Risk-based Therapy for Sickle Cell Anemia: A Feasibility Study

NCT02090296 | Withdrawn Phase 2 DMC

• 1 other identifier: RBTSCA
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

Sickle cell anemia (SCA) patients experience organ damage that begins at an early age and results in significant morbidity and early mortality. Although all SCA patients share the same genetic mutation, the clinical complications are highly variable with some patients experiencing frequent and severe complications, while others have few serious complications. If SCA severity could be predicted early in life, those patients at greatest risk for complications could receive treatment prior to the onset of organ damage. No general SCA severity predictor or one that can be informative early in life exists. The investigators preliminary research has identified the absolute reticulocyte count (ARC) as a potential early predictive risk marker for SCA complications in pediatric patients. A higher ARC between ages 2 and 6 months of age is associated with an increased risk of hospitalization in the first 3 years of life; the mean ARC for the 36 patients who were hospitalized for SCA complications was significantly higher than that of the remaining 23 in those who were not hospitalized. Moreover, total hospitalizations were nearly three times higher by age 2 years in those infants who had an ARC of \> 200 than for those infants whose ARC was \<200. The proposed study will determine if ARC can be used as a risk-stratifier in asymptomatic infants with SCA and ascertain its value in targeting hydroxyurea therapy to those infants at highest risk of SCA sequelae.

Conditions

Sickle Cell Anemia

Outcome Measures

Primary Outcomes (1)

• Sickle Cell Clinical Change from Baseline to Study Completion ( 18 months)

  Participants will be have a study visit at baseline and then every month for the 18 month duration of the study. Study visits will include a physical exam, laboratory monitoring and interval history to assess the change (if any) every 4 weeks of sickle cell complications.

  Every 4 weeks for the 18 months study duration

Study Arms (2)

Sugar water

PLACEBO COMPARATOR

Placebo arm

Other: Placebo

Hydroxyurea

ACTIVE COMPARATOR

Treatment Arm

Drug: Hydroxyurea

Interventions

Hydroxyurea DRUG

Also known as: Hydrea

Hydroxyurea

Placebo OTHER

Also known as: Sugar water

Sugar water

Eligibility Criteria

• Age: 6 Months - 12 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• ages 6-12 months
• Sickle cell anemia (HbSS)
• steady state absolute reticulocyte count between 2-6 months is available in the medical record

You may not qualify if:

• receiving hydroxyurea or chronic monthly blood transfusions
• patient enrolled in preliminary study

Sponsors & Collaborators

• Children's National Research Institute: lead

Study Sites (1)

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Hydroxyurea

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Attending Physician

Study Record Dates

• First Submitted: February 4, 2014
• First Posted: March 18, 2014
• Study Start: February 1, 2014
• Primary Completion: June 1, 2015
• Study Completion: June 1, 2015
• Last Updated: October 18, 2017

Record last verified: 2017-10

Locations

US (1)