Treatment of Hemoglobin SC Disease With Hydroxyurea

NCT02336373 | Terminated Phase 2 Results DMC

• 1 other identifier: H-35010 SCYTHE
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

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.

Conditions

Hemoglobin SC Disease

Keywords

Hemoglobin SC disease; hydroxyurea; quality of life; viscosity; red cell density

Outcome Measures

Primary Outcomes (1)

• Change in PedsQL SCDM

  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

  6 months

Secondary Outcomes (11)

• Change in HVR at 45s-1

  up to 7 months

• Change in HVR at 225s-1

  up to 7 months

• DRBC

  up to 7 months

• Change in HbF

  up to 7 months

• Change in MCV

  up to 7 months

Study Arms (1)

Hydroxurea

EXPERIMENTAL

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

Interventions

hydroxyurea DRUG

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

Also known as: Hydrea, Droxia

Hydroxurea

Eligibility Criteria

• Age: 5 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosis of HbSC disease
• Score equal or lower than 80 on the PedsQL™ Sickle Cell Disease Module version 3.0
• Have experienced a sickle cell disease related complication

You may not qualify if:

• Hydroxyurea usage in the last 3 months.
• Chronic RBC transfusion therapy.
• Pregnancy, or refusal to use medically effective birth control if female and sexually active.

Sponsors & Collaborators

• Baylor College of Medicine: lead

Study Sites (1)

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Hemoglobin SC Disease

Interventions

Hydroxyurea

Condition Hierarchy (Ancestors)

Anemia, Sickle Cell; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Urea; Amides; Organic Chemicals

Results Point of Contact

• Title: Vivien Sheehan, MD, PhD
• Organization: Baylor College of Medicine

vxsheeha@txch.org

(832) 824-4459

Study Officials

• Vivien Sheehan, MD

  Baylor College of Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: January 8, 2015
• First Posted: January 13, 2015
• Study Start: December 1, 2014
• Primary Completion: February 1, 2017
• Study Completion: March 31, 2017
• Last Updated: September 9, 2020
• Results First Posted: September 9, 2020

Record last verified: 2020-09

Locations

US (1)