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Enzyme Inhibitor

Combination Therapy for Brain Cancer

Phase 2
Waitlist Available
Led By Sabine Mueller, MD, PhD
Research Sponsored by University of California, San Francisco
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diarrhea < grade 2 by Common Terminology Criteria for Adverse Events (CTCAE) version (v) 5.0
No history of congestive heart failure or family history of long QT syndrome.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 6 months after administration of onc201 in the maintenance phase
Awards & highlights

Study Summary

This trial is testing if combining the drugs ONC201, panobinostat, and paxalisib is effective for treating patients with diffuse midline gliomas.

Who is the study for?
This trial is for patients aged 2 to 39 with diffuse midline gliomas, including those with spinal cord tumors. They must have completed standard radiation therapy and not be pregnant or breastfeeding. Participants need stable vital signs, controlled seizures if present, and agree to use contraception. Those with immune disorders or uncontrolled illnesses are excluded.Check my eligibility
What is being tested?
The phase II trial tests ONC201 combined with panobinostat or paxalisib in treating diffuse midline gliomas (DMGs). These drugs inhibit enzymes that may stop tumor growth. The effectiveness of different drug combinations will be assessed for patients who've had little success with other treatments.See study design
What are the potential side effects?
Potential side effects include issues related to enzyme inhibition which could affect cell growth and organ function, as well as typical reactions from cancer therapies such as fatigue, digestive problems, blood-related issues, and increased risk of infection.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My diarrhea is mild and not severe.
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I have never had heart failure and no family history of long QT syndrome.
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I weigh more than 10 kg, making me eligible for ONC201 treatment.
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My heart is healthy enough for the trial, confirmed by an echocardiogram.
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My blood tests show enough neutrophils and platelets without recent transfusions.
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I don't have shortness of breath at rest, can exercise without lung problems, and my oxygen level is above 92%.
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My heart's electrical activity (QTC) is less than 470 msec.
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My blood sugar is under 125 mg/dL without diabetes medication.
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I am between 2 and 39 years old.
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I have a confirmed diagnosis of recurrent DMG, including spinal cord tumors, and have completed standard radiation therapy.
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My kidney function is normal or only slightly below normal.
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I have DMG, completed standard radiation, and for 2B, confirmed H3K27M or WHO III/IV.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~6 months after administration of onc201 in the maintenance phase
This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months after administration of onc201 in the maintenance phase for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Overall survival at 7 months (OS7) - Cohort 3A & 3B Only
Progression-free survival at 6 months (PFS6) - Cohorts 1A, 1B, 2A, 2B Only

Side effects data

From 2021 Phase 2 trial • 30 Patients • NCT03394027
60%
Fatigue
40%
Alkaline phosphatase increased
40%
Aspartate aminotransferase increased
40%
Lymphocyte count decreased
40%
Dizziness
30%
Alanine aminotransferase increased
30%
Diarrhea
20%
Hypophosphatemia
20%
Abdominal pain
20%
Anemia
20%
Nausea
20%
Dyspnea
10%
Sepsis
10%
Lethargy
10%
Proteinuria
10%
Pain
10%
Vaginal discharge
10%
Paresthesia
10%
Pleural effusion
10%
Vaginal hemorrhage
10%
Edema limbs
10%
Flu like symptoms
10%
Gastroesophageal reflux disease
10%
Headache
10%
Atrial flutter
10%
Cardiac arrest
10%
Dehydration
10%
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, Death
10%
Sinus tachycardia
10%
Stroke
10%
Thromboembolic event
10%
Urinary tract infection
10%
Anorexia
10%
Arthralgia
10%
Back pain
10%
Constipation
10%
Lymphedema
10%
Malaise
10%
Mucosal infection
10%
Myalgia
10%
Rash maculo-papular
10%
Rhinorrhea
10%
Vascular access complication
10%
Weight loss
10%
Cough
10%
Creatinine increased
100%
80%
60%
40%
20%
0%
Study treatment Arm
Cohort 3-Endometrial Cancer (Female Only)
Cohort 1-Hormone Receptor (HR) + Breast Cancer (Male and Female)
Cohort 2-Triple Negative Breast Cancer (Male and Female)

Trial Design

3Treatment groups
Experimental Treatment
Group I: ARM 6: Paxalisib (Day -1), Radiation+Paxalisib , Paxalisib+ONC201Experimental Treatment3 Interventions
Patients may receive a safety lead in of ONC201. During trial validation phase, patients without prior biopsy receive paxalisib PO on day -1 prior to standard of care biopsy. During the radiation/re-irradiation phase, patients without prior radiation therapy or have disease progression after radiation therapy undergo weekly radiation therapy and receive paxalisib PO daily during radiation therapy. During the maintenance phase, patients receive ONC201 PO weekly and paxalisib PO QD. Cycles repeat every 28 days (4 weeks) in the absence of adverse events of unacceptable toxicity
Group II: ARM 4: ONC201 (Day -1,-2), Radiation+ONC201, Paxalisib+ONC201Experimental Treatment3 Interventions
Patients may receive a safety lead in of ONC201. During the trial validation phase, patients without prior biopsy receive ONC201 PO on days -2 and -1 prior to standard of care biopsy. During the radiation/re-irradiation phase, patients may receive ONC201 PO weekly during radiation therapy. During the maintenance phase, patients receive ONC201 PO weekly and paxalisib PO QD. Cycles repeat every 28 days (4 weeks) in the absence of adverse events or unacceptable toxicity
Group III: ARM 2: ONC201 (Day -1), Radiation+ONC201, Paxalisib+ONC201Experimental Treatment3 Interventions
Patients may receive a safety lead in of ONC201. During the trial validation phase, patients without prior biopsy receive ONC201 PO on day -1 prior to standard of care biopsy. During the radiation/re-irradiation phase, patients without prior radiation therapy or have disease progression after radiation therapy undergo weekly radiation therapy and receive ONC201 PO weekly during radiation therapy. During the maintenance phase, patients receive ONC201 PO weekly and paxalisib PO daily (QD). Cycles repeat every 28 days (4 weeks) in the absence of adverse events of unacceptable toxicity
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Radiation Therapy
2017
Completed Phase 3
~7250
ONC201
2017
Completed Phase 2
~60

Find a Location

Who is running the clinical trial?

National Institute of Neurological Disorders and Stroke (NINDS)NIH
1,339 Previous Clinical Trials
649,247 Total Patients Enrolled
University of California, San FranciscoLead Sponsor
2,500 Previous Clinical Trials
15,236,328 Total Patients Enrolled
Mithil Prasad FoundationUNKNOWN
1 Previous Clinical Trials
256 Total Patients Enrolled

Media Library

ONC201 (Enzyme Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05009992 — Phase 2
Gliomas Research Study Groups: ARM 6: Paxalisib (Day -1), Radiation+Paxalisib , Paxalisib+ONC201, ARM 2: ONC201 (Day -1), Radiation+ONC201, Paxalisib+ONC201, ARM 4: ONC201 (Day -1,-2), Radiation+ONC201, Paxalisib+ONC201
Gliomas Clinical Trial 2023: ONC201 Highlights & Side Effects. Trial Name: NCT05009992 — Phase 2
ONC201 (Enzyme Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05009992 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this a new clinical trial?

"As of now, there are 42 different ongoing medical trials that involve Panobinostat. These studies are being conducted in 37 countries and 151 cities around the world. The first known study was sponsored by Secura Bio Inc., who worked with 18 patients back in 2010. This initial study completed its Phase 1 approval stage before any other trial had begun. In the decade since this original research, 249 more studies have been completed on the topic."

Answered by AI

What patients does Panobinostat usually help?

"Panobinostat is a medication that's commonly used to treat arrhythmia. It can also help patients suffering from chronic pancreatic diseases, as well as acute conditions like shock and pancreatitis."

Answered by AI

Does this research still need participants?

"That is correct. The online information on clinicaltrials.gov reveals that this trial, which was initially posted on 10/20/2021, currently has open enrollment. A total of 216 individuals are needed for the study to be conducted at 10 distinct locations."

Answered by AI

Is this trial taking place in several Canadian locations?

"Currently, this clinical trial is enrolling patients at 10 sites. They are located in cities such as Baltimore, Hackensack, San Francisco and others. If you decide to participate in this study, please try and select the site nearest you to avoid excessive travel."

Answered by AI

Which patients would be a good fit for this particular type of research?

"This trial is recruiting 216 people, within the ages of 2 and 39 who currently have diffuse intrinsic pontine glioma. It is required that patients also meet the following criteria: COHORT 2A AND 2B: Diagnosis of DMG with imaging and/or pathology consistent with a DMG, including spinal cord tumors, who have complete standard-of-care radiation therapy. In cohort 2B, previous tumor tissue confirmation of DMG is mandatory and pathology must be consistent with a DMG including diffuse midline glioma H3K27M mutant; WHO grade III and IV H3 wildtype gli"

Answered by AI

How many individuals are being given this medication as part of the trial?

"That is accurate. The information available on clinicaltrials.gov reveals that this study began recruiting patients on 10/20/2021 and is still doing so at present. There are 10 different facilities where patients can enroll, and the goal is to have 216 total participants by the end of the recruitment period."

Answered by AI

Has the FDA cleared panobinostat for patient use?

"Panobinostat's safety is estimated to be a 2. This is due to the fact that it is still in Phase 2 clinical trials, meaning while there is evidence of safety, none exists yet for efficacy."

Answered by AI

Are there any other historical examples of Panobinostat being used in treatment?

"Panobinostat was first researched in 2010 at Augusta University. Since then, there have been 249 completed trials with 42 more underway as of now. Many of the active trials are based in Baltimore, Maryland."

Answered by AI

Does this trial have an age limit and, if so, what is it?

"The recruitment team is looking for patients who are under the age of 39 and have been living for more than 2 years."

Answered by AI

Who else is applying?

What state do they live in?
Arizona
South Dakota
How old are they?
65+
< 18
What site did they apply to?
University of California, San Francisco
Children's National Medical Center
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
Neuro-OncologyJournal
Imipridones Affect Tumor Bioenergetics and Promote Cell Lineage Differentiation in Diffuse Midline Gliomas
Przystal, Justyna M, Chiara Cianciolo Cosentino, Sridevi Yadavilli, Jie Zhang, Sandra Laternser, Erin R Bonner, Rachna Prasad, et al.. 2022. “Imipridones Affect Tumor Bioenergetics and Promote Cell Lineage Differentiation in Diffuse Midline Gliomas”. Neuro-oncology. Oxford University Press (OUP). doi:10.1093/neuonc/noac041.
clinicaltrials.govStudy
Combination Therapy for the Treatment of Diffuse Midline Gliomas
Sabine Mueller, MD, PhD 2021. "Combination Therapy for the Treatment of Diffuse Midline Gliomas". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05009992.
~57 spots leftby Dec 2025
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