Tailored therapy for medulloblastoma shows promise but trial cut short
NCT ID NCT04402073
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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A.O Landeskrankenhaus - Innsbruck Universitaetsklinik
Innsbruck, Austria
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AKH unikliniken
Vienna, Austria
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AUSL Bologna - Ospedale Bellaria
Bologna, Italy
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Austin Health - Austin hospital
Melbourne, Australia
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Azienda Ospedaliera Citta della Salute e della Scienza di Torino
Torino, Italy
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CHRU de Lille
Lille, France
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CHU de Nice - Hopital Pasteur
Nice, France
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CHU de Toulouse - Institut Claudius Regaud - IUCT oncopole
Toulouse, France
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Centre Hospitalier Universitaire Vaudois - Lausanne
Lausanne, Switzerland
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Centre Leon Berard
Lyon, France
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Erasmus MC
Rotterdam, Netherlands
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HELIOS Kliniken - HELIOS Klinikum Erfurt GmbH
Erfurt, Germany
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Hopital de La Timone (APHM)
Marseille, France
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Hopital la Pitie-Salpetriere
Paris, France
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Hospital Clinic de Barcelona
Barcelona, Spain
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Hospital Universitari Vall d'Hebron
Barcelona, Spain
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Hospital Universitario 12 De Octubre
Madrid, Spain
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Hospital Universitario Ramon y Cajal
Madrid, Spain
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IRCCS - Istituto Neurologico Carlo Besta
Milan, Italy
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Institut Catala d'Oncologia - ICO Badalona - Hospital Germans Trias i Pujol
Badalona, Spain
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Institut de Cancerologie de l'Ouest (ICO) - Saint Herblain
Saint-Herblain, France
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John Hunter Children's Hospital
New Lambton Heights, Australia
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Klinikum Rechts der isar Der Technische Universitaet Muenchen
Munich, Germany
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Knappschaft Krankenhaus Langendreer
Bochum, Germany
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Ludwig-Maximilians-Universitaet Muenchen - Campus Grosshadern
Munich, Germany
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Peter Maccallum Cancer Institute
Melbourne, Australia
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Prince Of Wales Hospital
Sydney, Australia
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Princess Alexandra Hospital - University Of Queensland
Brisbane, Australia
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Royal Adelaide Hospital
Adelaide, Australia
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Royal North Shore Hospital
Sydney, Australia
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Sir Charles Gairdner Hospital
Nedlands, Australia
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Sydney Children's Hospital
Sydney, Australia
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ULSS2 - Marca Trevigniana
Treviso, Italy
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Univ. Mainz - Universitaetsmedizin der Johannes Gutenberg Universitaet Mainz-University Medical Center
Mainz, 55131, Germany
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Univ. of Florence -Azienda Ospedaliero-Universitaria Careggi
Florence, Italy
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Universitaets Krankenhaus Eppendorf - Universitaetsklinikum Hamburg-Eppendorf KE - University Cancer Center
Hamburg, Germany
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UniversitaetsMedizin Mannheim
Mannheim, Germany
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Universitaetskliniken Regensburg - Universitaetsklinikum Regensburg
Regensburg, 93053, Germany
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Universitaetsklinikum - Essen
Essen, 45147, Germany
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Universitaetsklinikum Bonn
Bonn, Germany
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Universitaetsklinikum Carl Gustav Carus
Dresden, Germany
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Universitaetsklinikum Freiburg - Klinik fuer Neurochirurgie
Freiburg im Breisgau, Germany
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Universitaetsklinikum Heidelberg - UniversitaetsKlinikum Heidelberg - Head Hospital
Heidelberg, Germany
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Universitaetsklinikum Leipzig-Klinik fuer Strahlentherapie und Radioonkologie
Leipzig, Germany
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Universitaetsklinikum Tuebingen- Crona Kliniken
Tübingen, Germany
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Universitaetsmedizin Goettingen - Georg-August Universitaet
Goettigen, Germany
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Universitair Medisch Centrum Groningen
Groningen, Netherlands
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University Frankfurt - Goethe Univ. - University Hospital Frankfurt -Senckenberg Institute of Neurooncology
Frankfurt, Germany
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University Hospital zurich
Zurich, 8091, Switzerland
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Westmead Hospital - Crown Princess Mary Cancer Center
Westmead, Australia