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Long Term Effects of Hydroxyurea Therapy in Children With Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT00305175
Recruitment Status : Completed
First Posted : March 21, 2006
Last Update Posted : July 28, 2020
Sponsor:
Collaborator:
Children's Hospital Medical Center, Cincinnati
Information provided by (Responsible Party):
St. Jude Children's Research Hospital

Study Description
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Brief Summary:

The primary objectives of this prospective, observational study are (1) to describe the long-term cellular, molecular, and clinical effects of hydroxyurea therapy in sickle cell disease, and (2) to perform hydroxyurea pharmacokinetics studies.

This study will follow sickle cell patients being treated with hydroxyurea for a long period of time to evaluate the long-term cellular and molecular effects of the drug on the patients' body. This study will consist of two patient groups. One group will be made up of patients who have received hydroxyurea therapy before entering the study. The second group will be made up of patients who have not received hydroxyurea before study entry.


Condition or disease
Sickle Cell Disease

Detailed Description:

Many years of study have documented the severe effects of sickle cell disease. Some of these effects include hemolysis (the break down of red blood cells), blockages in the blood vessels, and damage to the organs systems of the body. Hydroxyurea, which is given by mouth, is used to effectively prevent blockages in the blood vessels of patients with sickle cell disease. The hydroxyurea dosage varies and the responses of the body to this drug are not well understood.

This study will follow sickle cell patients being treated with hydroxyurea for a long period of time to evaluate the long-term cellular and molecular effects of the drug on the patients' body. This study will consist of two patient groups. One group will be made up of patients who have received hydroxyurea therapy before entering the study (Old Cohort). The second group will be made up of patients who have not received hydroxyurea before study entry (New Cohort).

This is not a therapeutic drug trial. Subjects for this study will receive hydroxyurea therapy for accepted clinical indications, and will be treated per best clinical management using treatment algorithms established at St. Jude Children's Research Hospital and other pediatric sickle cell programs across the United States. Hydroxyurea therapy data (such as dosing and duration of therapy) will not be dictated by this study, but will be collected to correlate with long-term outcomes. Hydroxyurea dose escalation to a stable MTD will occur according to published guidelines.

Study Design
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Study Type : Observational
Actual Enrollment : 260 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Long Term Effects of Hydroxyurea Therapy in Children With Sickle Cell Disease
Actual Study Start Date : March 3, 2006
Actual Primary Completion Date : April 23, 2015
Actual Study Completion Date : April 23, 2015

Resource links provided by the National Library of Medicine

Drug Information available for: Hydroxyurea

Groups and Cohorts
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Group/Cohort
Patients With Prior Hydroxyurea
Patients who have received hydroxyurea therapy before entering the study.
Patients Without Prior Hydroxyurea
Patients who have not received hydroxyurea before study entry.


Outcome Measures
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Primary Outcome Measures :
  1. DNA damage from hydroxyurea therapy-variable-diversity-joining (VDJ) recombination events defined as the number of events per microgram of genomic DNA; [ Time Frame: Every 3 years ]
  2. DNA damage from hydroxyurea therapy-percentage of HJB in immature (CD71+) erythrocytes [ Time Frame: Every 3 years ]

Secondary Outcome Measures :
  1. Brain function as measured by MRI/MRA and TCD [ Time Frame: Every 3 years ]
    optional test

  2. Splenic function as measured by Spleen Scan [ Time Frame: Every 3 years ]
    optional test

  3. Kidney function as measured by BUN/creatinine and Urinalysis, glomerular filtration rate (GFR) [ Time Frame: Every 3 years ]
    optional test

  4. Lung function as measured by forced vital capacity (FVC) (%), forced vital volume in 1 second (FVC1) (%), and tricuspid regurgitation (TR) jet on Echocardiogram (ECHO) [ Time Frame: Every 3 years ]
    collected if performed for clinical purposes

  5. Growth as measured by height and weight [ Time Frame: Every visit ]

Biospecimen Retention:   Samples With DNA
Whole Blood

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   up to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Study participants will be patients with sickle cell disease who receive medical care from the Department of Hematology staff of St. Jude Children's Research Hospital. All patients on hydroxyurea therapy or patients who are initiating hydroxyurea therapy will be invited to participate.
Criteria

Inclusion Criteria:

  • Patients from birth up to age 30 years
  • Diagnosis of sickle cell disease
  • Patients who are receiving hydroxyurea therapy or plan to begin hydroxyurea therapy
Contacts and Locations
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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): NCT00305175


Locations
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United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Sponsors and Collaborators
St. Jude Children's Research Hospital
Children's Hospital Medical Center, Cincinnati
Investigators
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Principal Investigator: Jeremie Estepp, MD St. Jude Children's Research Hospital
More Information
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Additional Information:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT00305175    
Other Study ID Numbers: HUSTLE
First Posted: March 21, 2006    Key Record Dates
Last Update Posted: July 28, 2020
Last Verified: July 2020

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by St. Jude Children's Research Hospital:
Hydroxyurea
Sickle Cell Anemia
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn