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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 adherence

Neurological 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

Page last updated: August 23, 2015

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