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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how well the drug gemcitabine (Gemzar) can reach and affect a specific childhood brain tumor called Diffuse Intrinsic Pontine Glioma (DIPG). DIPG presents challenges for chemotherapy, and the study aims to determine if gemcitabine can effectively penetrate the tumor. Participants will receive a single dose of gemcitabine before a planned biopsy to check the drug's presence in the tumor. This trial suits children newly diagnosed with DIPG who are undergoing a biopsy as part of their standard medical care. As an Early Phase 1 trial, this research focuses on understanding how gemcitabine works in children with DIPG, offering a chance to contribute to groundbreaking treatment insights.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you need to stop taking your current medications, but it does allow the use of corticosteroids. It's best to discuss your specific medications with the study team.

Is there any evidence suggesting that gemcitabine is likely to be safe for humans?

Research has shown that gemcitabine is usually well-tolerated by patients, even those with serious conditions like high-grade glioma. Studies have found that, when combined with radiation therapy, gemcitabine is safe for humans, although some reports have noted deaths related to treatment at higher doses. At the right dose, most people handle the treatment well. Importantly, gemcitabine has been used in children with other types of recurring solid tumors and leukemia, and doctors are familiar with its dosing and possible side effects, aiding in its safe use. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Gemcitabine is unique because it offers a targeted approach to treating brain tumors with potentially fewer side effects. Unlike standard treatments like surgery, radiation, or traditional chemotherapy, gemcitabine specifically attacks tumor cells by hindering their DNA replication, which could lead to more effective tumor reduction. Researchers are excited about this treatment because it is administered in a single dose right before surgery, potentially enhancing its effectiveness without prolonged exposure to the drug. This approach might improve recovery times and reduce the overall treatment burden for patients.

What evidence suggests that gemcitabine might be an effective treatment for DIPG?

Research has shown that gemcitabine, the treatment under study in this trial, can penetrate the brain, making it a potential treatment for brain tumors. Studies have found that gemcitabine can help treat brain tumors, despite the usual difficulty medicines face in entering the brain. In lab tests, gemcitabine successfully stopped the growth of DIPG cancer cells. Previous research also indicates that gemcitabine is safe for children with other types of cancer. While there is hope for its use in treating DIPG, more human studies are needed to confirm its effectiveness for this specific condition. ^{1 2 3 6 7}

Who Is on the Research Team?

Adam Green, MD

Principal Investigator

University of Colorado, Denver

Are You a Good Fit for This Trial?

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

Patients must meet one of the specified performance scores

Informed consent and assent obtained as appropriate.

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

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 for a Trial Participant

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)

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4746478/

Gemcitabine Plus Radiation Therapy for High Grade Glioma Outcomes were similar in an analogous study delivering neoadjuvant gemcitabine plus treosulfan prior to standard radiation, with a median survival of 12 months.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC2361913/

Gemcitabine twice weekly as a radiosensitiser for the ... The 44% 5-month survival observed needs to be challenged in a future phase III study comparing the combination with WBRT alone.

3. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1359644617303483

Gemcitabine and glioblastoma: challenges and current ... However, despite the aggressive therapeutic regimen, most GBM patients quickly develop tumor recurrences that inevitably lead to death (median survival 14.6 ...

4. ascopubs.org ascopubs.org/doi/10.1200/jco.2015.33.15_suppl.e22180

Experimental study of therapeutic activity of gemcitabine in ... Gemcitabine is known to exert a therapeutic effect on brain tumors despite the limited permeability of the blood-brain barrier (BBB).

5. asco.org asco.org/abstracts-presentations/ABSTRACT115991

Therapeutic effect of gemcitabine on experimental tumors ... Conclusions: In our series of experiment we showed the potency of gemcitabine in treatment of various intracranial tumors. Until our work we can not find any ...

6. bccancer.bc.ca bccancer.bc.ca/drug-database-site/Drug%20Index/Gemcitabine_monograph.pdf

DRUG NAME: Gemcitabine Gemcitabine, a pyrimidine analog, is structurally similar to cytarabine, but has a wider spectrum of antitumour activity.

7. cancercareontario.ca cancercareontario.ca/en/node/44121

gemcitabine The optimum regimen for safe administration of gemcitabine with therapeutic doses of radiation has not yet been determined. Radiation injury has been observed ...