26DT052; Scaling Busulfan Dose to Body Surface Area in Children
25. august 2026 oppdatert av: Children's Hospital of Philadelphia
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.
Studieoversikt
Status
Har ikke rekruttert ennå
Forhold
Intervensjon / Behandling
Detaljert beskrivelse
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.
Studietype
Intervensjonell
Registrering (Antatt)
38
Fase
- Fase 1
Kontakter og plasseringer
Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.
Studiekontakt
- Navn: Meghan Donnelly, MPH
- Telefonnummer: 267-426-9343
- E-post: donnellymt@chop.edu
Studer Kontakt Backup
- Navn: Ruchi Singh, PhD
- E-post: singhr3@chop.edu
Studiesteder
-
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Pennsylvania
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Philadelphia, Pennsylvania, Forente stater, 19103
- Children's Hospital of Philadelphia
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Hovedetterforsker:
- Frank Balis, MD
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Ta kontakt med:
- Meghan Donnelly, MPH
- Telefonnummer: 267-426-9343
- E-post: donnellymt@chop.edu
-
-
Deltakelseskriterier
Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Barn
- Voksen
Tar imot friske frivillige
Nei
Beskrivelse
Inclusion Criteria:
- 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
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
Studieplan
Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: Ikke-randomisert
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Eksperimentell: 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.
|
Busulfan is a cell cycle non-specific alkylating agent which is approved by the Food and Drug Administration (FDA) and is commercially available.
|
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Eksperimentell: 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.
|
Busulfan is a cell cycle non-specific alkylating agent which is approved by the Food and Drug Administration (FDA) and is commercially available.
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Number of participants who achieve a therapeutic drug exposure after the first dose of Busulfan
Tidsramme: 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)
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1 day
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Glutathione sample concentration over the 4-day course of busulfan administration.
Tidsramme: 4 days
|
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.
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4 days
|
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Number of participants with sinusoidal obstruction
Tidsramme: 100 days post transplant
|
Target adverse events (SOS, engraftment failure) will be tracked in all patients for the first 100 days post-transplant.
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100 days post transplant
|
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Number of participants with engraftment failure
Tidsramme: 100 days post transplant
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Target adverse events (SOS, engraftment failure) will be tracked in all patients for the first 100 days post-transplant.
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100 days post transplant
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Samarbeidspartnere og etterforskere
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Samarbeidspartnere
Studierekorddatoer
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Studer hoveddatoer
Studiestart (Antatt)
1. september 2026
Primær fullføring (Antatt)
1. september 2030
Studiet fullført (Antatt)
1. september 2031
Datoer for studieregistrering
Først innsendt
25. august 2026
Først innsendt som oppfylte QC-kriteriene
25. august 2026
Først lagt ut (Faktiske)
28. august 2026
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
28. august 2026
Siste oppdatering sendt inn som oppfylte QC-kriteriene
25. august 2026
Sist bekreftet
1. august 2026
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- 26-024352
Plan for individuelle deltakerdata (IPD)
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