Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)
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|ClinicalTrials.gov Identifier: NCT01316549|
Recruitment Status : Recruiting
First Posted : March 16, 2011
Last Update Posted : July 26, 2018
|Condition or disease|
|Hematologic Malignancies Nonmalignant Diseases Immunodeficiencies Hemoglobinopathies Genetic Inborn Errors of Metabolism Fanconi Anemia Thalassemia Sickle Cell Disease|
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 sampling.
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.
|Study Type :||Observational|
|Estimated Enrollment :||75 participants|
|Official Title:||Population Pharmacokinetics of Fludarabine in Pediatric Patients Undergoing Hematopoietic Cell Transplantation|
|Study Start Date :||September 2010|
|Estimated Primary Completion Date :||July 2021|
|Estimated Study Completion Date :||July 2021|
- Analysis of the Area under the Plasma Concentration versus Time Curve (AUC) of fludarabine for HCT in pediatric patients. [ Time Frame: 2hours post start of infusion ]
- Analysis of the Area under the Plasma Concentration versus Time Curve (AUC) of fludarabine for HCT in pediatric patients. [ Time Frame: 3hours post start of infusion ]
- Analysis of the Area under the Plasma Concentration versus Time Curve (AUC) of fludarabine for HCT in pediatric patients. [ Time Frame: 6hours post start of infusion ]
- Analysis of the Area under the Plasma Concentration versus Time Curve (AUC) of fludarabine for HCT in pediatric patients. [ Time Frame: 24hours post start of infusion ]
- Event free survival according to the AUC of fludarabine [ Time Frame: 1month post transplant ]
- Event free survival according to the AUC of fludarabine [ Time Frame: 3months post transplant ]
- Event free survival according to the AUC of fludarabine [ Time Frame: 1 year post transplant ]
Biospecimen Retention: Samples With DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01316549
|Contact: Janel R Long-Boyle, PharmD, PhDfirstname.lastname@example.org|
|Contact: Chris Dvorak, MDemail@example.com|
|United States, California|
|University of California, San Francisco||Recruiting|
|San Francisco, California, United States, 94143|
|Contact: Janel R Long-Boyle, PharmD, PhD 415-514-2746 firstname.lastname@example.org|
|Principal Investigator: Janel R Long-Boyle, PharmD, PhD|
|Principal Investigator:||Janel R Long-Boyle, PharmD, PhD||University of California, San Francisco|