Long Term Effects of Hydroxyurea Therapy in Children With Sickle Cell Disease
Information source: St. Jude Children's Research Hospital
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Sickle Cell Disease
Phase: N/A
Status: Active, not recruiting
Sponsored by: St. Jude Children's Research Hospital Official(s) and/or principal investigator(s): Jeremie Estepp, MD, Principal Investigator, Affiliation: St. Jude Children's Research Hospital
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.
Clinical Details
Official title: Long Term Effects of Hydroxyurea Therapy in Children With Sickle Cell Disease
Study design: Observational Model: Cohort, Time Perspective: Prospective
Primary outcome: DNA damage from hydroxyurea therapy-variable-diversity-joining (VDJ) recombination events defined as the number of events per microgram of genomic DNA;DNA damage from hydroxyurea therapy-percentage of HJB in immature (CD71+) erythrocytes
Secondary outcome: Brain function as measured by MRI/MRA and TCDSplenic function as measured by Spleen Scan Kidney function as measured by BUN/creatinine and Urinalysis, glomerular filtration rate (GFR) Lung function as measured by forced vital capacity (FVC) (%), forced vital volume in 1 second (FVC1) (%), and tricuspid regurgitation (TR) jet on Echocardiogram (ECHO) Growth as measured by height and weight
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
Minimum age: N/A.
Maximum age: 30 Years.
Gender(s): Both.
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
Locations and Contacts
St. Jude Children's Research Hospital, Memphis, Tennessee 38105, United States
Additional Information
St. Jude Children's Research Hospital Clinical Trials Open at St. Jude
Starting date: March 2006
Last updated: May 11, 2015
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