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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 weeks3 visits
Treatment ~ Varies
Follow Up ~ 28 days
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~28 days
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 • NCT0303420030%
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
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have been recently diagnosed with a brain tumor called diffuse intrinsic pontine glioma (DIPG), whether or not it has been confirmed by a biopsy.You are currently using illegal drugs or have been diagnosed with alcoholism.You have had an allergic reaction to ONC201 or its ingredients in the past.You are between 2 and 18 years old.If you have a specific type of brain tumor called midline glioma, you can join the study with or without biopsy confirmation as long as the site investigator approves.You are able to easily swallow and keep down capsules by mouth.You have received treatment with ONC201 before.You must have waited a certain amount of time since your last treatment before starting the study: 5 times the half-life of any experimental drug, 4 weeks from most chemotherapy (except 23 days for temozolomide and 6 weeks from nitrosoureas), 6 weeks from antibody treatment, or 4 weeks (or 5 half-lives, whichever is shorter) from other anti-tumor treatments. If you have had radiation therapy, you must wait at least 2 weeks after local palliative radiotherapy. For one of the study arms, you must wait at least 90 days from prior radiation, unless the progressive lesion is outside of the high-dose radiation target volume or there is unequivocal evidence of progressive tumor on a biopsy specimen.You must weigh enough to receive the dose of ONC201 required for the study. The minimum weight requirement ranges from 10-27.5kg, depending on the dose level.You have glioma and a specific mutation in your H3 K27M genes. You have received at least one prior therapy, but evidence of progression is not required. You have had no more than two recurrences from radiotherapy and/or chemotherapy. If your tumor's H3 K27M status is unknown and archival tumor tissue is not available, a post-mortem biopsy is required.You have a type of brain tumor called diffuse intrinsic pontine glioma (DIPG) that is located in the pons region of the brain, with or without confirmation through a biopsy. If the tumor's H3 K27M status is unknown and there is no available previous biopsy, a biopsy may be required after death.You have a type of brain tumor called midline glioma, and you may or may not have had a biopsy to confirm the diagnosis. You may be eligible to participate in this trial.You are currently involved in or planning to participate in another study involving experimental drugs or devices.You have a medical or mental health condition that is not under control or a social situation that may make it difficult to follow the study requirements.You have a specific type of brain tumor called recurrent glioma with a certain genetic mutation. You have already received at least one prior treatment, and you must be willing to have multiple spinal tap procedures and sedated MRIs. You do not need to show evidence of tumor growth to receive the study medication, and you cannot have had more than two recurrences of the tumor after prior treatment. If you have signs of elevated intracranial pressure such as worsening headaches or sleepiness, you cannot have the spinal tap procedure.You have a specific type of brain tumor called glioma with a certain mutation (H3 K27M) or a subtype of glioma called diffuse intrinsic pontine glioma (DIPG). You must have completed your first round of radiation treatment between 2-12 weeks ago. It is okay if the tumor has not gotten worse so that you can receive ONC201 as a maintenance treatment or if the tumor has come back.Children with a specific type of brain tumor called high-grade glioma are eligible if they have a genetic mutation called histone H3 K27M. The tumor must have gotten worse since their last MRI scan and they must have had radiation therapy before.You are able to perform daily activities on your own without much help, and for younger patients, you are able to carry out age-appropriate tasks without much assistance.
Research Study Groups:
This trial has the following groups:- Group 1: ONC201 in newly diagnosed DIPG
- Group 2: Dose Expansion Cohort in relapsed/refractory H3 K27M glioma
- Group 3: H3 K27M CSF Biopsy
- Group 4: Liquid ONC201 in relapsed/refractory H3 K27M glioma
- Group 5: Midline Glioma Biopsy
- Group 6: ONC201 in relapsed/refractory H3 K27M glioma
- Group 7: ONC201 given on two consecutive days of each week
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.