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MedulloblastomaPhase 2PersoMed-INCT04402073

Personalized Risk-Adapted Therapy in Post-Pubertal Patients With Newly-Diagnosed Medulloblastoma

A Phase 2 study testing Sonidegib, Cisplatin and Lomustine in people with Medulloblastoma, run by European Organisation for Research and Treatment of Cancer - EORTC at 50 sites in 8 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
Search all trials

Trial summary

Medulloblastoma is a rare brain malignancy, mainly affecting children. Treatment of this rapidly growing tumor begins with maximal surgical removal plus radiation and chemotherapy. Treatment toxicity is high. Read more about this study

Who is it for?
Ages 15 years and older
Conditions the study is for
Medulloblastoma
What is being tested?
Sonidegib, Cisplatin and Lomustine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
50 sitesin 8 countries
Study dates
11 Nov 2022to 10 Sep 2025
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 15 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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)
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  • 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.

Who cannot join

  • 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.
Show all 13Show fewer
  • 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.

Trial locations and contacts

50 sites in 8 countries.

50 sites in 8 countries

Germany17 sites

  • Knappschaft Krankenhaus Langendreer

    Bochum

  • Universitaetsklinikum Bonn

    Bonn

  • Universitaetsklinikum Carl Gustav Carus

    Dresden

  • HELIOS Kliniken - HELIOS Klinikum Erfurt GmbH

    Erfurt

  • Universitaetsklinikum - Essen

    Essen, 45147

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

    Frankfurt

  • Universitaetsklinikum Freiburg - Klinik fuer Neurochirurgie

    Freiburg im Breisgau

  • Universitaetsmedizin Goettingen - Georg-August Universitaet

    Goettigen

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

    Hamburg

  • Universitaetsklinikum Heidelberg - UniversitaetsKlinikum Heidelberg - Head Hospital

    Heidelberg

  • Universitaetsklinikum Leipzig-Klinik fuer Strahlentherapie und Radioonkologie

    Leipzig

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

    Mainz, 55131

  • UniversitaetsMedizin Mannheim

    Mannheim

  • Klinikum Rechts der isar Der Technische Universitaet Muenchen

    Munich

  • Ludwig-Maximilians-Universitaet Muenchen - Campus Grosshadern

    Munich

  • Universitaetskliniken Regensburg - Universitaetsklinikum Regensburg

    Regensburg, 93053

  • Universitaetsklinikum Tuebingen- Crona Kliniken

    Tübingen

Other trials in Germany

Australia10 sites

  • Royal Adelaide Hospital

    Adelaide

  • Princess Alexandra Hospital - University Of Queensland

    Brisbane

  • Austin Health - Austin hospital

    Melbourne

  • Peter Maccallum Cancer Institute

    Melbourne

  • Sir Charles Gairdner Hospital

    Nedlands

  • John Hunter Children's Hospital

    New Lambton Heights

  • Prince Of Wales Hospital

    Sydney

  • Royal North Shore Hospital

    Sydney

  • Sydney Children's Hospital

    Sydney

  • Westmead Hospital - Crown Princess Mary Cancer Center

    Westmead

Other trials in Australia

France7 sites

  • CHRU de Lille

    Lille

  • Centre Leon Berard

    Lyon

  • Hopital de La Timone (APHM)

