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Tailored therapy for medulloblastoma shows promise but trial cut short

NCT ID NCT04402073

Disease control Sponsor: European Organisation for Research and Treatment of Cancer - EORTC Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 trial tested a personalized treatment approach for post-pubertal patients with newly diagnosed medulloblastoma, a rare brain cancer. The experimental group received a targeted drug called sonidegib along with reduced radiation, based on the tumor's genetic subtype. The study aimed to improve survival and reduce side effects, but it was terminated early after enrolling only 20 participants, limiting what can be concluded.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Sonidegib (a targeted drug that blocks a specific cancer pathway), cisplatin, and lomustine (chemotherapy drugs)
What this could lead to
If successful, this approach could lead to more effective, less toxic treatments for medulloblastoma in adolescents and young adults by tailoring therapy to the tumor's genetic profile.
What could go wrong
The trial was terminated early with only 20 participants, so results are very limited. It is unclear if the personalized approach improves outcomes or reduces side effects compared to standard care.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

20 people

The number who actually took part.

Started

Nov 2022

Finished

Sep 2025

Lead sponsor

A research network

The lead sponsor is a research network or cooperative group.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

15 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Newly diagnosed, histologically proven, genetically classified, centrally confirmed medulloblastoma (WNT M0-1, SHH M0-1 (p53wt), Group 4 M0-1) * Molecular subtype: medulloblastoma, SHH-activated and TP53-wildtype, M0-1; medulloblastoma, WNT-activated, M0-1; medulloblastoma, Group 4, M0-1 * Histologic subtype: medulloblastoma, classic (CMB); medulloblastoma, desmoplastic/nodular (DNMB); medulloblastoma, with extensive nodularity (MBEN); medulloblastoma, large cell/anaplastic (LCA) * Adult (18 years and above): in WNT-activated and Group 4 medulloblastoma * Post-pubertal, defined as females with a bone age of at least 15 years and males with a bone age of at least 17 years, or adult (greater than 18 y of age) (see appendix N) in SHH-activated and TP53-wildtype medulloblastoma * Availability of prognostic markers (MYC/MYCN amplification, MYC/MYCN mutation) * Availability of paraffin embedded tumour tissue (FFPE) (1 block or 30 unstained slides) and whole blood sample (10 ml) for central review * For patients with SHH activated tumours: exclusion of germline alteration of TP53, PTCH, SUFU, BRCA2 and PALB2 if known before randomization * Clinical status within 2 weeks of randomization: Karnofsky 50-100. NANO-score 0 to 9 (allowing full-blown cerebellar symptoms) * Clinically standard-risk (centrally assessed MRI review) defined as: total or near total surgical resection with less than or equal to 1.5 cm2 (measured in axial plane) of residual tumour on early post-operative MRI, without and with contrast; no CNS metastasis on MRI (cranial and spinal); Chang stage M0-1 with no clinical evidence of extra-CNS metastasis * Full recovery from surgery or any post-surgical complication (e.g. Bleeding, infections etc) * Pre-surgery and/or post-surgery MRI available. * Baseline brain MRI and spinal MRI available within 2 weeks of randomization. * Normal liver, renal and haematological function within 2 weeks of randomization. * WBC greater than or equal to 3×10\^9/L * ANC greater than or equal to 1.5×10\^9/L * Platelet count of greater than or equal to 100×10\^9/L independent of transfusion * Hemoglobin greater than or equal to 10 g/dl * Total Bilirubin less than or equal to 1.5 ULN * ALT (SGPT), AST (SGOT), alkaline phosphatase (ALP) less than or equal to 2.5 × ULN * Serum creatinine less than 1.5 x ULN or creatinine clearance (CrCl) greater than 30 mL/min (using the Cockcroft-Gault formula) * Negative serum or urine pregnancy test within 7 days of randomization for WOCBP. * Patients of childbearing / reproductive potential (WOCBP) must use two methods of adequate birth control, including a highly effective method and a barrier method during the study treatment period and for at least 20 months after the last study treatment is mandatory for the patients that received sonidegib, for all other patients this period is at least 6 months after the last study treatment. A highly effective method of birth control is defined as those which result in low failure rate (i.e. less than 1% per year) when used consistently and correctly. Male patients even those who have had a vasectomy must always use a condom during treatment and for 6 months after last treatment. Men should not donate semen during treatment and for at least 6 months after ending treatment (donation of semen for the semen analyses of the fertility project 1 b is allowed). Appendix H. * Female subjects who are breast feeding must discontinue nursing prior to the first dose of study treatment and until 20 months after the last study treatment. * Before patient registration/randomization, written informed consent must be given according to ICH/GCP, and national/local regulations. For patients less than 18 years of age, consent has to be obtained from the parent(s) or legal representative. Exclusion Criteria: * Prior treatment for medulloblastoma * Unavailability of central review pathology results. * Inability to start radiotherapy within 43 days of surgery * Significant sensorineural hearing deficit as defined by pure tone audiometry with bone conduction or air conduction and normal tympanogram showing impairment greater than or equal to 20 dB at 1-3 kHz * Any medical contraindication to radiotherapy or chemotherapy. * Hypersensitivity to contrast medium for MRI. * Hypersensitivity towards the active substance of any of study drugs or their excipients * Prior or current use of mitoxantrone, methotrexate, topotecan, imatinib, irinotecan or statins * Concurrent severe or uncontrolled medical disease (e.g., active systemic infection, diabetes, psychiatric disorder) that, in the opinion of the investigator, would compromise the safety of the patient or compromise the ability of the patient to complete the study * Prior or second invasive malignancy, except non-melanoma skin cancer, completely resected cervical carcinoma in situ, low risk prostate cancer (cT1-2a N0 and Gleason score less than or equal to 6 and PSA less than 10 ng/mL), either totally resected or irradiated with curative intent (with PSA of less than or equal to 0.1 ng/mL) or under active surveillance as per ESMO guidelines. Other cancers for which the subject has completed potentially curative treatment more than 5 years prior to diagnosis of medulloblastoma study entry are allowed * Known history or current evidence of active Hepatitis B (e.g., positive HBV surface antigen) or C (e.g., HCV RNA \[qualitative\] is detected) * Known or current evidence of Human Immunodeficiency Virus (HIV) infection (positive HIV-1/2 antibodies) * Presence of any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule; those conditions should be discussed with the patient before registration in the trial.

