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Completed

NCT Number: NCT03609814

Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)

Fludarabine and clofarabine are chemotherapy drugs used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of clofarabine and 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 individual factors cause changes in clofarabine and fludarabine drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects.

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Key information

Conditions

Hematologic Malignancies Anemia Anemia, Aplastic Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Hypoplastic, Congenital Anemia, Sickle Cell Bone Marrow Diseases Bone Marrow Failure Disorders Congenital Bone Marrow Failure Syndromes Congenital, Hereditary, and Neonatal Diseases and Abnormalities DNA Repair-Deficiency Disorders Fanconi Anemia Fanconi's Anemia Genetic Diseases, Inborn Genetic Inborn Errors of Metabolism Hematologic Diseases Hematologic Neoplasms Hemic and Lymphatic Diseases Hemoglobinopathies Immune System Diseases Immunodeficiencies Immunologic Deficiency Syndromes Metabolic Diseases Neoplasms Neoplasms by Site Nonmalignant Diseases Nutritional and Metabolic Diseases Sickle Cell Disease Thalassemia

Age range

Up to 17 year

Sex eligibility

All sexes

Study type

Observational

Primary location

University of California, San Francisco

San Francisco, California, 94143, United States

About this study

Fludarabine and clofarabine are nucleoside analogs 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 pharmacokinetic (PK) study investigating the clinical pharmacology of combination nucleoside analogue therapy in 24 children undergoing alloHCT at University of California, San Francisco Benioff Children's Hospital.

Patients would receive clofarabine and fludarabine regardless of whether or not they decide to consent to PK sampling.

Clofarabine and fludarabine doses will not be adjusted based on PK data.

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

Subjects will undergo PK sampling of clofarabine and fludarabine drug concentrations over the duration of combination therapy (3 to 5 days).

To evaluate sources of variability impacting clofarabine and fludarabine 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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Children 0-17 years of age
  • Undergoing alloHCT for the treatment of malignant or nonmalignant disorder
  • Receiving clofarabine and fludarabine-based preparative regimen

Exclusion criteria

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

Treatment and study plan

Clofarabine

Drug

Given IV

Fludarabine Injection

Drug

Given IV

Primary outcomes

  1. Analysis of the Area under the Plasma Concentration versus Time Curve (AUC) of fludarabine and clofarabine dual therapy for HCT in pediatric patients.

    Time frame: 2hours post start on infusion

  2. Analysis of the Area under the Plasma Concentration versus Time Curve (AUC) of fludarabine and clofarabine dual therapy for HCT in pediatric patients.

    Time frame: 3hours post start of infusion

  3. Analysis of the Area under the Plasma Concentration versus Time Curve (AUC) of fludarabine and clofarabine dual therapy for HCT in pediatric patients.

    Time frame: 6hours post start of infusion

  4. Analysis of the Area under the Plasma Concentration versus Time Curve (AUC) of fludarabine and clofarabine dual therapy for HCT in pediatric patients.

    Time frame: 24hours post start of infusion

Secondary outcomes

  1. Evaluate the event free survival according to the AUC of fludarabine and clofarabine dual therapy

    Time frame: 1month post transplant

  2. Evaluate the event free survival according to the AUC of fludarabine and clofarabine dual therapy

    Time frame: 3 months post transplant

  3. Evaluate the event free survival according to the AUC of fludarabine and clofarabine dual therapy

    Time frame: 1 year post transplant

Sponsors and collaborators

Lead sponsor

University of California, San Francisco

Other

Registry information

Official study title

Population Pharmacokinetics of the Nucleoside Analogues Clofarabine and Fludarabine in Pediatric Patients Undergoing Hematopoietic Cell Transplantation (alloHCT)

Important dates

Study start
2016
Primary completion
2020
Study completion
2020
First posted
Aug 1, 2018
Registry last updated
Oct 4, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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