110 Participants Needed

Pembrolizumab for Pediatric Brain Cancer

Recruiting at 12 trial locations
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise
Approved in 3 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial studies the safety and effectiveness of pembrolizumab in younger patients with aggressive or treatment-resistant brain tumors. Pembrolizumab is an immunotherapy drug that helps the immune system attack cancer by blocking a protein that stops immune cells from working effectively. Pembrolizumab has been used in various cancers, showing effective antitumor activity and is approved by the FDA for multiple uses.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, you must have recovered from any recent treatments and meet certain time requirements since your last dose of specific therapies before enrolling.

Is pembrolizumab generally safe for use in humans?

Pembrolizumab, also known as Keytruda, has been used in cancer treatment and is generally considered to have a tolerable safety profile. However, it can cause immune-related side effects, such as type 1 diabetes in rare cases, and other reactions like fatigue, nausea, and diarrhea. Severe side effects, though rare, can include inflammation of organs like the lungs and liver.12345

How is the drug pembrolizumab unique in treating pediatric brain cancer?

Pembrolizumab is unique because it is an immunotherapy drug that works by blocking the PD-1 receptor on immune cells, allowing them to attack cancer cells more effectively. This mechanism is different from traditional chemotherapy, which directly targets and kills cancer cells.12567

What data supports the effectiveness of the drug pembrolizumab for pediatric brain cancer?

Pembrolizumab has shown effectiveness in treating various cancers, including lung cancer and melanoma, by helping the immune system attack cancer cells. It has also been approved for use in both adults and children with certain types of solid tumors, suggesting it might be beneficial for pediatric brain cancer as well.258910

Who Is on the Research Team?

EI

Eugene I Hwang

Principal Investigator

Pediatric Brain Tumor Consortium

Are You a Good Fit for This Trial?

This trial is for young patients under 22 with specific brain tumors (high-grade gliomas, diffuse intrinsic pontine gliomas, hypermutated brain tumors, ependymoma or medulloblastoma) that are recurrent, progressive or refractory. They must have finished previous treatments and recovered from their effects. Participants need a performance score of >=70 and meet various health criteria including stable neurological deficits and adequate blood counts.

Inclusion Criteria

I am willing to take steroids for short periods if needed for side effects.
I agree to use two forms of birth control or abstain from sex for 4 months after the last dose.
My DIPG has not improved or has worsened after radiation therapy.
See 21 more

Exclusion Criteria

I don't have an active autoimmune disease needing ongoing steroids or immunosuppressants.
I am not currently on any cancer treatment or experimental drugs.
I have or had lung inflammation or significant lung disease.
See 10 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks
1 visit (in-person)

Treatment

Participants receive pembrolizumab intravenously every 21 days for up to 34 cycles

25.5 months
34 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 3 years
Every 3 months for 1 year, then every 6 months

What Are the Treatments Tested in This Trial?

Interventions

  • Pembrolizumab
Trial Overview The trial tests the safety and effectiveness of pembrolizumab in treating aggressive pediatric brain tumors. It's a phase I study to determine the best dose while observing how well it works against these cancers by potentially altering the immune system to hinder tumor growth.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Treatment (pembrolizumab)Experimental Treatment8 Interventions
Patients receive pembrolizumab IV over 30 minutes on day 1. Treatment repeats every 21 days for 34 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo standard MRI, DCE permeability MRI, DTI, DSC perfusion MRI, MR diffusion imaging and may undergo MR spectroscopy as well as CSF and blood sample collection during screening and on study.

