Busulfan Pharmacokinetic AnalysisPhase 1NCT07792369
26DT052; Scaling Busulfan Dose to Body Surface Area in Children
A Phase 1 study testing Busulfan (BU) in people with Busulfan Pharmacokinetic Analysis and Busulfan Dosing, run by Children's Hospital of Philadelphia at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Busulfan Pharmacokinetic Analysis, Busulfan Dosing, Hematopoietic Stem Cell Transplant (HSCT)
- What is being tested?
- Busulfan (BU)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2026to Sep 2031
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 1. At the time of enrollment, patients may not receive medications that significantly alter busulfan clearance, as specified below.
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Ruchi Singh, PhD contact
United States1 site
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19103
Meghan Donnelly, MPH, 267-426-9343, donnellymt@chop.edu
Frank Balis, MD
About this study
Official title: Pilot Study of Scaling Busulfan Dose to Body Surface Area in Children Undergoing Hematopoietic Stem Cell Transplantation
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.
Read the detailed description
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.
- Conditions
- Busulfan Pharmacokinetic Analysis, Busulfan Dosing, Hematopoietic Stem Cell Transplant (HSCT), Busulfan, Glutathione
- Being tested
- Busulfan (BU)
- Keywords
- Hematopoietic Stem Cell Transplant, Busulfan, Busulfan Dosing, Body Surface Area, glutathione
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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 group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: Busulfan (BU) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 1 day
Secondary outcomes (3)
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.
Measured: 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.
Measured: 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.
Measured: 100 days post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2026
- Study startSep 2026 (estimated)
- Primary completionSep 2030 (estimated)
- Study completionSep 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Busulfan Pharmacokinetic AnalysisStudies of the same condition, open and under way.
- More trials run by Children's Hospital of PhiladelphiaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 38 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Philadelphia
- Sponsor type
- University, hospital or other
- Collaborators
- Alex's Lemonade Stand Foundation
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07792369
- Sponsor's protocol ID
- 26-024352
- Status last verified
- Aug 2026
- First submitted
- 25 Aug 2026
- Last updated
- 28 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 28 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Scaling Busulfan Dose to Body Surface Area in Children Undergoing Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT07792369
- Acronym / sponsor ID
- 26-024352
- 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
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 38 participants (estimated)
- Lead sponsor
- Children's Hospital of PhiladelphiaUniversity, hospital or other
- Collaborators
- Alex's Lemonade Stand Foundation
- Study start
- Sep 2026 (estimated)
- Primary completion
- Sep 2030 (estimated)
- Study completion
- Sep 2031 (estimated)
- First posted
- 28 Aug 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group A | Experimental | 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 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. |
| Group B | Experimental | 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. |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Subject age: ≤21 years
- Body Surface Area (BSA):
- 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
Key exclusion criteria (3 listed)
- 1. At the time of enrollment, patients may not receive medications that significantly alter busulfan clearance, as specified below.
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Drug | Group A, Group B |
Primary outcome
Number of participants who achieve a therapeutic drug exposure after the first dose of Busulfan
Time frame: 1 day
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)
3 secondary outcomes
Glutathione sample concentration over the 4-day course of busulfan administration.
Time frame: 4 days
Number of participants with sinusoidal obstruction
Time frame: 100 days post transplant
Number of participants with engraftment failure
Time frame: 100 days post transplant
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Aug 2026.
- Individual participant dataSharing planned: Undecided
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Ruchi Singh, PhD contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Busulfan (BU) in people with Busulfan Pharmacokinetic Analysis and Busulfan Dosing, run by Children's Hospital of Philadelphia at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 28 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Meghan Donnelly, MPH (267-426-9343, donnellymt@chop.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants who achieve a therapeutic drug exposure after the first dose of Busulfan (time frame: 1 day). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Aug 2026.