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

NCT ID: NCT02090296

Last Updated: 2017-10-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

2014-02-28

Study Completion Date

2015-06-30

Brief Summary

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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.

Detailed Description

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Conditions

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Sickle Cell Anemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

NONE

Study Groups

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Sugar water

Placebo arm

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Hydroxyurea

Treatment Arm

Group Type ACTIVE_COMPARATOR

Hydroxyurea

Intervention Type DRUG

Interventions

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Hydroxyurea

Intervention Type DRUG

Placebo

Intervention Type OTHER

Other Intervention Names

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Hydrea Sugar water

Eligibility Criteria

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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
Minimum Eligible Age

6 Months

Maximum Eligible Age

12 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Children's National Research Institute

OTHER

Sponsor Role lead

Responsible Party

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Emily Riehm Meier

Attending Physician

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

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Children's National Medical Center

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

Site Status

Countries

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United States

Other Identifiers

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RBTSCA

Identifier Type: -

Identifier Source: org_study_id