Therapeutic Response Evaluation and Adherence Trial (TREAT)
Information source: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Anemia, Sickle Cell
Intervention: Hydroxyurea (Drug)
Phase: N/A
Status: Recruiting
Sponsored by: Children's Hospital Medical Center, Cincinnati Official(s) and/or principal investigator(s): Patrick McGann, MD, MS, Principal Investigator, Affiliation: Children's Hospital Medical Center, Cincinnati
Overall contact: Patrick McGann, MD, MS, Phone: 513-803-4991, Email: patrick.mcgann@cchmc.org
Summary
The primary objectives of this prospective study of hydroxyurea for children with sickle
cell anemia are 1) Develop and prospectively evaluate a population
pharmacokinetic/pharmacodynamics model to predict the maximum tolerated dose (MTD); 2)
Identify urine biomarkers of hydroxyurea adherence using a novel metabolomics approach; 3)
Identify pharmacogenomics modifiers of hydroxyurea MTD; and 4) Longitudinal monitoring of
the effect of hydroxyurea upon organ function and quality of life.
Clinical Details
Official title: Therapeutic Response Evaluation and Adherence Trial (TREAT): A Prospective Study of Hydroxyurea for Children With Sickle Cell Anemia
Study design: Endpoint Classification: Pharmacokinetics/Dynamics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Time to Reach Maximum Tolerated Dose (months)
Secondary outcome: Hydroxyurea adherenceNeurological function Red blood cell structure, function and survival Splenic function Kidney function Cardiac function (assessment and growth) Assessment of Quality of Life Assessment of Quality of Life as measured by the PedsQLTM core and sickle cell module
Detailed description:
There is now ample clinical evidence that hydroxyurea is a safe and effective medication for
adults and children with sickle cell anemia (SCA), and most hematologists agree the
short-term safety and efficacy of hydroxyurea has been proven. The National Heart, Lung, and
Blood Institute have recently released evidence-based guidelines for SCA, recommending that
hydroxyurea be offered to all affected children as young as nine months of age, regardless
of clinical severity. Despite the overwhelming evidence demonstrating safety and efficacy,
hydroxyurea remains underutilized for a variety of reasons. In this prospective study, the
investigators will utilize innovative strategies designed to address and overcome some of
the barriers that currently limit the use of hydroxyurea for children with SCA. The
investigators will utilize novel laboratory techniques and pharmacometric modeling in order
to accurately predict the most effective hydroxyurea dose referred to as the maximum
tolerated dose. The investigators aim to develop a screening urine test to objectively and
accurately determine adherence to hydroxyurea therapy. In addition, the study will document
critical laboratory and clinical characteristics of this unique population of patients with
SCA who begin hydroxyurea at a young age.
This study will follow two groups of patients. The first group, referred to as the New
Cohort, will include mostly young infants who are not receiving hydroxyurea therapy upon
entering the study. The starting dose of hydroxyurea for each of the participants in the New
Cohort will be individually determined using the novel population PK/PD dose-prediction
model. The second group of study participants, referred to as the Old Cohort, will include
patients who are already receiving hydroxyurea therapy upon study entry. Both the Old and
New Cohort (New Cohort) will be included in the development of a urine biomarker of
adherence and will be followed throughout the study to document the effect hydroxyurea has
upon organ function and quality of life. It is important to note that this is not a
therapeutic drug trial. Prior to enrollment in the study, participants, along with their
families and clinical providers, have decided to initiate hydroxyurea therapy for clinical
indications. Except for the dose prediction model for the New Cohort, participants will be
treated and monitored according to the routine clinical practice guidelines of the
Cincinnati Children's Hospital Comprehensive Sickle Cell Center.
Eligibility
Minimum age: 6 Months.
Maximum age: 21 Years.
Gender(s): Both.
Criteria:
Inclusion Criteria:
1. Diagnosis of sickle cell anemia (HbSS or Hbβ0-thalassemia)
2. Age 6 months to 18 years at the time of enrollment
3. Clinical decision by patient, family, and healthcare provider to initiate hydroxyurea
therapy
Exclusion Criteria:
1. Current treatment with chronic blood transfusions
Locations and Contacts
Patrick McGann, MD, MS, Phone: 513-803-4991, Email: patrick.mcgann@cchmc.org
Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio 45229, United States; Recruiting Patrick McGann, MD, MS, Phone: 513-803-4991, Email: patrick.mcgann@cchmc.org Adriane Hausfeld, RN,BSN,CCRP, Phone: (513)803-3236, Email: adriane.hausfeld@cchmc.org
Additional Information
Starting date: October 2014
Last updated: July 29, 2015
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