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Small Molecule

ONC201 in Pediatric H3 K27M Gliomas

Phase 1
Waitlist Available
Research Sponsored by Chimerix
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 28 days
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new oral medicine called ONC201 in children with specific types of brain tumors that are hard to treat. The medicine works by targeting and killing cancer cells with a particular mutation. The study aims to find the best dose and see how well the medicine works.

Eligible Conditions
  • Brain Tumor
  • Malignant Glioma
  • Diffuse Intrinsic Pontine Glioma

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~28 days
This trial's timeline: 3 weeks for screening, Varies for treatment, and 28 days for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
RP2D

Side effects data

From 2023 Phase 2 trial • 30 Patients • NCT03034200
30%
Pain
30%
Hypercalcemia
20%
Fatigue
20%
Anemia
20%
Creatinine increased
20%
Headache
20%
Hypotension
10%
Hypertension
10%
Insomnia
10%
Dizziness
10%
Urticaria
10%
Vascular disorders - Other, specify: Diaphoresis
10%
Surgical and medical procedures - Other, specify - Exploratory laparotomy of abdomen
10%
Serum sickness
10%
Brachial plexopathy
10%
Alkaline phosphatase increased
10%
Syncope
10%
Fall
10%
Fracture
10%
Sore throat
10%
Leukocytosis
10%
Chills
10%
Edema face
10%
Fever
10%
General disorders and administration site conditions - Other, specify - Hives
10%
Hypophosphatemia
10%
Dyspnea
10%
Anxiety
10%
Ascites
10%
Aspartate aminotransferase increased
10%
Atrial Flutter
10%
Bloating
10%
Bone pain
10%
Cognitive disturbance
10%
Diarrhea
10%
Dysgeusia
10%
Flatulence
10%
Gastroesophageal reflux disease
10%
General disorders and administration site conditions - Other, specify: Facial swelling
10%
General disorders and administration site conditions - Other, specify: Hives
10%
Hot flashes
10%
Investigations - Other, specify: Aspartate aminotransferase decreased
10%
Nausea
10%
Neuralgia
10%
Platelet count decreased
10%
Cardiac disorders - Other, specify: Abnormal EKG
10%
General disorders and administration site conditions - Other, specify: Neck swelling
100%
80%
60%
40%
20%
0%
Study treatment Arm
Arm A: Metastatic PC-PG
Arm B: Other NETs
Arm C: PC-PG + Other NETs

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

7Treatment groups
Experimental Treatment
Group I: ONC201 in relapsed/refractory H3 K27M gliomaExperimental Treatment1 Intervention
Pediatric patients with glioma who are positive for the H3 K27M mutation (positive testing in CLIA laboratory) and have completed at least one line of prior therapy. Evidence of progression is not required so that ONC201 may be administered to patients in the maintenance setting or to patients with recurrent/refractory disease.
Group II: ONC201 in newly diagnosed DIPGExperimental Treatment1 Intervention
Pediatric patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG), defined as tumors with a pontine epicenter and diffuse involvement of the pons, are eligible with or without histologic confirmation. If H3 K27M status of tumor is unknown or archival tumor tissue is not available, then patients must agree to submit a post-mortem biopsy specimen.
Group III: ONC201 given on two consecutive days of each weekExperimental Treatment1 Intervention
Pediatric patients with glioma who are positive for the H3 K27M mutation (positive testing in CLIA laboratory) and have completed at least one line of prior therapy will be enrolled to define the RP2D for single agent ONC201 given on two consecutive days of each week.
Group IV: Midline Glioma BiopsyExperimental Treatment1 Intervention
Pediatric patients midline gliomas are eligible with or without histologic confirmation and must be eligible for tumor biopsy as deemed by the site Investigator.
Group V: Liquid ONC201 in relapsed/refractory H3 K27M gliomaExperimental Treatment1 Intervention
Patients with glioma who are positive for the H3 K27M mutation (positive testing in CLIA laboratory) or have diagnosed diffuse intrinsic pontine glioma (DIPG), defined as tumors with a pontine epicenter and diffuse involvement of the pons, are eligible with or without histologic confirmation. Patients must be 2-12 weeks from completion of first-line radiation.
Group VI: H3 K27M CSF BiopsyExperimental Treatment1 Intervention
Pediatric patients with recurrent glioma who are positive for the H3 K27M mutation (positive testing in CLIA laboratory), have completed at least one line of prior therapy, must be willing to undergo serial lumbar puncture to obtain cerebrospinal fluid (CSF), and must be scheduled to undergo sedated MRIs.
Group VII: Dose Expansion Cohort in relapsed/refractory H3 K27M gliomaExperimental Treatment1 Intervention
Pediatric patients with previously-treated, histologically confirmed high-grade glioma with a known H3 K27M mutation, evidence of progressive disease contrast-enhanced brain MRI as defined by RANO-HGG criteria. Prior therapy with at least radiotherapy is required.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
ONC201
2017
Completed Phase 2
~60

Find a Location

Who is running the clinical trial?

ChimerixLead Sponsor
41 Previous Clinical Trials
3,957 Total Patients Enrolled

Media Library

ONC201 (Small Molecule) Clinical Trial Eligibility Overview. Trial Name: NCT03416530 — Phase 1
Brain Tumor Research Study Groups: ONC201 in newly diagnosed DIPG, Dose Expansion Cohort in relapsed/refractory H3 K27M glioma, H3 K27M CSF Biopsy, Liquid ONC201 in relapsed/refractory H3 K27M glioma, Midline Glioma Biopsy, ONC201 in relapsed/refractory H3 K27M glioma, ONC201 given on two consecutive days of each week
Brain Tumor Clinical Trial 2023: ONC201 Highlights & Side Effects. Trial Name: NCT03416530 — Phase 1
ONC201 (Small Molecule) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03416530 — Phase 1
~17 spots leftby Dec 2025