Actively Recruiting
Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication 2.0 Phase 3 Trial Comparing ONC201 to Everolimus
Led by Gustave Roussy, Cancer Campus, Grand Paris · Updated on 2026-02-05
433
Participants Needed
50
Research Sites
156 weeks
Total Duration
On this page
Sponsors
G
Gustave Roussy, Cancer Campus, Grand Paris
Lead Sponsor
I
Innovative Therapies For Children with Cancer Consortium
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are investigating treatment options for Diffuse Intrinsic Pontine Glioma (DIPG) and related diffuse midline gliomas with H3K28M mutation or similar molecular features. This phase 3, randomized, open-label trial compares the effects of ONC201 versus everolimus on progression-free survival. The study aims to determine whether ONC201 provides better outcomes than everolimus, focusing on patients including newly diagnosed DIPG and non-DIPG diffuse midline glioma cases. Participants are assigned to one of two treatment groups: one takes everolimus tablets daily at a dose capped at 10 mg, and the other takes ONC201 capsules twice a week with dose limits. Both treatments continue until tumor progression, unacceptable side effects, or withdrawal of consent. Patients experiencing disease relapse may switch to the other treatment after a 7-day washout period. All patients also receive radiotherapy over six weeks, starting within weeks of biopsy or surgery, with options for reirradiation at progression. During the study, participants undergo central reviews of disease status, and treatment effects are monitored through clinical, radiological, and pathological assessments. Outcome measures include progression-free survival up to two years after the last patient joins, overall survival, complications from biopsy procedures, and safety profiles of the drugs monitored for up to five years. Participants' health status and responses to treatments are regularly evaluated, ensuring careful observation throughout the trial period.
CONDITIONS
Brief Title
Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication 2.0
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of DIPG or related diffuse midline glioma with specific genetic markers confirmed by biopsy, molecular testing, or clinical and radiological criteria
- Eligible for cerebral or craniospinal radiotherapy
- Age over 6 months with no upper age limit
- No prior chemotherapy or brain radiation therapy for the current tumor
- Affiliated with a social security system or equivalent
- Written informed consent and age-appropriate assent obtained
- Karnofsky or Lansky performance status over 50%, excluding neurological deficits
- Use of effective contraception during and for 6 months after treatment
- Negative pregnancy test for sexually active females prior to randomization
- Adequate blood counts and organ function as defined by protocol
You will not qualify if you...
- Uncontrolled spontaneous massive intratumor bleeding or uncontrolled post-operative complications
- Use of anti-cancer treatments not allowed by protocol except corticosteroids and bevacizumab under specific conditions
- Other cancer diagnosed in the last 5 years
- Uncontrolled illness or active infection
- Co-morbid conditions impairing study participation
- Inability to comply with medical follow-up
- Previous irradiation of the brainstem for another tumor
- Participation in another investigational drug study during treatment
- Under guardianship or deprived of liberty, or unable to give consent
- Current organ toxicity above grade 2, especially cardiovascular or renal issues
- Specific cardiac history contraindicating ONC201 use
- Pregnant or breastfeeding women
- Known hypersensitivity or congenital intolerances related to study drugs
- Patients taking strong P450 or PgP inhibitors requiring careful monitoring or treatment adjustments
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 6 weeks
Participants undergo radiotherapy with 30 conventional daily fractions over approximately 6 weeks, starting within 4 weeks after biopsy or surgery for DIPG.
Daily visits for 6 weeks
Duration - Until unacceptable toxicity, tumor progression, or withdrawal
Participants receive either Everolimus daily or ONC201 capsules twice weekly during and after radiotherapy. Treatment continues until unacceptable toxicity, tumor progression, or withdrawal of consent. Participants may switch treatments after progression following a 7-day washout period.
Daily or twice weekly medication with visits as scheduled by the study team
Duration - Up to 5 years after randomization
Participants are monitored for safety, survival, and treatment effects for up to 5 years after randomization.
