Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
| Status: | Recruiting |
|---|---|
| Conditions: | Other Indications, Blood Cancer, Infectious Disease, HIV / AIDS, Anemia, Anemia, Hematology |
| Therapuetic Areas: | Hematology, Immunology / Infectious Diseases, Oncology, Other |
| Healthy: | No |
| Age Range: | Any - 17 |
| Updated: | 10/17/2018 |
| Start Date: | October 2015 |
| End Date: | December 2021 |
| Contact: | Danna D Chan, Pharm.D. |
| Email: | danna.chan@ucsf.edu |
| Phone: | 415-596-4594 |
Population Pharmacokinetics (PK) of Melphalan in Pediatric Patients Undergoing Allogeneic Hematopoietic Cell Transplantation (HCT)
Melphalan is a chemotherapy drug used extensively in bone marrow transplantation. The goal of
this study is to determine what causes some children to have different drug concentrations of
melphalan in their bodies and if drug levels are related to whether or not a child
experiences severe side-effects during their bone marrow transplant. The hypothesis is that
certain clinical and individual factors cause changes in melphalan drug levels in pediatric
bone marrow transplant patients and that high levels may cause severe side-effects.
this study is to determine what causes some children to have different drug concentrations of
melphalan in their bodies and if drug levels are related to whether or not a child
experiences severe side-effects during their bone marrow transplant. The hypothesis is that
certain clinical and individual factors cause changes in melphalan drug levels in pediatric
bone marrow transplant patients and that high levels may cause severe side-effects.
Melphalan is an alkylating agent with potent antitumor and immunosuppressive properties used
in conditioning regimens of pediatric allogeneic hematopoietic cell transplantation (alloHCT)
to promote stem cell engraftment.
This is a single-center, prospective, non-interventional pharmacokinetics (PK) study
investigating the clinical pharmacology of melphalan in 24 children undergoing allogeneic
hematopoietic stem cell transplant (alloHCT) at UCSF Benioff Children's Hospital.
Patients would receive melphalan regardless of whether or not they decide to consent to PK
sampling.
Melphalan doses will not be adjusted based on PK data.
We will apply the combination of a limited sampling strategy and population PK methodologies
to determine specific factors influencing melphalan exposure in pediatric alloHCT recipients.
Population PK methodologies support the use of sparse sampling and therefore allow us to
investigate drug levels in a pediatric population that would otherwise not be feasible using
traditional intensive PK sampling.
Subjects will undergo PK sampling of plasma melphalan drug concentrations over the duration
of melphalan therapy (3 to 5 days).
To evaluate sources of variability impacting melphalan exposure clinical data will be
obtained from the patient's medical chart on each day of PK sampling.
To assess exposure-response relationships neutrophil engraftment, treatment-related toxicity,
and survival data will be collected through day 100 post-transplant.
in conditioning regimens of pediatric allogeneic hematopoietic cell transplantation (alloHCT)
to promote stem cell engraftment.
This is a single-center, prospective, non-interventional pharmacokinetics (PK) study
investigating the clinical pharmacology of melphalan in 24 children undergoing allogeneic
hematopoietic stem cell transplant (alloHCT) at UCSF Benioff Children's Hospital.
Patients would receive melphalan regardless of whether or not they decide to consent to PK
sampling.
Melphalan doses will not be adjusted based on PK data.
We will apply the combination of a limited sampling strategy and population PK methodologies
to determine specific factors influencing melphalan exposure in pediatric alloHCT recipients.
Population PK methodologies support the use of sparse sampling and therefore allow us to
investigate drug levels in a pediatric population that would otherwise not be feasible using
traditional intensive PK sampling.
Subjects will undergo PK sampling of plasma melphalan drug concentrations over the duration
of melphalan therapy (3 to 5 days).
To evaluate sources of variability impacting melphalan exposure clinical data will be
obtained from the patient's medical chart on each day of PK sampling.
To assess exposure-response relationships neutrophil engraftment, treatment-related toxicity,
and survival data will be collected through day 100 post-transplant.
Inclusion Criteria:
- Children 0-17 years of age
- Undergoing alloHCT for the treatment of malignant or nonmalignant disorder
- Receiving melphalan-based preparative regimen
Exclusion Criteria:
- Any child 7-17 years of age unwilling to provide assent
We found this trial at
1
site
San Francisco, California 94143
Principal Investigator: Janel R Long-Boyle, PharmD, PhD
Phone: 415-596-4594
Click here to add this to my saved trials
