Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation

Conditions:Cancer, Other Indications, Blood Cancer, Infectious Disease, HIV / AIDS, Anemia, Hematology
Therapuetic Areas:Hematology, Immunology / Infectious Diseases, Oncology, Other
Age Range:Any - 17
Start Date:September 2010
End Date:July 2021
Contact:Janel R Long-Boyle, PharmD, PhD

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Population Pharmacokinetics of Fludarabine in Pediatric Patients Undergoing Hematopoietic Cell Transplantation

Fludarabine 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 fludarabine 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
clinical and genetic factors cause changes in fludarabine drug levels in pediatric bone
marrow transplant patients and that high levels may cause severe side-effects.

Fludarabine is a nucleoside analog 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, pharmacokinetic-pharmacodynamic (PK-PD) study investigating the
clinical pharmacology of fludarabine in 45 children undergoing alloHCT at UCSF Benioff
Children's Hospital.

Patients would receive fludarabine regardless of whether or not they decide to consent to PK

Fludarabine doses will not be adjusted based on PK data.

We will apply the combination of a D-optimality-based limited sampling strategy and
population PK methodologies to determine specific factors influencing fludarabine exposure in
pediatric alloHCT recipients and identify exposure-response relationships.

Subjects will undergo PK sampling of plasma (f-ara-a) and intracellular (f-ara-ATP) drug
concentrations over the duration of fludarabine therapy (3 to 5 days).

To evaluate sources of variability impacting fludarabine exposure clinical data will be
obtained from the patient's medical chart on each day of PK sampling.

A single blood draw for the collection of DNA and genotyping of single nucleotide
polymorphisms of genes involved in fludarabine activation, transport or elimination will
occur in all patients.

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 fludarabine-based preparative regimen

Exclusion Criteria:

- Any child 7-17 years of age unwilling to provide assent

- Parent or guardian unwilling to provide written consent
We found this trial at
San Francisco, California 94143
Principal Investigator: Janel R Long-Boyle, PharmD, PhD
Phone: 415-514-2746
San Francisco, CA
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