Pembrolizumab for Pediatric Brain Cancer

Recruiting at 12 trial locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: National Cancer Institute (NCI)

No Placebo Group

Breakthrough Therapy

Approved in 3 Jurisdictions

Trial Summary

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.

Research Team

Eugene I Hwang

Principal Investigator

Pediatric Brain Tumor Consortium

Eligibility Criteria

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.

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.

Treatment Details

Interventions

• Pembrolizumab (Checkpoint Inhibitor)

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.

Participant Groups

Group I: Treatment (pembrolizumab) Experimental Treatment 8 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.

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials

14,080

Recruited

41,180,000+