DrugLib.com — Drug Information Portal

Rx drug information, pharmaceutical research, clinical trials, news, and more



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 TCD

Splenic 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

Page last updated: August 23, 2015

-- advertisement -- The American Red Cross
 
Home | About Us | Contact Us | Site usage policy | Privacy policy

All Rights reserved - Copyright DrugLib.com, 2006-2017