26DT052; Scaling Busulfan Dose to Body Surface Area in Children

Part of paid clinical trials in Philadelphia, Pennsylvania.

Sponsor
Children's Hospital of Philadelphia
Study ID
NCT07792369
Phase
PHASE1
Status
Not Yet Recruiting

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Conditions

  • Busulfan
  • Busulfan Dosing
  • Busulfan Pharmacokinetic Analysis
  • Glutathione
  • Hematopoietic Stem Cell Transplant (HSCT)

Eligibility Criteria

Sex
ALL
Age
N/A - 21 Years
Healthy Volunteers
Not accepted

Interventions

  • Busulfan (BU) — DRUG
    Busulfan is a cell cycle non-specific alkylating agent which is approved by the Food and Drug Administration (FDA) and is commercially available.

Study Details

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.

Key Dates

First listed
Aug 28, 2026
Start date
Sep 30, 2026
Status verified
Aug 2026
Primary completion
Sep 30, 2030
Completion
Sep 30, 2031

Study Design

Enrollment
38 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Group A
    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.
  • Experimental: Group B
    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.

Primary Outcome Measure

Number of participants who achieve a therapeutic drug exposure after the first dose of Busulfan [ Time Frame: 1 day ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19103
Meghan Donnelly, MPH
[email protected]
267-426-9343
Frank Balis, MD (PRINCIPAL_INVESTIGATOR)

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By research site
Children's Hospital of Philadelphia· Philadelphia, PA

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