Pembrolizumab is already approved in United States, European Union, United Kingdom for the following indications:

🇺🇸
Approved in United States as KEYTRUDA for:
  • Head and neck squamous cell carcinoma (HNSCC) with PD-L1 CPS ≥1
  • Melanoma
  • Non-small cell lung cancer (NSCLC)
  • Urothelial carcinoma
  • Colorectal cancer
  • Gastric cancer
  • Hepatocellular carcinoma
  • Renal cell carcinoma
  • Cervical cancer
  • Endometrial carcinoma
🇪🇺
Approved in European Union as KEYTRUDA for:
  • Head and neck squamous cell carcinoma (HNSCC) with PD-L1 CPS ≥1
  • Melanoma
  • Non-small cell lung cancer (NSCLC)
  • Urothelial carcinoma
  • Colorectal cancer
  • Gastric cancer
  • Hepatocellular carcinoma
  • Renal cell carcinoma
  • Cervical cancer
  • Endometrial carcinoma
🇬🇧
Approved in United Kingdom as KEYTRUDA for:
  • Untreated metastatic or unresectable recurrent head and neck squamous cell carcinoma (HNSCC) with PD-L1 CPS ≥1

Find a Clinic Near You

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials
14,080
Recruited
41,180,000+

Published Research Related to This Trial

Pembrolizumab, a PD-1 inhibitor, has demonstrated clinical effectiveness in treating various solid tumors, particularly in patients with PD-L1-positive non-small-cell lung cancer and unresectable/metastatic melanoma.
Early-phase trials and ongoing studies are focused on further confirming the clinical benefits of pembrolizumab in thoracic malignancies, highlighting its potential as a significant treatment option in cancer therapy.
Pembrolizumab for the treatment of thoracic malignancies: current landscape and future directions.Karim, S., Leighl, N.[2017]
Pembrolizumab (Keytruda) was approved by the FDA for treating metastatic non-small cell lung cancer (mNSCLC) in patients with tumors expressing PD-L1, showing significant improvements in overall survival (OS) and progression-free survival (PFS) in two major clinical trials with thousands of participants.
In the KEYNOTE-024 trial, pembrolizumab demonstrated a 40% reduction in the risk of death compared to chemotherapy, while in the KEYNOTE-010 trial, it also showed a significant survival advantage over chemotherapy in patients who had previously progressed on treatment.
FDA Approval Summary: Pembrolizumab for Treatment of Metastatic Non-Small Cell Lung Cancer: First-Line Therapy and Beyond.Pai-Scherf, L., Blumenthal, GM., Li, H., et al.[2022]
In a phase II trial involving 15 patients with resectable non-small cell lung cancer (NSCLC), neoadjuvant treatment with pembrolizumab showed a major pathologic response in 27% of patients, indicating promising antitumor activity before surgery.
The treatment was found to be feasible and safe, with only 33% of patients experiencing moderate adverse events, and no postoperative mortality, suggesting that pembrolizumab does not compromise surgical outcomes.
Neoadjuvant anti-programmed death-1 immunotherapy by pembrolizumab in resectable non-small cell lung cancer: First clinical experience.Eichhorn, F., Klotz, LV., Kriegsmann, M., et al.[2022]

Citations

Pembrolizumab for the treatment of thoracic malignancies: current landscape and future directions. [2017]
FDA Approval Summary: Pembrolizumab for Treatment of Metastatic Non-Small Cell Lung Cancer: First-Line Therapy and Beyond. [2022]
Neoadjuvant anti-programmed death-1 immunotherapy by pembrolizumab in resectable non-small cell lung cancer: First clinical experience. [2022]
Pembrolizumab for the treatment of PD-L1 positive advanced or metastatic non-small cell lung cancer. [2020]
First Tissue-Agnostic Drug Approval Issued. [2021]
Programmed Cell Death-1 Inhibitor-Induced Type 1 Diabetes Mellitus. [2022]
FDA Approval Summary: Accelerated Approval of Pembrolizumab for Second-Line Treatment of Metastatic Melanoma. [2021]
Management of pembrolizumab-induced steroid refractory mucositis with infliximab: A case report. [2020]
Pembrolizumab joins the anti-PD-1 armamentarium in the treatment of melanoma. [2017]
Pembrolizumab in paediatric patients with advanced melanoma or a PD-L1-positive, advanced, relapsed, or refractory solid tumour or lymphoma (KEYNOTE-051): interim analysis of an open-label, single-arm, phase 1-2 trial. [2020]
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