Actively Recruiting
Combination Therapy for the Treatment of Diffuse Midline Gliomas
Led by University of California, San Francisco · Updated on 2025-12-17
360
Participants Needed
32
Research Sites
401 weeks
Total Duration
On this page
Sponsors
U
University of California, San Francisco
Lead Sponsor
T
The Chad-Tough Defeat DIPG Foundation
Collaborating Sponsor
AI-Summary
What this Trial Is About
This phase II trial determines if the combination of ONC201 with different drugs is effective for treating participants with diffuse midline gliomas (DMGs). Despite years of research, little to no progress has been made to improve outcomes for participants with DMGs, and there are few treatment options. This trial will utilize an adaptive platform design in that the different treatment arms for each cohort will be opened and closed based on ongoing preclinical investigation as well as evolving outcome data from the trial. Novel agents will be continuously added to this study as pre-clinical data emerge to suggest additive or synergistic activity when combined ONC201. Should a novel agent not have an RP2D at the time of incorporation into this study, a phase 1 lead-in will be performed prior to initiation of combination therapy (via study amendment).
CONDITIONS
Official Title
Combination Therapy for the Treatment of Diffuse Midline Gliomas
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 2 and 39 years
- Diagnosis of diffuse midline glioma (DMG) or diffuse intrinsic pontine glioma (DIPG) confirmed by imaging or pathology
- Recovered from acute side effects of prior therapies and beyond expected ongoing toxicities
- Body weight of at least 10 kilograms
- Willingness to provide adequate tumor tissue samples
- Stable or decreasing corticosteroid dose prior to baseline MRI
- Adequate organ function including blood counts, kidney and liver function, and cardiac health
- Female participants of child-bearing potential and males must agree to use contraception
- Karnofsky or Lansky performance score of at least 50 for participants over 16 years or 16 and under respectively
- For Cohort 6, participants must be within 4-14 weeks after completing radiation therapy and have thalamic or pontine DMG
- Prior use of temozolomide during radiation allowed under defined dosing conditions
You will not qualify if you...
- Prior exposure to radiation therapy or re-irradiation depending on cohort
- Thalamic and cerebellar H3K27M mutant DMG except under specific conditions
- Diagnosis of histone H3 wildtype grade II diffuse astrocytoma
- Currently receiving other investigational or anti-cancer agents
- Known immune disorders requiring systemic immunosuppressive therapy
- Uncontrolled infections or systemic illnesses
- Pregnant or breastfeeding females
- Active illicit drug use or alcoholism
- History of allergic reactions to study agents
- Evidence of disseminated or multifocal disease not allowed except in Cohort 5
- Additional malignancies requiring active treatment within 3 years
- Use of potent CYP3A4/5 inhibitors or inducers around study drug administration
- For Cohort 6, exclusion includes tumors outside thalamus or pons, significant mass effect or herniation, bleeding risks, active viral infections, immunosuppressive disorders, recent live vaccinations, and disseminated disease
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 32 locations
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
3
University of California, San Diego / Rady Children's Hospital, San Diego
San Diego, California, United States, 92123
Actively Recruiting
4
University of California, San Francisco
San Francisco, California, United States, 94143
Actively Recruiting
5
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
6
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
7
Indiana University Riley Children's Hospital
Indianapolis, Indiana, United States, 46202
Actively Recruiting
8
Johns Hopkins University
Baltimore, Maryland, United States, 21287
Actively Recruiting
9
Dana-Farber Cancer Institute Harvard University
Boston, Massachusetts, United States, 02215-6024
Actively Recruiting
10
University of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
11
Children's Hospital Minnesota
Minneapolis, Minnesota, United States, 55404
Actively Recruiting
12
Washington University in St. Louis
St Louis, Missouri, United States, 63110
Actively Recruiting
13
Hackensack Meridian Health
Hackensack, New Jersey, United States, 07601
Actively Recruiting
14
New York University
New York, New York, United States, 10016
Actively Recruiting
15
Duke University
Durham, North Carolina, United States, 27708
Actively Recruiting
16
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
17
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
18
University of Utah
Salt Lake City, Utah, United States, 84101
Actively Recruiting
19
Seattle Children's Hospital
Seattle, Washington, United States, 98101
Actively Recruiting
20
John Hunter Children's Hospital
New Lambton Heights, New South Wales, Australia, 2305
Actively Recruiting
21
The Children's Hospital at Westmead
Westmead, New South Wales, Australia, 2152
Actively Recruiting
22
Queensland Children's Hospital
South Brisbane, Queensland, Australia
Actively Recruiting
23
Monash Children's Hospital
Clayton, Victoria, Australia, 3168
Actively Recruiting
24
The Royal Children's Hospital Melbourne
Melbourne, Victoria, Australia, 3052
Actively Recruiting
25
Perth Children's Hospital
Nedlands, Washington, Australia, 6009
Actively Recruiting
26
Women and Children's Hospital
Adelaide, Australia
Actively Recruiting
27
Sydney Children's Hospital
Sydney, Australia, 2031
Actively Recruiting
28
Sheba Medical Center
Tel Litwinsky, Ramat Gan, Israel
Actively Recruiting
29
Shaare Zedek Medical Center
Jerusalem, Israel, 9103102
Actively Recruiting
30
Princess Maxima Center
Utrecht, Netherlands
Active, Not Recruiting
31
Starship Children's Hospital
Auckland, New Zealand
Actively Recruiting
32
The University Children's Hospital in Zurich
Zurich, Switzerland
Actively Recruiting
Research Team
K
Kelly Hitchner
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
6
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here