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

NCT07792369 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 26-024352
• Study Type: interventional
• Target: 38
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

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

Keywords

Hematopoietic Stem Cell Transplant; Busulfan; Busulfan Dosing; Body Surface Area; glutathione

Outcome Measures

Primary Outcomes (1)

• 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)

  1 day

Secondary Outcomes (3)

• Glutathione sample concentration over the 4-day course of busulfan administration.

  4 days

• Number of participants with sinusoidal obstruction

  100 days post transplant

• Number of participants with engraftment failure

  100 days post transplant

Study Arms (2)

Group A

EXPERIMENTAL

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)

Group B

EXPERIMENTAL

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)

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.

Group A; Group B

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Subject age: ≤21 years
• Body Surface Area (BSA):
• Group A: BSA ≥0.5 m2
• Group B: BSA \<0.5 m2
• Planned for once-daily busulfan-containing conditioning regimen pre-bone marrow transplant
• Scheduled to have TDM after the first dose of busulfan
• Diagnosis: both benign and malignant conditions are eligible

You may not qualify if:

• \. 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 washout periods, 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

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead
• Alex's Lemonade Stand Foundation: collaborator

Study Sites (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19103, United States

Location

MeSH Terms

Interventions

Busulfan

Intervention Hierarchy (Ancestors)

Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds

Central Study Contacts

Meghan Donnelly, MPH

CONTACT

267-426-9343; donnellymt@chop.edu

Ruchi Singh, PhD

CONTACT

singhr3@chop.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 25, 2026
• First Posted: August 28, 2026
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): September 1, 2030
• Study Completion (Estimated): September 1, 2031
• Last Updated: August 28, 2026

Record last verified: 2026-08

Locations

US (1)