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Conditions

The condition(s) this trial relates to.

medulloblastoma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Medulloblastoma

Contacts and locations

Locations

  • A.O Landeskrankenhaus - Innsbruck Universitaetsklinik

    Innsbruck, Austria

  • AKH unikliniken

    Vienna, Austria

  • AUSL Bologna - Ospedale Bellaria

    Bologna, Italy

  • Austin Health - Austin hospital

    Melbourne, Australia

  • Azienda Ospedaliera Citta della Salute e della Scienza di Torino

    Torino, Italy

  • CHRU de Lille

    Lille, France

  • CHU de Nice - Hopital Pasteur

    Nice, France

  • CHU de Toulouse - Institut Claudius Regaud - IUCT oncopole

    Toulouse, France

  • Centre Hospitalier Universitaire Vaudois - Lausanne

    Lausanne, Switzerland

  • Centre Leon Berard

    Lyon, France

  • Erasmus MC

    Rotterdam, Netherlands

  • HELIOS Kliniken - HELIOS Klinikum Erfurt GmbH

    Erfurt, Germany

  • Hopital de La Timone (APHM)

    Marseille, France

  • Hopital la Pitie-Salpetriere

    Paris, France

  • Hospital Clinic de Barcelona

    Barcelona, Spain

  • Hospital Universitari Vall d'Hebron

    Barcelona, Spain

  • Hospital Universitario 12 De Octubre

    Madrid, Spain

  • Hospital Universitario Ramon y Cajal

    Madrid, Spain

  • IRCCS - Istituto Neurologico Carlo Besta

    Milan, Italy

  • Institut Catala d'Oncologia - ICO Badalona - Hospital Germans Trias i Pujol

    Badalona, Spain

  • Institut de Cancerologie de l'Ouest (ICO) - Saint Herblain

    Saint-Herblain, France

  • John Hunter Children's Hospital

    New Lambton Heights, Australia

  • Klinikum Rechts der isar Der Technische Universitaet Muenchen

    Munich, Germany

  • Knappschaft Krankenhaus Langendreer

    Bochum, Germany

  • Ludwig-Maximilians-Universitaet Muenchen - Campus Grosshadern

    Munich, Germany

  • Peter Maccallum Cancer Institute

    Melbourne, Australia

  • Prince Of Wales Hospital

    Sydney, Australia

  • Princess Alexandra Hospital - University Of Queensland

    Brisbane, Australia

  • Royal Adelaide Hospital

    Adelaide, Australia

  • Royal North Shore Hospital

    Sydney, Australia

  • Sir Charles Gairdner Hospital

    Nedlands, Australia

  • Sydney Children's Hospital

    Sydney, Australia

  • ULSS2 - Marca Trevigniana

    Treviso, Italy

  • Univ. Mainz - Universitaetsmedizin der Johannes Gutenberg Universitaet Mainz-University Medical Center

    Mainz, 55131, Germany

  • Univ. of Florence -Azienda Ospedaliero-Universitaria Careggi

    Florence, Italy

  • Universitaets Krankenhaus Eppendorf - Universitaetsklinikum Hamburg-Eppendorf KE - University Cancer Center

    Hamburg, Germany

  • UniversitaetsMedizin Mannheim

    Mannheim, Germany

  • Universitaetskliniken Regensburg - Universitaetsklinikum Regensburg

    Regensburg, 93053, Germany

  • Universitaetsklinikum - Essen

    Essen, 45147, Germany

  • Universitaetsklinikum Bonn

    Bonn, Germany

  • Universitaetsklinikum Carl Gustav Carus

    Dresden, Germany

  • Universitaetsklinikum Freiburg - Klinik fuer Neurochirurgie

    Freiburg im Breisgau, Germany

  • Universitaetsklinikum Heidelberg - UniversitaetsKlinikum Heidelberg - Head Hospital

    Heidelberg, Germany

  • Universitaetsklinikum Leipzig-Klinik fuer Strahlentherapie und Radioonkologie

    Leipzig, Germany

  • Universitaetsklinikum Tuebingen- Crona Kliniken

    Tübingen, Germany

  • Universitaetsmedizin Goettingen - Georg-August Universitaet

    Goettigen, Germany

  • Universitair Medisch Centrum Groningen

    Groningen, Netherlands

  • University Frankfurt - Goethe Univ. - University Hospital Frankfurt -Senckenberg Institute of Neurooncology

    Frankfurt, Germany

  • University Hospital zurich

    Zurich, 8091, Switzerland

  • Westmead Hospital - Crown Princess Mary Cancer Center

    Westmead, Australia

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Other studies related to the condition(s) this trial covers.