Hematopoietic and Lymphoid Cell NeoplasmNCT05359237
Vincristine Pharmacokinetics in Infants
An observational study of Hematopoietic and Lymphoid Cell Neoplasm and Malignant Solid Neoplasm, run by Children's Oncology Group at 29 sites in 3 countries.
Trial summary
This pilot trial compares drug exposure levels using a new method for dosing vincristine in infants and young children compared to the standard dosing method based on body surface area (BSA) in older children. Vincristine is an anticancer drug used to a variety of childhood cancers. Read more about this study
- Who is it for?
- Ages Up to 12 years
- Conditions the study is for
- Hematopoietic and Lymphoid Cell Neoplasm, Malignant Solid Neoplasm
- What is being tested?
- Biospecimen Collection and Vincristine
- Type of treatment
- Injection or infusion, Surgery or procedure
- How many people
- 83 participants
- Where
- 29 sitesin 3 countries
- Study dates
- 16 Nov 2022to 30 Sep 2026
- Phase
- Observational
- 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 12 years
- All sexes
- No healthy volunteers
- Child
Study population: Infants and young children receiving treatment regimen that includes vinCRIStine
Who may be able to join
- Patients must be =< 12 years of age at the time of study enrollment. Patients will be stratified into 4 age groups:
- 0 to 6 months
- 6 months and 1 day to 12 months
- 12 months and 1 day to 36 months
- 36 months and 1 day to 12 years with a BSA ≥ 0.6 m^2
- Newly diagnosed and relapsed cancer diagnosis that is being treated with vinCRIStine at the 1.5 mg/m^2 dose level
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- Any disease status
- Patients must have a Lansky performance status of 50 or higher
- Patients must be receiving a treatment regimen that includes 1.5 mg/m^2 vinCRIStine (maximum dose 2 mg)
- Patients with a BSA < 0.6 m^2 must be dosed according to the Children's Oncology Group (COG) BSA-banded infant dosing table for the 1.5mg/m2 dose level for vinCRIStine
- Note: Patients can be studied after any dose of vinCRIStine
- Patients who are NOT enrolled on a COG clinical trial and who have a BSA < 0.6 m^2 and who are being dosed according to another infant dosing method (e.g., the 30-Rule) can receive a dose of vinCRIStine from the infant dosing table for the pharmacokinetic study. These patients will NOT be part of the Dose Modification Assessment
- Patients with a seizure disorder may be enrolled if on allowable anticonvulsants and well controlled as evidenced by no increase in seizure frequency in the prior 7 days
- Nervous system toxicities (Common Terminology Criteria for Adverse Events [CTCAE]) version (v)5 resulting from prior therapy must be grade =< 2
- Central venous access device in place (e.g., percutaneous indwelling central catheter [PICC], port, Broviac) or scheduled to be placed prior to the dose of vinCRIStine and that can be used for pharmacokinetic (PK) sampling
- VinCRIStine may be given as an outpatient, as long as all sample time points can be collected, which will require return for hour 24 sampling
Who cannot join
- Azoles antifungals and macrolide antibiotics: Patients who are currently receiving an azole or macrolide (e.g., fluconazole, isavuconazole, itraconazole, posaconazole, voriconazole, ketoconazole, eryromycin, clarithromycin, azithromycin, roxithromycin, or telithromycin) are not eligible
- CYP3A4/5 inducers/inhibitors: Patients receiving any medications or substances that are considered moderate or strong inhibitors or inducers of CYP3A4/5 are not eligible. Moderate or strong inducers or inhibitors of CYP3A4/5 should be avoided from 14 days prior to enrollment to the end of the study.
- Note the following are allowed:
- Dexamethasone for CNS tumors or metastases, on a stable dose
- Aprepitant for management of nausea and vomiting
- Anticonvulsants: Patients receiving moderate or strong CYP3A4/5 enzyme inducing anticonvulsants are not eligible.
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- Patients with Charcot-Marie-Tooth disease
- A baseline neurological disorder with manifestations that overlap with vinCRIStine-associated neurotoxicities
- Patients being treated on a Children Oncology Group (COG) clinical trial, that does not use the infant dosing tables for vinCRIStine are not eligible for this study.
- Patients receiving a modified dose (< 1.5 mg/m^2) of vinCRIStine due to prior toxicity
- Patients who in the opinion of the investigator may not be able to comply with the sampling requirements of the study
Trial locations and contacts
29 sites in 3 countries.
29 sites in 3 countries
United States27 sites
C S Mott Children's Hospital
Ann Arbor, Michigan, 48109
Children's Hospital Colorado
Aurora, Colorado, 80045
Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, 21287
Children's Hospital of Alabama
Birmingham, Alabama, 35233
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Lurie Children's Hospital-Chicago
Chicago, Illinois, 60611
University of Chicago Comprehensive Cancer Center
Chicago, Illinois, 60637
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Nationwide Children's Hospital
Columbus, Ohio, 43205
UT Southwestern/Simmons Cancer Center-Dallas
Dallas, Texas, 75390
Duke University Medical Center
Durham, North Carolina, 27710
Cook Children's Medical Center
Fort Worth, Texas, 76104
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
Houston, Texas, 77030
Riley Hospital for Children
Indianapolis, Indiana, 46202
Children's Hospital Los Angeles
Los Angeles, California, 90027
Saint Jude Children's Research Hospital
Memphis, Tennessee, 38105
University of Minnesota/Masonic Cancer Center
Minneapolis, Minnesota, 55455
Vanderbilt University/Ingram Cancer Center
Nashville, Tennessee, 37232
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
New York, New York, 10032
Children's Hospital of Orange County
Orange, California, 92868
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
UCSF Medical Center-Mission Bay
San Francisco, California, 94158
Seattle Children's Hospital
Seattle, Washington, 98105
Washington University School of Medicine
St Louis, Missouri, 63110
New York Medical College
Valhalla, New York, 10595
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Australia1 site
Queensland Children's Hospital
South Brisbane, Queensland, 4101
Canada1 site
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, H3T 1C5
About this study
Official title: A Pharmacokinetic Study of VinCRIStine in Infants Dosed According to BSA-Banded Infant Dosing Tables and Older Children Dosed by Traditional BSA Methods
This pilot trial compares drug exposure levels using a new method for dosing vincristine in infants and young children compared to the standard dosing method based on body surface area (BSA) in older children. Vincristine is an anticancer drug used to a variety of childhood cancers. The doses anticancer drugs in children must be adjusted based on the size of the child because children vary significantly in size (height, weight, and BSA) and ability to metabolize drugs from infancy to adolescence. The dose of most anticancer drugs is adjusted to BSA, which is calculated from a patient's weight and height. However, infants and young children have more severe side effects if the BSA is used to calculate their dose, so new dosing models have to be made to safely give anticancer drugs to the youngest patients. This new method uses a BSA-banded approach to determine the dose. Collecting blood samples before and after a dose of the drug will help researchers determine whether this new vincristine dosing method results in equivalent drug levels in the blood over time in infants and young children compared to older children.
Read the detailed description
PRIMARY OBJECTIVE:
- To validate body surface area (BSA)-banded infant dosing tables by comparing vinCRIStine drug exposure, defined as the area under the concentration-time curve for the elimination phase (AUCelim), in infants and young children dosed according to the table to older children dosed according to BSA.
SECONDARY OBJECTIVE:
- To estimate intra- and inter-age group variability (CV) using non-compartmental analysis (NCA) and population pharmacokinetic (PK) methods.
EXPLORATORY OBJECTIVES:
- To correlate higher AUCelim with the presence of functionally impaired single nucleotide polymorphisms (SNP) of CYP3A4 and CYP3A5.
II. To assess vinCRIStine dose modifications in infants receiving weekly vinCRIStine dosed according to the BSA-banded infant dosing tables.
OUTLINE:
Patients receive vincristine intravenously (IV) per standard of care (SOC). Patients undergo collection of blood samples at baseline (before first vincristine dose), and 2, 6-8, and 18-24 hours after a dose of vincristine. Patients may also undergo collection of blood samples with a second SOC vincristine dose at the same time points.
