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

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ClinicalTrials.gov Identifier: NCT02090296
Recruitment Status : Withdrawn (Participant withdrew consent)
First Posted : March 18, 2014
Last Update Posted : October 18, 2017
Sponsor:
Information provided by (Responsible Party):
Emily Riehm Meier, Children's National Research Institute

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.

Condition or disease Intervention/treatment Phase
Sickle Cell Anemia Drug: Hydroxyurea Other: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: Reticulocyte as Risk Marker: Targeted Therapy for Infants With Sickle Cell Anemia
Study Start Date : February 2014
Actual Primary Completion Date : June 2015
Actual Study Completion Date : June 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia
Drug Information available for: Hydroxyurea

Arm Intervention/treatment
Placebo Comparator: Sugar water
Placebo arm
Other: Placebo
Other Name: Sugar water

Active Comparator: Hydroxyurea
Treatment Arm
Drug: Hydroxyurea
Other Name: Hydrea




Primary Outcome Measures :
  1. 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.



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Ages Eligible for Study:   6 Months to 12 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02090296


Locations
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United States, District of Columbia
Children's National Medical Center
Washington, D.C., District of Columbia, United States, 20010
Sponsors and Collaborators
Children's National Research Institute
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Responsible Party: Emily Riehm Meier, Attending Physician, Children's National Research Institute
ClinicalTrials.gov Identifier: NCT02090296    
Other Study ID Numbers: RBTSCA
First Posted: March 18, 2014    Key Record Dates
Last Update Posted: October 18, 2017
Last Verified: October 2017
Additional relevant MeSH terms:
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Anemia
Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Hydroxyurea
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors