Glial Tumor of BrainEarly Phase 1NCT04051047
Gemcitabine in Newly-Diagnosed Diffuse Midline Glioma
A Early Phase 1 study testing Gemcitabine and Tumor biopsy and blood draw in people with Glial Tumor of Brain and Diffuse Intrinsic Pontine Glioma, run by University of Michigan Rogel Cancer Center at 1 site in United States.
Trial summary
The primary aim of this study is to determine the presence of gemcitabine in childhood diffuse midline gliomas (DMG) (previously classified as diffuse intrinsic pontine glioma [DIPG]) after systemic treatment with the drug. Read more about this study
- Who is it for?
- Ages 3 to 17 years
- Conditions the study is for
- Glial Tumor of Brain, Diffuse Intrinsic Pontine Glioma, Glioma
- What is being tested?
- Gemcitabine and Tumor biopsy and blood draw
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Nov 2019to Dec 2021
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
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 3 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age greater than or equal to 3 years and less than 18 years at the time of enrollment.
- Patients must meet both of the first two conditions, OR the third:
- Clinical findings consistent with a presumed new diagnosis of diffuse midline glioma (DMG) in the opinion of the treating neuro-oncologist, AND
- Brain MRI findings consistent with a new diagnosis of DMG based on multidisciplinary consensus after review of imaging
- OR, recurrent DMG requiring tumor resection or biopsy
- Adequate bone marrow, liver, renal and metabolic function (per protocol)
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- Adequate coagulation defined as Prothrombin time (PT) and activated partial thromboplastin time (aPTT) ≤ upper limit of normal (ULN ) for age
- Patients must meet one of the following performance scores:
- ECOG performance status scores of 0, 1, or 2; 8
- Karnofsky score of ≥ 60 for patients > 16 years of age; or
- Lansky score of ≥ 60 for patients ≤ 16 years of age
- DMG biopsy/resection is planned for the clinical care of the patient independent of study participation by the treating pediatric neurosurgeon and neuro-oncologist.
- Informed consent and assent obtained as appropriate.
Who cannot join
- Pregnant or breastfeeding patients are not eligible due to teratogenic effects seen in animal/human studies
- Patients who have received any tumor-directed therapy prior to biopsy are not eligible. Concurrent treatment with corticosteroids is allowed.
- Any tumor-specific or clinical features that make surgical intervention unsafe in the opinion of the treating neurosurgeon.
- Patients with personal or family history of bleeding disorders are not eligible.
- Patients with uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements are not eligible.
- Patients with known hypersensitivity to gemcitabine
Trial locations and contacts
1 site in 1 country.
United States1 site
The University of Michigan Rogel Cancer Center
Ann Arbor, Michigan, 48109
About this study
Official title: An Early Phase 1 Trial of Gemcitabine in Newly-Diagnosed Diffuse Midline Glioma
The primary aim of this study is to determine the presence of gemcitabine in childhood diffuse midline gliomas (DMG) (previously classified as diffuse intrinsic pontine glioma [DIPG]) after systemic treatment with the drug.
Read the detailed description
Participants in this study will be given a one-time, intravenous (IV) dose of gemcitabine prior to having standard-of-care surgery. During surgery, biopsies will be obtained for clinical and research purposes along with a blood sample. Cerebrospinal Fluid (CSF) is optional and will only be obtained if clinically indicated. This will be determined by the investigators and the provider performing the procedure.
Because patients will be undergoing a biopsy/resection as part of their standard-of-care therapy, this is an optimal time to obtain a tumor biopsy for this study. The biopsy will serve to see if the study drug is penetrating the tumor. Patients will then enter a follow-up period for 30 days post-surgery.
This trial is in conjunction with a University of Colorado trial started in 2016, "Gemcitabine in Children With Newly-Diagnosed Diffuse Intrinsic Pontine Glioma" (NCT02992015).
- Being tested
- Gemcitabine, Tumor biopsy and blood draw
- Keywords
- Glioma
- Medical subject headings
- Diffuse Intrinsic Pontine Glioma, Glioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GemcitabineDrug
Also called Gemzar
Participants receive a one time IV dose of gemcitabine prior to having standard of care surgery.
Tumor biopsy and blood drawProcedure or surgery
As part of standard of care, all participants will undergo tumor biopsy and collection of peripheral blood.
| Study group | Role | What they receive |
|---|---|---|
| Gemcitabine | Experimental | Gemcitabine will be given at 2100 mg/m2 IV over 30 minutes within 4 hours of planned surgical procedure. The surgical procedure is standard of care. Receives: Drug: Gemcitabine; Procedure: Tumor biopsy and blood draw |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with detectable intratumoral gemcitabine after systemic treatment with the drug
Gemcitabine will be considered to be detectable in a participant's tumor sample if gemcitabine or one of its metabolites (difluorodeoxyuridine [dFdU] or gemcitabine triphosphate [dFdCTP]) is detected at a measurable level in any of the participant's tumor samples. Undetectable gemcitabine will be defined as gemcitabine, dFdU, and dFdCTP levels below the detectable limits for all of an individual participant's samples, in the setting of detectable gemcitabine, dFdU, or dFdCTP in the plasma and/or cerebrospinal fluid (CSF) for that patient, and the pathologic review showing viable tumor tissue.
Measured: Day 1
Secondary outcomes (1)
Concentration of gemcitabine in patient tumor sample
Gemcitabine, dFdU and dFdCTP will be quantified in the available tumor sample(s), in plasma, and in CSF (if available). For this pilot trial, gemcitabine PK results will be analyzed descriptively, and this trial is not designed for statistical analysis.