Patients are followed for dose modifications for a period of 42 days (when receiving weekly dosing of vincristine).
- Being tested
- Biospecimen Collection, Vincristine
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biospecimen CollectionProcedure or surgery
Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection
Undergo collection of blood samples
VincristineDrug
Also called LCR, Leurocristine, VCR, Vincrystine
Given IV per standard of care
| Study group | Role | What they receive |
|---|---|---|
| Observational (SOC vincristine, biospecimen collection) | Patients receive vincristine IV per SOC. Patients undergo collection of blood samples at baseline (before first vincristine dose), and 2, 6-8, and 18-24 hours after a dose of vincristine. Patients may also undergo collection of blood samples with a second SOC vincristine dose at the same time points. Receives: Procedure: Biospecimen Collection; Drug: Vincristine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Area under the concentration time curve for the elimination phase (AUCelim) by age group
Estimate (95% CI) of the mean area under the concentration time curve for the elimination phase assessed at 0, 2, 6-8, and 18-24 hours after vinCRISTine dose by age group.
Measured: Up to 24 hours post vincristine dose
Secondary outcomes (1)
Intra- and inter-age coefficient of variation (CV)
Estimate of the intra- and inter-age coefficient of variation using non-compartmental analysis (NCA) and population pharmacokinetic (PK) methods.
Measured: Up to 42 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2022
- Study start16 Nov 2022
- Primary completion30 Sep 2026 (estimated)
- Study completion30 Sep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematopoietic and Lymphoid Cell NeoplasmStudies of the same condition, open and under way.
- More trials run by Children's Oncology GroupThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 83 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Oncology Group
- Sponsor type
- Research network
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Emily Blauel, Principal investigator, Pediatric Early Phase Clinical Trial Network
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05359237
- Sponsor's protocol ID
- PEPN22P1
- Other IDs
- NCI-2022-03576, PEPN22P1, PEPN22P1, UM1CA228823
- Status last verified
- Feb 2026
- First submitted
- 28 Apr 2022
- Last updated
- 6 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pharmacokinetic Study of VinCRIStine in Infants Dosed According to BSA-Banded Infant Dosing Tables and Older Children Dosed by Traditional BSA Methods
- ClinicalTrials.gov ID
- NCT05359237
- Acronym / sponsor ID
- PEPN22P1
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 83 participants (estimated)
- Lead sponsor
- Children's Oncology GroupResearch network
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 16 Nov 2022 (actual)
- Primary completion
- 30 Sep 2026 (estimated)
- Study completion
- 30 Sep 2026 (estimated)
- First posted
- 3 May 2022 (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.
This pilot trial compares drug exposure levels using a new method for dosing vincristine in infants and young children compared to the standard dosing method based on body surface area (BSA) in older children. Vincristine is an anticancer drug used to a variety of childhood cancers. The doses anticancer drugs in children must be adjusted based on the size of the child because children vary significantly in size (height, weight, and BSA) and ability to metabolize drugs from infancy to adolescence. The dose of most anticancer drugs is adjusted to BSA, which is calculated from a patient's weight and height. However, infants and young children have more severe side effects if the BSA is used to calculate their dose, so new dosing models have to be made to safely give anticancer drugs to the youngest patients. This new method uses a BSA-banded approach to determine the dose. Collecting blood samples before and after a dose of the drug will help researchers determine whether this new vincristine dosing method results in equivalent drug levels in the blood over time in infants and young children compared to older children.
Detailed description
PRIMARY OBJECTIVE:
- To validate body surface area (BSA)-banded infant dosing tables by comparing vinCRIStine drug exposure, defined as the area under the concentration-time curve for the elimination phase (AUCelim), in infants and young children dosed according to the table to older children dosed according to BSA.
SECONDARY OBJECTIVE:
- To estimate intra- and inter-age group variability (CV) using non-compartmental analysis (NCA) and population pharmacokinetic (PK) methods.
