for people ages up to 17 years (full criteria)
study started
estimated completion
Principal Investigator
by Janel R Long-Boyle (ucsf)



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.

Official Title

Population Pharmacokinetics (PK) of Melphalan in Pediatric Patients Undergoing Allogeneic Hematopoietic Cell Transplantation (HCT)


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.


Hematologic Malignancies Nonmalignant Diseases Immunodeficiencies Hemoglobinopathies Genetic Inborn Errors of Metabolism Fanconi's Anemia Thalassemia Sickle Cell Disease Melphalan Pharmacokinetics Pediatric Allogeneic Hematologic Neoplasms Anemia, Sickle Cell Fanconi Anemia Metabolism, Inborn Errors


You can join if…

Open to people ages up to 17 years

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

You CAN'T join if...

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


  • University of California, San Francisco
    San Francisco California 94143 United States

Lead Scientist at University of California Health


in progress, not accepting new patients
Start Date
Completion Date
University of California, San Francisco
Study Type
Last Updated