A Phase 1 interventional study of Busulfan (BU) in Busulfan Pharmacokinetic Analysis, Busulfan Dosing and Hematopoietic Stem Cell Transplant (HSCT), sponsored by Children's Hospital of Philadelphia. Not yet recruiting at 1 site in United States. Open to participants aged Up to 21 Years. Per ClinicalTrials.gov, last updated 2026-08-28.
Sponsored by Children's Hospital of Philadelphia · Phase 1, Interventional, and Treatment
The goal of this pilot study is to determine if scaling the first busulfan dose to Body Surface Area (BSA) in children with a BSA ≥0.5 m2 and using a BSA-banded dosing table for infants (BSA \<0.5 m2) increases the fraction of patients achieving a therapeutic drug exposure after the first dose.
Busulfan is a drug used in conditioning regimens for bone marrow transplantation. Busulfan levels outside the desired range can cause excessive side effects or failure of bone marrow engraftment. The initial dose of busulfan is currently scaled to body weight; on subsequent days the dose may be adjusted to achieve busulfan levels in a therapeutic range. However, only half of children receiving a busulfan dose scaled to body weight achieve therapeutic blood levels after the first dose. We performed computer simulations of alternative dosing methods for infants and children, and identified that dosing based on body surface area (BSA) could significantly increase the number of children achieving therapeutic blood levels after the first dose. This study will test whether this new dosing method results in a higher percentage of patients achieving the desired busulfan blood levels after the first dose. The study will enroll up to 38 children receiving high doses of busulfan as part of their standard conditioning regimen prior to bone marrow transplant. We will use the busulfan blood levels that are routinely measured after the first dose to determine the effectiveness of our new dosing method.
Children's Hospital of Philadelphia is the lead sponsor of 489 studies on the registry; 90 are open to participants now.
Of its 28 completed or terminated interventional studies of FDA-regulated products, 22 (79%) have results posted.
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Body Surface Area (BSA):
Exclusion Criteria:
1. At the time of enrollment, patients may not receive medications that significantly alter busulfan clearance, as specified below.
a. If patients had received the drugs listed below prior to enrollment, the following , based on ≥ 6 times drug t½, are required.
Deferasirox: ≥7 days Metronidazole: ≥7 days Ketoconazole, voriconazole: ≥7 days Itraconazole, posaconazole: ≥14 days Phenytoin: ≥21 days
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Washout period
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients receiving once daily busulfan in their pre-transplant conditioning regimen will have the first busulfan dose scaled to their BSA instead of their body weight. On day 1, patients with BSA ≥0.5 m2 will receive 100 mg/m2 Busulfan. Subsequent doses of busulfan on days 2-4 will be guided by standard care, including therapeutic drug monitoring (TDM) to achieve an AUC within the therapeutic range.
Drug: Busulfan (BU)
Patients receiving once daily busulfan in their pre-transplant conditioning regimen will have the first busulfan dose scaled to their BSA instead of their body weight. On day 1, patients with BSA \<0.5 m2 the dose will be selected from a BSA-banded dosing table. Subsequent doses of busulfan on days 2-4 will be guided by standard care, including therapeutic drug monitoring (TDM) to achieve an AUC within the therapeutic range.
Drug: Busulfan (BU)
Busulfan is a cell cycle non-specific alkylating agent which is approved by the Food and Drug Administration (FDA) and is commercially available.
Number of participants who achieve a therapeutic drug exposure after the first dose of Busulfan
Dosing for patients in Group A (BSA \>=0.5m2) will be scaled to Body Surface Area, and patients in Group B (BSA \<0.5m2) will be dosed based on the infant dosing table for Day 1 dose. The value range for therapeutic drug exposure is 36,000 μM/min (Lower Bound) - 6,000 μM/min (Upper Bound)
Time frame: 1 day
Glutathione sample concentration over the 4-day course of busulfan administration.
Plasma glutathione samples will be obtained daily to determine glutathione concentration over the 4 days busulfan is administered. A 4-day total of gluathione sample concentration will be reported.
Time frame: 4 days
Number of participants with sinusoidal obstruction
Target adverse events (SOS, engraftment failure) will be tracked in all patients for the first 100 days post-transplant.
Time frame: 100 days post transplant
Number of participants with engraftment failure
Target adverse events (SOS, engraftment failure) will be tracked in all patients for the first 100 days post-transplant.
Time frame: 100 days post transplant
Plan to share: Undecided
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Studies of the drug tested here: Busulfan
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