A Pharmacokinetic Study of Melphalan in Children
Information source: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov processed this data on August 20, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Hematopoietic Stem Cell Transplantation
Intervention: Melphalan (Drug)
Phase: Phase 1
Status: Recruiting
Sponsored by: Children's Hospital Medical Center, Cincinnati Official(s) and/or principal investigator(s): Sharat Chandra, MD, Principal Investigator, Affiliation: Children's Hospital Medical Center, Cincinnati Parinda Mehta, MD, Principal Investigator, Affiliation: Children's Hospital Medical Center, Cincinnati
Overall contact: Sara K Loveless, BSN, RN, Phone: 513-803-7656, Email: sara.loveless@cchmc.org
Summary
The purpose of this study is to examine the pharmacokinetics (PK) of melphalan in children
undergoing hematopoietic stem cell transplantation (HSCT). Melphalan is an important
component of HSCT preparative regimens, but can be associated with significant toxicity. PK
data is a powerful clinical tool that, when used to develop individualized treatment plans
for a specific patient, may ultimately increase the likelihood of selecting the right dose
for the right patient and/or of reducing the number of adverse drug events. The
investigators' goal is to establish baseline pediatric melphalan PK data. These data may be
used for patient specific dosing of melphalan in the future to minimize toxicity and improve
transplant outcomes.
Clinical Details
Official title: Melphalan Pharmacokinetics in Children Undergoing Hematopoietic Stem Cell Transplantation: A Pilot Study
Study design: Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Basic Science
Primary outcome: To characterize the pharmacokinetics of melphalan in children undergoing HSCT
Secondary outcome: Perform measurement of acute kidney injury marker, KIM-1Perform measurement of acute kidney injury marker, NGAL
Detailed description:
Hematopoietic stem cell transplantation (HSCT) continues to be the only curative therapy for
patients with many hematological diseases. The adverse effects associated with chemotherapy
agents used as part of HSCT preparative regimens are not insignificant and can be life
threatening at times. The investigators' central hypothesis is that increased systemic
exposure to melphalan due to variation in PK in younger children leads to increased toxicity
seen in them. Although the PK of melphalan has been studied in animal models and in adult
patients, limited data exists in pediatric patients especially those undergoing allogeneic
HSCT. The objective of this study is to describe the pharmacokinetics of melphalan in
children undergoing hematopoietic stem cell transplantation. Thirty patients who are
scheduled to receive melphalan as part of their preparative regimen will be enrolled. Prior
to the start of the preparative regimen, study participants will receive a test dose of
melphalan (10% of the standard dose). Blood samples will be collected at specific time
points prior to and after the administration of the test dose and again around the full
standard dose of melphalan. This study will establish that a novel method (dry blood spot
assay) of determining PK of melphalan can be utilized in the clinical setting. Additionally,
urine samples will be collected to measure markers of kidney injury, which will help
correlate melphalan exposure with toxicity.
Eligibility
Minimum age: N/A.
Maximum age: N/A.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- Patients undergoing either allogeneic RIC HSCT, or autologous transplant containing
melphalan as part of the preparative regimen at CCHMC will be included.
Exclusion Criteria:
- Failure to sign informed consent, or inability to undergo informed consent process.
- It is not medically advisable to obtain the specimens necessary for this study.
Locations and Contacts
Sara K Loveless, BSN, RN, Phone: 513-803-7656, Email: sara.loveless@cchmc.org
Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio 45229, United States; Recruiting
Additional Information
Starting date: March 2015
Last updated: March 11, 2015
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