EXPLORATORY OBJECTIVES:
- To correlate higher AUCelim with the presence of functionally impaired single nucleotide polymorphisms (SNP) of CYP3A4 and CYP3A5.
II. To assess vinCRIStine dose modifications in infants receiving weekly vinCRIStine dosed according to the BSA-banded infant dosing tables.
OUTLINE:
Patients receive vincristine intravenously (IV) per standard of care (SOC). Patients undergo collection of blood samples at baseline (before first vincristine dose), and 2, 6-8, and 18-24 hours after a dose of vincristine. Patients may also undergo collection of blood samples with a second SOC vincristine dose at the same time points.
Patients are followed for dose modifications for a period of 42 days (when receiving weekly dosing of vincristine).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Observational (SOC vincristine, biospecimen collection) | – | Procedure: Biospecimen Collection, Drug: VincristinePatients receive vincristine IV per SOC. Patients undergo collection of blood samples at baseline (before first vincristine dose), and 2, 6-8, and 18-24 hours after a dose of vincristine. Patients may also undergo collection of blood samples with a second SOC vincristine dose at the same time points. |
Who may qualify
- Ages
- Up to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Infants and young children receiving treatment regimen that includes vinCRIStine
Key inclusion criteria (16 listed)
- Patients must be =< 12 years of age at the time of study enrollment. Patients will be stratified into 4 age groups:
- Newly diagnosed and relapsed cancer diagnosis that is being treated with vinCRIStine at the 1.5 mg/m^2 dose level
- Any disease status
- Patients must have a Lansky performance status of 50 or higher
- Patients must be receiving a treatment regimen that includes 1.5 mg/m^2 vinCRIStine (maximum dose 2 mg)
Key exclusion criteria (11 listed)
- Azoles antifungals and macrolide antibiotics: Patients who are currently receiving an azole or macrolide (e.g., fluconazole, isavuconazole, itraconazole, posaconazole, voriconazole, ketoconazole, eryromycin, clarithromycin, azithromycin, roxithromycin, or telithromycin) are not eligible
- CYP3A4/5 inducers/inhibitors: Patients receiving any medications or substances that are considered moderate or strong inhibitors or inducers of CYP3A4/5 are not eligible. Moderate or strong inducers or inhibitors of CYP3A4/5 should be avoided from 14 days prior to enrollment to the end of the study.
- Anticonvulsants: Patients receiving moderate or strong CYP3A4/5 enzyme inducing anticonvulsants are not eligible.
- Patients with Charcot-Marie-Tooth disease
- A baseline neurological disorder with manifestations that overlap with vinCRIStine-associated neurotoxicities
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 |
|---|---|---|
| Biospecimen CollectionAlso: Biological Sample Collection, Biospecimen Collected, Specimen CollectionUndergo collection of blood samples | Procedure or surgery | Observational (SOC vincristine, biospecimen collection) |
| VincristineAlso: LCR, Leurocristine, VCR, VincrystineGiven IV per standard of care | Drug | Observational (SOC vincristine, biospecimen collection) |
Primary outcome
Area under the concentration time curve for the elimination phase (AUCelim) by age group
Time frame: Up to 24 hours post vincristine dose
Estimate (95% CI) of the mean area under the concentration time curve for the elimination phase assessed at 0, 2, 6-8, and 18-24 hours after vinCRISTine dose by age group.
1 secondary outcome
Intra- and inter-age coefficient of variation (CV)
Time frame: Up to 42 days
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 6 Feb 2026.
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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. This study is not recruiting at the moment.
Emily Blauel principal investigator
Pediatric Early Phase Clinical Trial Network
29 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hematopoietic and Lymphoid Cell Neoplasm and Malignant Solid Neoplasm, run by Children's Oncology Group at 29 sites in 3 countries.
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 16 inclusion and 11 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: under way, not looking for participants, as of the sponsor's last update on 6 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in United States, Australia, Canada. 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.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Area under the concentration time curve for the elimination phase (AUCelim) by age group (time frame: Up to 24 hours post vincristine dose). 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 6 Feb 2026.