    Marseille

  • CHU de Nice - Hopital Pasteur

    Nice

  • Hopital la Pitie-Salpetriere

    Paris

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

    Saint-Herblain

  • CHU de Toulouse - Institut Claudius Regaud - IUCT oncopole

    Toulouse

Other trials in France

Italy5 sites

  • AUSL Bologna - Ospedale Bellaria

    Bologna

  • Univ. of Florence -Azienda Ospedaliero-Universitaria Careggi

    Florence

  • IRCCS - Istituto Neurologico Carlo Besta

    Milan

  • Azienda Ospedaliera Citta della Salute e della Scienza di Torino

    Torino

  • ULSS2 - Marca Trevigniana

    Treviso

Other trials in Italy

Spain5 sites

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

    Badalona

  • Hospital Clinic de Barcelona

    Barcelona

  • Hospital Universitari Vall d'Hebron

    Barcelona

  • Hospital Universitario 12 De Octubre

    Madrid

  • Hospital Universitario Ramon y Cajal

    Madrid

Other trials in Spain

Austria2 sites

  • A.O Landeskrankenhaus - Innsbruck Universitaetsklinik

    Innsbruck

  • AKH unikliniken

    Vienna

Other trials in Austria

Netherlands2 sites

  • Universitair Medisch Centrum Groningen

    Groningen

  • Erasmus MC

    Rotterdam

Other trials in Netherlands

Switzerland2 sites

  • Centre Hospitalier Universitaire Vaudois - Lausanne

    Lausanne

  • University Hospital zurich

    Zurich, 8091

Other trials in Switzerland

About this study

Official title: Personalized Risk-Adapted Therapy in Post-Pubertal Patients With Newly-Diagnosed Medulloblastoma (PersoMed-I)

Medulloblastoma is a rare brain malignancy, mainly affecting children. Treatment of this rapidly growing tumor begins with maximal surgical removal plus radiation and chemotherapy. Treatment toxicity is high. Post-pubertal and pediatric medulloblastomas are biologically and prognostically different, which mandates age-adapted treatment strategies. Patients after puberty bear an intermediate to high prognostic risk. This means that a large number of these patients, are faced with death and/or disability (mainly neurocognitive). Therefore, the scientific and medical need is high. One of the genetic subgroups of medulloblastoma, the SHH-subgroup (Sonic HedgeHog- subgroup), is highly overrepresented in medulloblastoma patients after puberty. This subgroup can be treated with a targeted therapy. The investigators will therefore randomize patients and treat SHH-subgroup patients with sonidegib and a reduction of radiotherapy dose in the experimental arm of the trial. The hypothesis that this personalized risk-adapted therapy will improve outcomes in view of increased efficacy and decreased toxicity.

Conditions
Medulloblastoma
Being tested
Sonidegib, Cisplatin, Lomustine, Vincristine, radiotherapy
Keywords
brain, medullobalstoma, MRI, radiotherapy, biomarkers
Medical subject headings
Medulloblastoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • SonidegibDrug

    Sonidegib is a selective smoothened inhibitor that inhibits the sonic hedgehog-signaling pathway. It is used in patients with locally advanced basal cell carcinoma (BCC) that has recurred following surgery or radiation therapy, or those who are not candidates for surgery or radiation therapy.

  • CisplatinDrug

    Cisplatin (CIS) is a platinum derivate used in the treatment in several epithelial tumours. The application route is through the veins.

  • LomustineDrug

    Lomustine is a nitrosourea used in the treatment of brain tumours and Hodgkin's disease. The application route is oral.

  • VincristineDrug

    Vincristine sulfate (VCR) is an inhibitor of microtubule formation in the mitotic spindle, resulting in an arrest of dividing cells at the metaphase stage. Vincristine sulfate is indicated in acute leukemia, Hodgkin's disease, non-Hodgkin's lymphomas, rhabdomyosarcoma, neuroblastoma, and Wilms' tumor. The application route is through the vein.

  • radiotherapyRadiation

    Radiotherapy to the cranio-spinal axis of 35.2 Gy in 22 daily fractions of 1.6 Gy, followed by an additional boost to the tumour site of 19.8 Gy in 11 daily fractions of 1.8 Gy, summing up to a total dose of 55.0 Gy in 33 daily fractions of 1.6/1.8 Gy.

