Gemcitabine in Newly-Diagnosed Diffuse Intrinsic Pontine Glioma

  • STATUS
    Recruiting
  • End date
    Dec 24, 2023
  • participants needed
    10
  • sponsor
    University of Colorado, Denver
Updated on 24 March 2022
platelet count
renal function
prothrombin
neutrophil count
brain tumor
platelet transfusion

Summary

Diffuse Intrinsic Pontine Glioma (DIPG) is an aggressive childhood brain tumor that, despite many past clinical trials, has never been shown to respond to chemotherapy. Radiation therapy (RT) is effective in extending life but is not curative; median overall survival is 11 months. It is still unclear why the hundreds of clinical trials involving chemotherapy of DIPG have failed to demonstrate any activity against the tumor. Given that many agents tried in clinical trials cross the blood brain barrier (BBB), it is possible that there are factors specific to DIPG and its location that prevent adequate drug penetration. Gemcitabine has been selected for this study because there is strong evidence of DIPG cell line inhibition in vitro and good BBB penetration. Furthermore, pediatric dosing and toxicity has been established in prior studies of children with relapsed solid tumors and leukemia.

The primary aim of this study is to determine the presence of gemcitabine in childhood DIPG tissue after systemic treatment with the drug. The secondary aim is to quantify the intratumoral gemcitabine concentration after systemic treatment.

Participants in this study will be given a one time 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. Because patients will be undergoing this biopsy as part of their standard of care therapy here at Children's Hospital Colorado, 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.

Details
Condition Diffuse Intrinsic Pontine Glioma
Treatment Gemcitabine
Clinical Study IdentifierNCT02992015
SponsorUniversity of Colorado, Denver
Last Modified on24 March 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Age greater than or equal to 3 years and less than 18 years at the time of enrollment
Patients must meet both of the following
Clinical findings consistent with a presumed new diagnosis of DIPG
Brain MRI findings consistent with a new diagnosis of DIPG in the opinion of the treating pediatric neuro-oncologist and neurosurgeon
Organ Function Requirements
Adequate bone marrow function defined as
Platelet count ≥100,000/µl (no platelet transfusion for more than 3 days)
Hemoglobin >8 g/dl and absolute neutrophil count (ANC) ≥1,000/µl
Adequate coagulation defined as
Prothrombin time (PT) and activated partial thromboplastin time (aPTT) ≤ upper limit of normal (ULN) for age
Adequate renal function defined as
Creatinine clearane or radioisotope GFR > 70 ml/min/1.73 m2 or
Maximum serum creatinine (mg/dL) based on age/gender as follows
Male 3 to <6 yrs: 0.8 mg/dL
Female 3 to <6 yrs: 0.8 mg/dL
Male 6 to <10 yrs: 1.0 mg/dL
Female 6 to <10 yrs: 1.0 mg/dL
Male 10 to <13 yrs : 1.2 mg/dL
Female 10 to <13 yrs: 1.2 mg/dL
Male 13 to <16 yrs: 1.5 mg/dL
Female 13 to <16 yrs: 1.4 mg/dL
Male 16 to <18 yrs : 1.7 mg/dL
Female 16 to <18 yrs: 1.4 mg/dL
The threshold creatinine values in this Table were derived from the Schwartz formula
for estimating GFR (Schwartz et al. J. Peds, 106:522, 1985) utilizing child length and
stature data published by the CDC
Adequate liver function defined as
Total bilirubin <3x ULN for age and SGOT (AST) and SGPT (ALT) <2.5x ULN for age
ECOG performance status scores of 0, 1, or 2
Patients must meet one of the following performance scores
Karnofsky score of ≥ 60 for patients > 16 years of age; or
Lansky score of ≥ 60 for patients ≤ 16 years of age
Pontine tumor biopsy 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

Exclusion Criteria

Patients with intratumoral hemorrhage larger than 0.5 cm on preoperative imaging
Patients with personal or family history of bleeding disorders
Pregnant or breastfeeding patients due to teratogenic effects seen in animal/human
studies
Patients who have received any tumor-directed therapy prior to biopsy. Concurrent
treatment with corticosteroids is allowed
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
Clear my responses

How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar

Primary Contact

site

0/250

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Loading...

Browse trials for

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

user name

Added by • 

 • 

Private

Reply by • Private
Loading...

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.
Loading...

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note