Periodic visits over several years as scheduled by the study team
Trial Site Locations
Total: 50 locations
1
Aarhus Universitetshospital Skejby
Aarhus, Denmark, 8200
Actively Recruiting
2
Rigshospitalet
Copenhagen, Denmark, 2100
Actively Recruiting
3
H.C. Andersen Children's Hospital, Odense Universitetshospital
Odense, Denmark, 5000
Actively Recruiting
4
Gustave Roussy
Villejuif, Val de Marne, France, 94805
Actively Recruiting
5
CHU d'Amiens-Picardie Site Sud
Amiens, France, 80054
Actively Recruiting
6
Institut de Cancérologie de l'Ouest (ICO) - Site Paul Papin
Angers, France, 49055
Actively Recruiting
7
CHU d'Angers - Bâtiment Robert Debré
Angers, France, 49933
Actively Recruiting
8
CHU Besançon - Hôpital Jean Minjoz
Besançon, France, 25030
Actively Recruiting
9
CHU de Bordeaux - Groupe hospitalier Saint André - Hôpital Saint André
Bordeaux, France, 33000
Actively Recruiting
10
CHU de Bordeaux - Groupe hospitalier Pellegrin - Hôpital des enfants
Bordeaux, France, 33076
Actively Recruiting
11
CHRU de Brest - Hôpital Morvan
Brest, France, 29609
Actively Recruiting
12
CHU de Caen - Hôpital Côte de Nacre
Caen, France, 14033
Actively Recruiting
13
CHU Estaing
Clermont-Ferrand, France, 63003
Actively Recruiting
14
Centre Jean Perrin
Clermont-Ferrand, France, 63011
Actively Recruiting
15
CHU François Mitterrand
Dijon, France, 21079
Actively Recruiting
16
CHU Grenoble Alpes - Hôpital Albert Michallon
Grenoble, France, 38700
Not Yet Recruiting
17
CHU Grenoble Alpes - Hôpital Couple-Enfant
Grenoble, France, 38700
Actively Recruiting
18
Centre Oscar Lambret
Lille, France, 59020
Actively Recruiting
19
Hôpital Roger Salengro
Lille, France, 59037
Not Yet Recruiting
20
Hôpital de la mère et de l'enfant
Limoges, France, 87042
Actively Recruiting
21
Centre Léon Bérard
Lyon, France, 69373
Actively Recruiting
22
Hôpital Pierre Wertheimer
Lyon, France, 69500
Actively Recruiting
23
Hôpital de La Timone
Marseille, France, 13005
Actively Recruiting
24
Hôpital Arnaud de Villeneuve
Montpellier, France, 34090
Actively Recruiting
25
CHRU Nancy - Hôpital central
Nancy, France, 54035
Actively Recruiting
26
CHRU Nancy Brabois - Hôpital d'enfants
Nancy, France, 54500
Actively Recruiting
27
CHU de Nice - Hôpital Pasteur 2
Nice, France, 06000
Actively Recruiting
28
CHU de Nice - Hôpital L'Archet 2
Nice, France, 06202
Actively Recruiting
29
Hôpital Saint Louis
Paris, France, 75010
Actively Recruiting
30
Hôpitaux Universitaires La Pitié Salpêtrière-Charles Foix
Paris, France, 75013
Actively Recruiting
31
Institut Curie
Paris, France, 75248
Actively Recruiting
32
CHU Poitiers
Poitiers, France, 86021
Actively Recruiting
33
CHU de Reims - American Memorial Hospital 2
Reims, France, 51092
Actively Recruiting
34
Centre Eugène Marquis
Rennes, France, 35042
Actively Recruiting
35
CHU Rennes - Hôpital Sud
Rennes, France, 35203
Actively Recruiting
36
CHU Rouen Normandie - Hôpital Charles-Nicolle
Rouen, France, 76000
Actively Recruiting
37
CHU de Saint-Denis de La Réunion
Saint-Denis, France, 97400
Actively Recruiting
38
CHU de Saint-Etienne - Hôpital Nord
Saint-Etienne, France, 42270
Actively Recruiting
39
CHU de Saint-Pierre de La Réunion Sud
Saint-Pierre, France, 97488
Not Yet Recruiting
40
Centre Régional Lutte Contre le Cancer Paul STRAUSS
Strasbourg, France, 67065
Actively Recruiting
41
Hôpital de Hautepierre
Strasbourg, France, 67200
Actively Recruiting
42
Hôpital Foch
Suresnes, France, 92150
Not Yet Recruiting
43
Hôpital des enfants
Toulouse, France, 31059
Actively Recruiting
44
Institut Universitaire du Cancer de Toulouse-Oncopole (IUCT-O) - Institut Claudius Regaud
Toulouse, France, 31059
Actively Recruiting
45
CHRU Tours - Hôpital Clocheville
Tours, France, 37000
Actively Recruiting
46
CHRU Tours - Hôpital Bretonneau
Tours, France, 37044
Actively Recruiting
47
Hospital Vall D´Hebron
Barcelona, Spain, 8035
Actively Recruiting
48
Hospital Universitario Niño Jesus
Madrid, Spain, 28009
Actively Recruiting
49
Hospital Universitario y Politécnico de La Fe
Valencia, Spain, 46026
Actively Recruiting
50
Karolinska University Hospital
Stockholm, Sweden, 17176
Actively Recruiting
Research Team
J
Jacques GRILL, MD, PhD
A
Anne-Sophie BLANC, PharmD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
2
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