What side effects are associated with this type of intervention?

Common treatments for ependymoma include surgery, radiation therapy, and chemotherapy. When focusing on PD-1 inhibitors like pembrolizumab, which are being studied for their efficacy in treating recurrent or refractory ependymoma, the known side effects include fatigue, rash, pruritus, diarrhea, and nausea. More severe side effects can include immune-mediated reactions such as pneumonitis, colitis, hepatitis, endocrinopathies, and nephritis. These immune-related adverse events occur because PD-1 inhibitors enhance the immune system's activity, which can sometimes lead to it attacking normal tissues.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Ependymoma that involve PD-1 inhibitors like Pembrolizumab. Traditional treatments for Ependymoma typically include surgery, radiation therapy, and in some cases, chemotherapy. Immunotherapy, including PD-1 inhibitors, is still under investigation for this type of brain tumor, as evidenced by ongoing clinical trials such as the one studying Pembrolizumab in younger patients with recurrent or refractory Ependymoma.

What other types of research exist?

Promising alternatives to Pembrolizumab for Ependymoma patients may include other immune checkpoint inhibitors such as Nivolumab (PD-1 inhibitor) and Ipilimumab (CTLA-4 inhibitor). Additionally, targeted therapies like Everolimus, which has shown efficacy in other brain tumors, could be considered. Consulting with a healthcare provider for the latest clinical trial data and personalized treatment options is recommended.

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Checkpoint Inhibitor

Pembrolizumab for Pediatric Brain Cancer

Phase 1

Waitlist Available

Led By Eugene I Hwang

Research Sponsored by National Cancer Institute (NCI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients must have a recurrent, progressive or refractory DIPG following radiation therapy with or without chemotherapy

Patients must have received their last dose of known myelosuppressive anticancer therapy at least three (3) weeks prior to study enrollment or at least six (6) weeks if prior nitrosourea

Must not have

Patients who are receiving any other anti-cancer or investigational drug therapy

History of or ongoing pneumonitis or significant interstitial lung disease

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 3 years

Awards & highlights

No Placebo-Only Group

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Pembrolizumab may cause side effects like inflammation in organs such as lungs (pneumonitis), infusion reactions during treatment administration, fatigue, digestive issues possibly leading to diarrhea or constipation, potential blood disorders affecting cell counts, increased risk of infections due to immune system changes.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My DIPG has not improved or has worsened after radiation therapy.

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I received my last cancer treatment affecting my bone marrow 3 weeks ago, or 6 weeks ago if it was a specific type called nitrosourea.

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I received my last dose of a monoclonal antibody or similar treatment over 28 days ago and have recovered from any side effects.

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I have a high-grade brain tumor diagnosed with specific imaging.

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My cancer is a specific type of brain tumor that has not responded to treatment, including radiation.

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I have available tumor samples for testing.

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I can breathe normally without extra oxygen and my oxygen level is above 93%.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am not currently on any cancer treatment or experimental drugs.

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I have or had lung inflammation or significant lung disease.

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I have a brain tumor with a high mutation rate, including conditions like CMMRD or Lynch syndrome.

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I have multiple areas of disease in the organs of my body.

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I do not have any serious illnesses or infections unrelated to my cancer.

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I have another type of cancer besides the one being studied.

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I have been treated with anti-CTLA4 or anti-CD137 before.

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I have an active hepatitis B or C infection.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 3 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 3 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Body Weight Changes

Incidence of adverse events

Sustained objective response (partial response + complete response)

Secondary study objectives

Biomarker expression levels

Changes in quantitative imaging parameters

Event-free survival

+3 more

Side effects data

From 2024 Phase 3 trial • 804 Patients • NCT03040999

64%

Radiation skin injury

63%

Stomatitis

58%

Anaemia

56%

Nausea

48%

Dry mouth

45%

Constipation

45%

Weight decreased

44%

Dysphagia

42%

Neutrophil count decreased

33%

Dysgeusia

33%

Vomiting

32%

Fatigue

31%

White blood cell count decreased

28%

Hypomagnesaemia

26%

Decreased appetite

25%

Hypothyroidism

25%

Hypokalaemia

24%

Lymphocyte count decreased

24%

Platelet count decreased

23%

Oropharyngeal pain

23%

Blood creatinine increased

22%

Diarrhoea

22%

Odynophagia

20%

Hypoacusis

20%

Alanine aminotransferase increased

20%

Hyponatraemia

19%

Tinnitus

19%

Oral candidiasis

19%

Asthenia

16%

Pyrexia

16%

Cough

15%

Aspartate aminotransferase increased

15%

Rash

14%

Insomnia

13%

Acute kidney injury

13%

Pharyngeal inflammation

13%

Pruritus

12%

Dysphonia

12%

Gamma-glutamyltransferase increased

11%

Pneumonia

11%

Dehydration

10%

Hyperthyroidism

10%

Hypoalbuminaemia

10%

Hypocalcaemia

10%

Headache

10%

Productive cough

Neck pain

Peripheral sensory neuropathy

Gastrooesophageal reflux disease

Hiccups

Hyperglycaemia

Hyperuricaemia

Dizziness

Hypophosphataemia

Urinary tract infection

Ear pain

Localised oedema

Hyperkalaemia

Erythema

Oral pain

Abdominal pain upper

Arthralgia

Anxiety

Febrile neutropenia

Dyspepsia

Saliva altered

Back pain

Oedema peripheral

Hypertension

Dyspnoea

Nasopharyngitis

Alopecia

Dry skin

Sepsis

Pneumonia aspiration

Trismus

Pneumonitis

Laryngeal oedema

Malnutrition

Pharyngeal haemorrhage

Cellulitis

Septic shock

Systemic infection

Clostridium difficile colitis

Cardiac arrest

Death

Bronchitis

Hepatitis

Immune-mediated hepatitis

Oesophagitis

General physical health deterioration

Hypophagia

Tumour haemorrhage

Cerebrovascular accident

Syncope

Acute respiratory failure

Aspiration

Colitis

Mouth haemorrhage

Hypersensitivity

Acute myocardial infarction

Abscess neck

Device related infection

Stoma site infection

Vascular device infection

Wound infection

Hypercalcaemia

Pulmonary embolism

Respiratory failure

100%

80%

60%

40%

20%

Study treatment Arm

Pembrolizumab + CRT Followed by Pembrolizumab

Placebo + CRT Followed by Placebo

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Biospecimen Collection

2004

Completed Phase 3

~2030

Conventional Magnetic Resonance Imaging

2021

N/A

~20

Diffusion Tensor Imaging

2017

Completed Phase 2

~310

Magnetic Resonance Spectroscopic Imaging

2012

Completed Phase 1

~430

Pembrolizumab

2017

Completed Phase 3

~3150

Diffusion Weighted Imaging

2006

Completed Phase 2

~150

0 / 15Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

PD-1 inhibitors, such as Pembrolizumab, function by blocking the PD-1 pathway, which tumors use to evade immune detection. By inhibiting this pathway, these drugs enhance the immune system's ability to recognize and attack tumor cells. This mechanism is crucial for Ependymoma patients, as it provides an alternative treatment option that can potentially improve outcomes when conventional therapies like surgery and radiation are insufficient. Immunotherapy, therefore, represents a promising avenue for targeting and eliminating cancer cells through the body's own immune response.

Narrative review of immune checkpoint inhibitors and radiation therapy for brain metastases.Recurrent glioma clinical trial, CheckMate-143: the game is not over yet.The Role of Immune Checkpoint Inhibition in the Treatment of Brain Tumors.