Study groups in NCT04402073
Study groupRoleWhat they receive
standard armsOtherCriteria: Adult SHH (p53wt) M0-1, adult WNT M0-1, adult Group 4 M0-1. Radiotherapy to the cranio-spinal axis of 35.2 Gy in 22 daily fractions of 1.6 Gy, followed by an additional boost to the tumour site of 19.8 Gy in 11 daily fractions of 1.8 Gy, summing up to a total dose of 55.0 Gy in 33 daily fractions of 1.6/1.8 Gy. Criteria: Post pubertal < 18 y SHH (p53wt) M0. Radiotherapy to the cranio-spinal axis of 23.4 Gy in 13 daily fractions of 1.8 Gy, followed by an additional boost to the tumour site of 30.6 Gy in 17 daily fractions of 1.8 Gy, summing up to a total dose of 54.0 Gy in 30 daily fractions of 1.8 Gy.
Receives: Drug: Cisplatin; Drug: Lomustine; Drug: Vincristine; Radiation: radiotherapy
experimental armsExperimentalRadiotherapy Criteria: Adult and post-pubertal SHH (p53wt) M0; adult WNT M0, adult Group 4 M0. Radiotherapy to the cranio-spinal axis of 23.4 Gy in 13 daily fractions of 1.8 Gy, followed by an additional boost to the tumour site of 30.6 Gy in 17 daily fractions of 1.8 Gy, summing up to a total dose of 54.0 Gy in 30 daily fractions of 1.8 Gy. SMO-inhibitor Criteria: Adult and post-pubertal SHH (p53wt) M0. Sonidegib 200 mg/day (daily) from first day of radio-chemotherapy until end of maintenance chemotherapy, including 6w chemotherapy break.
Receives: Drug: Sonidegib; Drug: Cisplatin; Drug: Lomustine; Drug: Vincristine; Radiation: radiotherapy

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Progression Free Survival

    compare progression-free survival (PFS) by central review of a personalized intensity-modulated therapy (experimental arm; sonidegib) vs. standard therapy in the SHH-activated subgroup in post-pubertal patients with newly diagnosed standard risk medulloblastoma.

    Measured: 91 months after the date of recruitment of the first patient

Secondary outcomes (5)

  1. Progression Free Survival (PFS)

    Measured: when 43 PFS events are observed which is estimated to occur 91 months after the date of recruitment of the first patient.

  2. overall survival (OV)

    Measured: when 43 PFS events are observed which is estimated to occur 91 months after the date of recruitment of the first patient.

  3. safety and tolerability profile: CTCAE

    This study will use the International Common Terminology Criteria for Adverse Events (CTCAE), version 5.0, for adverse event reporting (with neurological, kidney, auditory, endocrine and radiotherapy associated as AE of special interest). The highest CTCAE v. 5 grading per cycle and per patient will be computed at the EORTC HQ for analysis . Safety and tolerability analyses will be performed in the safety population. Severe grades (3/4) which did not resolve after treatment discontinuation or emerged during follow-up will be identified and listed.

    Measured: when 43 PFS events are observed which is estimated to occur 91 months after the date of recruitment of the first patient.

  4. health-related quality of life (HRQoL)

    The primary HRQoL endpoint that is considered relevant for this study is social functioning. The other scales from the QLQ-C30 and BN20 will be considered as exploratory in nature.

    A difference of 10 points on the 100-point QLQ-C30 social functioning scale between the two arms will be considered as clinically relevant.

    Measured: when 43 PFS events are observed which is estimated to occur 91 months after the date of recruitment of the first patient.

  5. overall survival

    Measured: when 43 PFS events are observed which is estimated to occur 91 months after the date of recruitment of the first patient.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 May 2020
  2. Study start11 Nov 2022
  3. Primary completion16 Jun 2025
  4. Study completion10 Sep 2025

Why it stopped: Poor accrual

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Health services research
Allocation
Randomised
Intervention model
Parallel groups
Model description
Phase II, comparative, randomized (experimental : standard arm 1:1, no blinding, no active or placebo control, parallel group, therapeutic
Masking
None (open label)
Enrolment
20 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04402073
Sponsor's protocol ID
1634
Acronym
PersoMed-I
Status last verified
Oct 2025
First submitted
1 Apr 2020
Last updated
23 Oct 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Sonidegib, Cisplatin and Lomustine in people with Medulloblastoma, run by European Organisation for Research and Treatment of Cancer - EORTC at 50 sites in 8 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 25 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 23 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 50 sites in Germany, Australia, France, Italy, Spain and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Poor accrual). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Progression Free Survival (time frame: 91 months after the date of recruitment of the first patient). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04402073.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Oct 2025; AzTrials last refreshed its copy on 6 Nov 2026.

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