervention/treatment
Experimental: Hydroxurea
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.
Drug: hydroxyurea
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
Other Names:
  • Hydrea
  • Droxia




Primary Outcome Measures :
  1. 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



Secondary Outcome Measures :
  1. 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.

  2. Change in HVR at 225s-1 [ Time Frame: up to 7 months ]
    Change in hematocrit viscosity ratio at 225s

  3. DRBC [ Time Frame: up to 7 months ]
    Change in percent dense red blood cells

  4. Change in HbF [ Time Frame: up to 7 months ]
    Change in fetal hemaglobin

  5. Change in MCV [ Time Frame: up to 7 months ]
    Change in mean corpuscular volume

  6. Change in MCHC [ Time Frame: up to 7 months ]
    Change in mean corpuscular hemoglobin concentration

  7. Change in Hb [ Time Frame: up to 7 months ]
    Change in hemoglobin

  8. Change in ARC [ Time Frame: up to 7 months ]
    Change in absolute reticulocyte count

  9. Change in ANC [ Time Frame: up to 7 months ]
    Change in absolute neutrophil count

  10. Change in LDH [ Time Frame: up to 7 months ]
    Change in lactate dehydrogenase

  11. Change in UB Levels [ Time Frame: up to 7 months ]
    Change in unconjugated bilirubin levels



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   5 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

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


Locations
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United States, Texas
Texas Children's Hospital
Houston, Texas, United States, 77030
Sponsors and Collaborators
Baylor College of Medicine
Investigators
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Principal Investigator: Vivien Sheehan, MD Baylor College of Medicine
  Study Documents (Full-Text)

Documents provided by Vivien Sheehan, Baylor College of Medicine:
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Responsible Party: Vivien Sheehan, Assistant Professor, Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT02336373    
Other Study ID Numbers: H-35010 SCYTHE
First Posted: January 13, 2015    Key Record Dates
Results First Posted: September 9, 2020
Last Update Posted: September 9, 2020
Last Verified: September 2020
Keywords provided by Vivien Sheehan, Baylor College of Medicine:
Hemoglobin SC disease
hydroxyurea
quality of life
viscosity
red cell density
Additional relevant MeSH terms:
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Hemoglobin SC Disease
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Hydroxyurea
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors