Clinical trialNCT05009992RecruitingPhase 2Interventional

Combination Therapy for the Treatment of Diffuse Midline Gliomas

SponsorUniversity of California, San FranciscoEnrollment360Diffuse Intrinsic Pontine GliomaDiffuse Midline Glioma, H3 K27M-MutantRecurrent Diffuse Intrinsic Pontine GliomaRecurrent Diffuse Midline Glioma, H3 K27M-MutantRecurrent WHO Grade III GliomaWHO Grade III Glioma

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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).

clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a FindMyKOL profile link to it; the rest are shown as registered.

Sabine Mueller, MD, PhD
University of California, San Francisco
Principal Investigator
clinicaltrials.gov ↗

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

32 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

Australia

John Hunter Children'S Hospital
New Lambton Heights, New South Wales, Australia
Recruiting
the Children'S Hospital at Westmead
Westmead, New South Wales, Australia
Active, not recruiting
Queensland Children'S Hospital
South Brisbane, Queensland, Australia
Active, not recruiting
Monash Children'S Hospital
Clayton, Victoria, Australia
Recruiting
the Royal Children'S Hospital Melbourne
Melbourne, Victoria, Australia
Active, not recruiting
Perth Children'S Hospital
Nedlands, Washington, Australia
Recruiting
Women and Children'S Hospital
Adelaide, Australia
Recruiting
Sydney Children'S Hospital
Sydney, Australia
Active, not recruiting

Israel

Sheba Medical Center
Tel HaShomer, Ramat Gan, Israel
Recruiting
Shaare Zedek Medical Center
Jerusalem, Israel
Recruiting

Netherlands

Princess Maxima Center
Utrecht, Netherlands
Active, not recruiting

New Zealand

Starship Children'S Hospital
Auckland, New Zealand
Recruiting

Switzerland

the University Children'S Hospital in Zurich
Zurich, Switzerland
Recruiting

United States

University of Alabama at Birmingham
Birmingham, Alabama, United States
Girish Dhall, MD
Recruiting
Children'S Hospital Los Angeles
Los Angeles, California, United States
Ashley Margol, MDTom Davidson, MD
Recruiting
University of California, San Diego / Rady Children'S Hospital, San Diego
San Diego, California, United States
Recruiting
University of California, San Francisco
San Francisco, California, United States
Sabine Mueller, MD, PhD
Recruiting
Children'S National Hospital
Washington D.C., District of Columbia, United States
Recruiting
Ann & Robert H. Lurie Children'S Hospital of Chicago
Chicago, Illinois, United States
Ashley S Plant-Fox, MD
Recruiting
Indiana University Riley Children'S Hospital
Indianapolis, Indiana, United States
Scott Coven, MD
Recruiting
Johns Hopkins University
Baltimore, Maryland, United States
Kenneth Cohen, MD
Recruiting
Dana-Farber Cancer Institute Harvard University
Boston, Massachusetts, United States
Recruiting
University of Michigan
Ann Arbor, Michigan, United States
Andrea Franson, MDCarl Koschmann, MD
Recruiting
Children'S Hospital Minnesota
Minneapolis, Minnesota, United States
Recruiting
Washington University in St. Louis
St Louis, Missouri, United States
Recruiting
Hackensack Meridian Health
Hackensack, New Jersey, United States
Derek Hanson, MD
Recruiting
New York University
New York, New York, United States
Recruiting
Duke University
Durham, North Carolina, United States
Daniel Landi, MD
Recruiting
Nationwide Children'S Hospital
Columbus, Ohio, United States
Margot Lazow, MD
Recruiting
Children'S Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Cassie Kline, MD
Recruiting
University of Utah
Salt Lake City, Utah, United States
Recruiting
Seattle Children'S Hospital
Seattle, Washington, United States
Recruiting

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

DNX-2401ONC201Paxalisib

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 20 Oct 2021
Primary completion: 31 Dec 2027
Completion: 30 Jun 2029
Last update posted: 26 Jun 2026
Registered enrollment: 360
Registry id: NCT05009992

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 4 Nov 2026. Registry facts are shown as registered by the study sponsor; FindMyKOL does not interpret them.