Treatment of Adult Patients With Hemoglobin SC Disease (SCYTHE)

NCT02640573 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: 37290 IM-SCYTHE
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 2

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 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.

Conditions

Hemoglobin SC Disease

Outcome Measures

Primary Outcomes (1)

• Fetal Hemoglobin Response to HU

  Change in HbF on hydroxyurea from baseline

  Baseline to 6 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.

Drug: Hydroxyurea

Interventions

Hydroxyurea DRUG

Treat symptomatic HbSC patients to MTD on hydroxyurea, and assess for clinical improvement using the AdultsQLTM 3.0 Sickle Cell Disease Module after 6 months at MTD, compared to entrance scores

Hydroxurea

Eligibility Criteria

• Age: 18 Years - 69 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

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.
• Current phlebotomy therapy

Sponsors & Collaborators

• Baylor College of Medicine: lead
• The University of Texas Health Science Center, Houston: collaborator

Study Sites (2)

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

University of Texas Houston

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
• Organization: Baylor College of Medicine

vsheehan@bcm.edu

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: December 17, 2015
• First Posted: December 29, 2015
• Study Start: October 12, 2015
• Primary Completion: November 16, 2017
• Study Completion: November 16, 2017
• Last Updated: January 27, 2020
• Results First Posted: January 27, 2020

Record last verified: 2020-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)