RecruitingPhase 3NCT05476939

Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication 2.0

Biological Medicine for Diffuse Intrinsic Pontine Glioma (DIPG) Eradication


Sponsor

Gustave Roussy, Cancer Campus, Grand Paris

Enrollment

433 participants

Start Date

Sep 29, 2022

Study Type

INTERVENTIONAL

Conditions

Summary

The BIOMEDE 2.0 study is the second stage of the BIOMEDE multi-arm, multistage rolling programme (adaptive platform protocol). It is a multicenter, randomized, open-label, controlled phase-3 trial evaluating efficacy of ONC201 in comparison with everolimus (primary objective based on internal comparison) and subsequently to historical controls. Two treatment groups will be compared. A switch between treatment groups is allowed after confirmation of the disease progression (real-time central review blinded to the treatment arm allocation). Study treatment will be continued until centrally confirmed disease progression (either radiologically or histologically), unacceptable toxicity or consent withdrawal. The final conclusion of the trial will be successful for ONC201, if ONC201 is found significantly superior to everolimus in terms of centrally-reviewed PFS (Progression-free survival) from randomization (internal comparison) either overall, considering ND-DMG and DIPG-patients together, or in the subgroup of ND-DMG patients alone. In other cases, Everolimus will remain the standard arm unless it appears associated with an excess of toxicity compared to ONC201 which could then be discussed as a new standard.


Eligibility

Min Age: 6 Months

Plain Language Summary

Simplified for easier understanding

This study is researching new treatments for Diffuse Intrinsic Pontine Glioma (DIPG) — a rare and aggressive brain tumor that forms in the brainstem, mostly in children. The study tests different targeted therapies based on the tumor's specific genetic profile. **You may be eligible if...** - You have been diagnosed with DIPG based on imaging, biopsy, or molecular testing - Your tumor has specific genetic features (H3K28M mutation or related changes) - You may also qualify if you have a related tumor type called a diffuse midline glioma in another brainstem location - Parents or guardians can consent on behalf of children **You may NOT be eligible if...** - The diagnosis has not been confirmed by the required clinical, imaging, or molecular criteria - You do not meet the specific genetic or tumor location requirements - Your treating team determines that a biopsy is not safely possible and no alternative molecular diagnosis is available Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGEverolimus

Tablets of 2.5 mg or 10 mg. The prescribed dose is 5 mg/m²/day, orally, once daily. Dose will be capped at 10 mg once daily.

DRUGONC201

Capsules of 125mg. The prescribed dose is 375mg/m², orally, once daily at Day 1 and Day 2 of each week. Dose will be capped at 625 mg per dose.

RADIATIONRadiotherapy

All patients will be treated with 30 conventional single daily fractions of 1.8 Gy to a total of 54 Gy over a planned period of 6 weeks. Dose may be increased up to 60 Gy for adult patients with supratentorial ND-DMG. The clinical target volume will include all the areas of abnormality on T2/FLAIR sequences with a 1-cm margin. Radiotherapy will have to start within a maximum of 4 weeks for DIPG, up to 6 weeks for other DMG H3K28-altered (ND-DMG), after the biopsy or last surgery. The study medication will be started at Day 1 (+3 days max) of radiotherapy. Reirradiation is permitted only at disease progression according to local practice. In case of metastatic disease or intramedullary tumors, patients can be included in the study. In this situation, radiotherapy will have to start within a maximum of 4 weeks for DIPG, up to 6 weeks for other DMG H3K28-altered (ND-DMG), after the biopsy or last surgery while targeted treatment will start at the end of the irradiation.


Locations(50)

Aarhus Universitetshospital Skejby

Aarhus, Denmark

Rigshospitalet

Copenhagen, Denmark

H.C. Andersen Children's Hospital, Odense Universitetshospital

Odense, Denmark

Gustave Roussy

Villejuif, Val de Marne, France

CHU d'Amiens-Picardie Site Sud

Amiens, France

Institut de Cancérologie de l'Ouest (ICO) - Site Paul Papin

Angers, France

CHU d'Angers - Bâtiment Robert Debré

Angers, France

CHU Besançon - Hôpital Jean Minjoz

Besançon, France

CHU de Bordeaux - Groupe hospitalier Saint André - Hôpital Saint André

Bordeaux, France

CHU de Bordeaux - Groupe hospitalier Pellegrin - Hôpital des enfants

Bordeaux, France

CHRU de Brest - Hôpital Morvan

Brest, France

CHU de Caen - Hôpital Côte de Nacre

Caen, France

CHU Estaing

Clermont-Ferrand, France

Centre Jean Perrin

Clermont-Ferrand, France

CHU François Mitterrand

Dijon, France

CHU Grenoble Alpes - Hôpital Albert Michallon

Grenoble, France

CHU Grenoble Alpes - Hôpital Couple-Enfant

Grenoble, France

Centre Oscar Lambret

Lille, France

Hôpital Roger Salengro

Lille, France

Hôpital de la mère et de l'enfant

Limoges, France

Centre Léon Bérard

Lyon, France

Hôpital Pierre Wertheimer

Lyon, France

Hôpital de La Timone

Marseille, France

Hôpital Arnaud de Villeneuve

Montpellier, France

CHRU Nancy - Hôpital central

Nancy, France

CHRU Nancy Brabois - Hôpital d'enfants

Nancy, France

CHU de Nice - Hôpital Pasteur 2

Nice, France

CHU de Nice - Hôpital L'Archet 2

Nice, France

Hôpital Saint Louis

Paris, France

Hôpitaux Universitaires La Pitié Salpêtrière-Charles Foix

Paris, France

Institut Curie

Paris, France

CHU Poitiers

Poitiers, France

CHU de Reims - American Memorial Hospital 2

Reims, France

Centre Eugène Marquis

Rennes, France

CHU Rennes - Hôpital Sud

Rennes, France

CHU Rouen Normandie - Hôpital Charles-Nicolle

Rouen, France

CHU de Saint-Denis de La Réunion

Saint-Denis, France

CHU de Saint-Etienne - Hôpital Nord

Saint-Etienne, France

CHU de Saint-Pierre de La Réunion Sud

Saint-Pierre, France

Centre Régional Lutte Contre le Cancer Paul STRAUSS

Strasbourg, France

Hôpital de Hautepierre

Strasbourg, France

Hôpital Foch

Suresnes, France

Hôpital des enfants

Toulouse, France

Institut Universitaire du Cancer de Toulouse-Oncopole (IUCT-O) - Institut Claudius Regaud

Toulouse, France

CHRU Tours - Hôpital Clocheville

Tours, France

CHRU Tours - Hôpital Bretonneau

Tours, France

Hospital Vall D´Hebron

Barcelona, Spain

Hospital Universitario Niño Jesus

Madrid, Spain

Hospital Universitario y Politécnico de La Fe

Valencia, Spain

Karolinska University Hospital

Stockholm, Sweden

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NCT05476939


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