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

October 16, 2017 updated by: Emily Riehm Meier, Children's National Research Institute

Reticulocyte as Risk Marker: Targeted Therapy for Infants With Sickle Cell Anemia

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.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Study Type

Interventional

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • District of Columbia
      • Washington, D.C., District of Columbia, United States, 20010
        • Children's National Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months to 1 year (CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

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

Exclusion Criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: PREVENTION
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
PLACEBO_COMPARATOR: Sugar water
Placebo arm
Other: Placebo
Other Names:
  • Sugar water
ACTIVE_COMPARATOR: Hydroxyurea
Treatment Arm
Drug: Hydroxyurea
Other Names:
  • Hydrea

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Sickle Cell Clinical Change from Baseline to Study Completion ( 18 months)
Time Frame: Every 4 weeks for the 18 months study duration
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Children's National Research Institute

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2014

Primary Completion (ACTUAL)

June 1, 2015

Study Completion (ACTUAL)

June 1, 2015

Study Registration Dates

First Submitted

February 4, 2014

First Submitted That Met QC Criteria

March 14, 2014

First Posted (ESTIMATE)

March 18, 2014

Study Record Updates

Last Update Posted (ACTUAL)

October 18, 2017

Last Update Submitted That Met QC Criteria

October 16, 2017

Last Verified

October 1, 2017

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RBTSCA

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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