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2019
- Study start30 Nov 2019 (estimated)
- Primary completionDec 2021 (estimated)
- Study completionDec 2021 (estimated)
Why it stopped: Withdrawn by Principal Investigator
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glial Tumor of BrainStudies of the same condition, open and under way.
- 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
- Early Phase 1
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Michigan Rogel Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- University of Colorado, Denver
- Responsible party
- Sponsor
- Investigators
- Carl Koschmann, M.D., Principal investigator, University of Michigan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04051047
- Sponsor's protocol ID
- UMCC 2018.159
- Other IDs
- HUM00135182
- Status last verified
- Nov 2019
- First submitted
- 7 Aug 2019
- Last updated
- 27 Nov 2019
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Early Phase 1 Trial of Gemcitabine in Newly-Diagnosed Diffuse Midline Glioma
- ClinicalTrials.gov ID
- NCT04051047
- Acronym / sponsor ID
- UMCC 2018.159
- Keywords
- Glioma
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Withdrawn by Principal Investigator
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of Michigan Rogel Cancer CenterUniversity, hospital or other
- Collaborators
- University of Colorado, Denver
- Study start
- 30 Nov 2019 (estimated)
- Primary completion
- Dec 2021 (estimated)
- Study completion
- Dec 2021 (estimated)
- First posted
- 9 Aug 2019 (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 primary aim of this study is to determine the presence of gemcitabine in childhood diffuse midline gliomas (DMG) (previously classified as diffuse intrinsic pontine glioma [DIPG]) after systemic treatment with the drug.
Detailed description
Participants in this study will be given a one-time, intravenous (IV) dose of gemcitabine prior to having standard-of-care surgery. During surgery, biopsies will be obtained for clinical and research purposes along with a blood sample. Cerebrospinal Fluid (CSF) is optional and will only be obtained if clinically indicated. This will be determined by the investigators and the provider performing the procedure.
Because patients will be undergoing a biopsy/resection as part of their standard-of-care therapy, this is an optimal time to obtain a tumor biopsy for this study. The biopsy will serve to see if the study drug is penetrating the tumor. Patients will then enter a follow-up period for 30 days post-surgery.
This trial is in conjunction with a University of Colorado trial started in 2016, "Gemcitabine in Children With Newly-Diagnosed Diffuse Intrinsic Pontine Glioma" (NCT02992015).
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Gemcitabine | Experimental | Drug: Gemcitabine, Procedure: Tumor biopsy and blood drawGemcitabine will be given at 2100 mg/m2 IV over 30 minutes within 4 hours of planned surgical procedure. The surgical procedure is standard of care. |
Who may qualify
- Ages
- 3 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (13 listed)
- Age greater than or equal to 3 years and less than 18 years at the time of enrollment.
- Patients must meet both of the first two conditions, OR the third:
- Adequate bone marrow, liver, renal and metabolic function (per protocol)
- Adequate coagulation defined as Prothrombin time (PT) and activated partial thromboplastin time (aPTT) ≤ upper limit of normal (ULN ) for age
- Patients must meet one of the following performance scores:
Key exclusion criteria (6 listed)
- Pregnant or breastfeeding patients are not eligible due to teratogenic effects seen in animal/human studies
- Patients who have received any tumor-directed therapy prior to biopsy are not eligible. Concurrent treatment with corticosteroids is allowed.
- Any tumor-specific or clinical features that make surgical intervention unsafe in the opinion of the treating neurosurgeon.
- Patients with personal or family history of bleeding disorders are not eligible.
- Patients with uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements are not eligible.
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 |
|---|---|---|
| GemcitabineAlso: GemzarParticipants receive a one time IV dose of gemcitabine prior to having standard of care surgery. | Drug | Gemcitabine |
| Tumor biopsy and blood drawAs part of standard of care, all participants will undergo tumor biopsy and collection of peripheral blood. | Procedure or surgery | Gemcitabine |
Primary outcome
Number of participants with detectable intratumoral gemcitabine after systemic treatment with the drug
Time frame: Day 1
Gemcitabine will be considered to be detectable in a participant's tumor sample if gemcitabine or one of its metabolites (difluorodeoxyuridine [dFdU] or gemcitabine triphosphate [dFdCTP]) is detected at a measurable level in any of the participant's tumor samples. Undetectable gemcitabine will be defined as gemcitabine, dFdU, and dFdCTP levels below the detectable limits for all of an individual participant's samples, in the setting of detectable gemcitabine, dFdU, or dFdCTP in the plasma and/or cerebrospinal fluid (CSF) for that patient, and the pathologic review showing viable tumor tissue.
1 secondary outcome
Concentration of gemcitabine in patient tumor sample
Time frame: Day 1
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Nov 2019.
- Individual participant dataSharing planned: No
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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.
Carl Koschmann, M.D. principal investigator
University of Michigan
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Gemcitabine and Tumor biopsy and blood draw in people with Glial Tumor of Brain and Diffuse Intrinsic Pontine Glioma, run by University of Michigan Rogel Cancer Center 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 13 inclusion and 6 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: withdrawn, as of the sponsor's last update on 27 Nov 2019. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Number of participants with detectable intratumoral gemcitabine after systemic treatment with the drug (time frame: Day 1). 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 27 Nov 2019; AzTrials last refreshed its copy on 26 Oct 2026.