A Pharmacokinetic Study of Melphalan in Children
| Status: | Recruiting |
|---|---|
| Conditions: | Blood Cancer |
| Therapuetic Areas: | Oncology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 11/30/2018 |
| Start Date: | March 2015 |
| End Date: | December 31, 2019 |
| Contact: | Sara K Loveless, BSN, RN |
| Email: | sara.loveless@cchmc.org |
| Phone: | 513-803-7656 |
Melphalan Pharmacokinetics in Children Undergoing Hematopoietic Stem Cell Transplantation: A Pilot Study
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.
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.
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. Up to forty 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.
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. Up to forty 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.
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.
We found this trial at
1
site
3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
Cincinnati, Ohio 45229
1-513-636-4200
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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