Long Term Effects of Hydroxyurea Therapy in Children With Sickle Cell Disease
- Full Text View
- Tabular View
- No Study Results Posted
- Disclaimer
- How to Read a Study Record
Purpose
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 |
|---|
|
Sickle Cell Disease |
| Study Type: | Observational |
| Study Design: | Observational Model: Cohort Time Perspective: Prospective |
| Official Title: | Long Term Effects of Hydroxyurea Therapy in Children With Sickle Cell Disease |
- 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 ] [ Designated as safety issue: Yes ]
- DNA damage from hydroxyurea therapy-percentage of HJB in immature (CD71+) erythrocytes [ Time Frame: Every 3 years ] [ Designated as safety issue: Yes ]
- Brain function as measured by MRI/MRA and TCD [ Time Frame: Every 3 years ] [ Designated as safety issue: No ]optional test
- Splenic function as measured by Spleen Scan [ Time Frame: Every 3 years ] [ Designated as safety issue: No ]optional test
- Kidney function as measured by BUN/creatinine and Urinalysis, glomerular filtration rate (GFR) [ Time Frame: Every 3 years ] [ Designated as safety issue: No ]optional test
- 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 ] [ Designated as safety issue: No ]collected if performed for clinical purposes
- Growth as measured by height and weight [ Time Frame: Every visit ] [ Designated as safety issue: No ]
Biospecimen Retention: Samples With DNA
Whole Blood
| Estimated Enrollment: | 300 |
| Study Start Date: | March 2006 |
| Estimated Study Completion Date: | January 2026 |
| Estimated Primary Completion Date: | January 2026 (Final data collection date for primary outcome measure) |
| Groups/Cohorts |
|---|
|
1
Patients who have received hydroxyurea therapy before entering the study.
|
|
2
Patients who have not received hydroxyurea before study entry.
|
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.
Eligibility| 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.
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| Contact: Kerri Nottage, MD | 1-866-278-5833 | info@stjude.org |
| United States, Tennessee | |
| St. Jude Children's Research Hospital | Recruiting |
| Memphis, Tennessee, United States, 38105 | |
| Contact: Kerri Nottage, MD 866-278-5833 info@stjude.org | |
| Principal Investigator: | Kerri Nottage, MD | St. Jude Children's Research Hospital |
More Information
Additional Information:
No publications provided by St. Jude Children's Research Hospital
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | St. Jude Children's Research Hospital |
| ClinicalTrials.gov Identifier: | NCT00305175 History of Changes |
| Other Study ID Numbers: | HUSTLE |
| Study First Received: | March 17, 2006 |
| Last Updated: | April 23, 2013 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by St. Jude Children's Research Hospital:
|
Hydroxyurea Sickle Cell Anemia |
Additional relevant MeSH terms:
|
Anemia, Sickle Cell Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn Hydroxyurea |
Antineoplastic Agents Therapeutic Uses Pharmacologic Actions Antisickling Agents Hematologic Agents Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action Nucleic Acid Synthesis Inhibitors |
ClinicalTrials.gov processed this record on May 23, 2013