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Sponsored by: |
St. Jude Children's Research Hospital |
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Information provided by: | St. Jude Children's Research Hospital |
ClinicalTrials.gov Identifier: | NCT00305175 |
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 patient s who have received hydroxyurea therapy before entering the study. The second group will be made up o patients who have not received hydroxyurea before study entry.
Condition |
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Sickle Cell Disease |
Study Type: | Observational |
Study Design: | Cohort, Prospective |
Official Title: | Long Term Effects of Hydroxyurea Therapy in Children With Sickle Cell Disease |
Whole Blood
Estimated Enrollment: | 285 |
Study Start Date: | March 2006 |
Estimated Study Completion Date: | January 2016 |
Estimated Primary Completion Date: | January 2016 (Final data collection date for primary outcome measure) |
Groups/Cohorts |
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1
Patients who have received hydroxyurea therapy before entering the study.
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2
Patients who have not received hydroxyurea before study entry.
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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). Participants from the New Cohort will have pharmacokinetic studies done at baseline and at 12 months; Old Cohort participants will have PK studies performed only once, when they are at their maximum tolerated dose.
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.
Ages Eligible for Study: | up to 30 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
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. Patients who have previous hydroxyurea exposure (but are no longer on hydroxyurea therapy) will also be invited to participate.
Inclusion Criteria:
Contact: Russell Ware, MD, PhD | 1-866-278-5833 | info@stjude.org |
United States, Tennessee | |
St. Jude Children's Research Hospital | Recruiting |
Memphis, Tennessee, United States, 38105 | |
Contact: Russell Ware, MD, PhD 866-278-5833 info@stjude.org |
Principal Investigator: | Russell Ware, MD, PhD | St. Jude Children's Research Hospital |
Responsible Party: | St. Jude Children's Research Hospital ( Russell Ware, MD PhD ) |
Study ID Numbers: | HUSTLE |
Study First Received: | March 17, 2006 |
Last Updated: | December 1, 2008 |
ClinicalTrials.gov Identifier: | NCT00305175 History of Changes |
Health Authority: | United States: Institutional Review Board |
Hydroxyurea Sickle Cell Anemia |
Anemia, Hemolytic, Congenital Genetic Diseases, Inborn Hydroxyurea Hematologic Diseases Hemoglobinopathies |
Anemia Sickle Cell Anemia Anemia, Hemolytic Hemoglobinopathy Anemia, Sickle Cell |
Antisickling Agents Molecular Mechanisms of Pharmacological Action Hematologic Diseases Hydroxyurea Antineoplastic Agents Hematologic Agents Anemia Anemia, Hemolytic |
Enzyme Inhibitors Pharmacologic Actions Anemia, Hemolytic, Congenital Genetic Diseases, Inborn Hemoglobinopathies Therapeutic Uses Anemia, Sickle Cell Nucleic Acid Synthesis Inhibitors |