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A new dosing strategy aims to hit the busulfan sweet spot in kids

NCT ID NCT07792369

Disease control Sponsor: Children's Hospital of Philadelphia Source: ClinicalTrials.gov ↗

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 28, 2026 · Last updated Aug 28, 2026

Summary

This pilot study tests whether scaling the first busulfan dose to body surface area helps children reach a therapeutic drug level after the first dose. Children up to age 21 who are scheduled for a stem cell transplant will receive busulfan either by body surface area or by an infant dosing table. Researchers will also measure glutathione levels over the four-day treatment to see how the body processes the drug.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Busulfan, a chemotherapy drug used to prepare children for stem cell transplants
What this could lead to
If successful, this could lead to more precise busulfan dosing for children, improving transplant outcomes and reducing side effects.
What could go wrong
This is a small pilot study, so results may not apply to all children. Busulfan can cause serious side effects, and dosing adjustments may not improve outcomes as hoped.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 38 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Sep 2026

An estimate. Start dates often move.

Expected to finish

Sep 2031

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Up to 21 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Subject age: ≤21 years 2. Body Surface Area (BSA): 1. Group A: BSA ≥0.5 m2 2. Group B: BSA \<0.5 m2 3. Planned for once-daily busulfan-containing conditioning regimen pre-bone marrow transplant 4. Scheduled to have TDM after the first dose of busulfan 5. Diagnosis: both benign and malignant conditions are eligible 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 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

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As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Busulfan Busulfan dosing Busulfan pharmacokinetic analysis Glutathione Hematopoietic stem cell transplant (HSCT)

How to take part

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  1. The places running it

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  2. The official record

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    Open the record ↗

  3. A doctor treating you

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Contacts and locations

Locations

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19103, United States

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