Gemcitabine for Brain Tumor

Overseen By Ashley Mettetal, BA

Age: < 18

Sex: Any

Trial Phase: Phase < 1

Sponsor: University of Colorado, Denver

Disqualifiers: Pregnancy, Prior tumor therapy, Bleeding disorders, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 3

Duration: 1 day

2 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Research Team

Adam Green, MD

Principal Investigator

University of Colorado, Denver

Eligibility Criteria

This trial is for children aged 3-18 with a new diagnosis of DIPG, a type of brain tumor. They must have normal organ function and no prior tumor-directed therapy. Surgery for biopsy must be planned as part of their care, and they can't be pregnant or have certain illnesses that would affect the study.

Inclusion Criteria

My child is newly diagnosed with DIPG based on clinical and MRI findings.

My blood, kidney, and liver tests are within normal ranges.

Patients must meet one of the specified performance scores

Exclusion Criteria

I do not have any severe illnesses that would stop me from following the study's requirements.

Pregnant or breastfeeding patients due to teratogenic effects seen in animal/human studies

I have had treatment for my tumor before a biopsy, and I may be taking corticosteroids.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a one-time IV dose of gemcitabine prior to standard of care surgery

1 day

1 visit (in-person)

Surgery and Biopsy

Standard of care surgery is performed, and biopsies are obtained for clinical and research purposes

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

1 visit (in-person)

Treatment Details

Interventions

• Gemcitabine

Trial Overview The trial tests Gemcitabine's ability to reach DIPG tissue in the brain after being given through IV. It aims to see if this drug, which has shown promise in lab studies and crosses into the brain well, can actually get into the tumor during surgery.

Participant Groups

Group I: Gemcitabine Experimental Treatment 1 Intervention

The entire therapy on this study is one dose of gemcitabine. No intrapatient dose modifications are necessary. Gemcitabine will be given at 2100 mg/m2 IV over 30 minutes within 4 hours of planned surgical procedure.

Gemcitabine is already approved in European Union, United States, Canada, Japan for the following indications:

Approved in European Union as Gemzar for:

• Pancreatic cancer
• Breast cancer
• Ovarian cancer
• Non-small cell lung cancer

Approved in United States as Gemzar for:

• Pancreatic cancer
• Breast cancer
• Ovarian cancer
• Non-small cell lung cancer

Approved in Canada as Gemzar for:

• Pancreatic cancer
• Breast cancer
• Ovarian cancer
• Non-small cell lung cancer

Approved in Japan as Gemzar for:

• Pancreatic cancer
• Breast cancer
• Ovarian cancer
• Non-small cell lung cancer

Who Is Running the Clinical Trial?

University of Colorado, Denver

Lead Sponsor

Trials

1,842

Recruited

3,028,000+

Children's Hospital Colorado

Collaborator

Trials

121

Recruited

5,135,000+