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Study on Personalized Treatment for Post-Pubertal Patients with Newly Diagnosed Medulloblastoma Using Sonidegib, Cisplatin, Lomustine, and Vincristine

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What is this study about?

This clinical trial is focused on studying a type of brain cancer called medulloblastoma, which is newly diagnosed in patients who are post-pubertal or adults. The study aims to explore a personalized treatment approach based on the patient’s age and specific characteristics of the tumor. The treatment involves adjusting the doses of radiation therapy and chemotherapy and introducing an investigative drug called sonidegib. The trial will compare this personalized treatment to the standard therapy in a subgroup of patients with a specific type of medulloblastoma known as SHH-activated.

Participants in the study will receive different medications, including cisplatin, lomustine, and vincristine, alongside the investigative drug sonidegib. Cisplatin is administered as a concentrate for infusion, lomustine is taken orally, and vincristine is given as an injection. The study will also involve a placebo group to help determine the effectiveness of the new treatment approach. The main goal is to see if the personalized treatment can help patients live longer without the cancer getting worse, which is known as progression-free survival.

The study will take place over several weeks, with regular monitoring and assessments to track the progress of the disease and any side effects of the treatment. Participants will undergo various tests, including MRI scans, to evaluate the tumor’s response to the treatment. The trial will also collect information on the overall survival of patients and any adverse events they may experience. Additionally, the study will assess the impact of the treatment on patients’ quality of life and cognitive functions. The trial is expected to continue until 2030, with the aim of providing valuable insights into the effectiveness of personalized treatment for medulloblastoma.

1 Initial registration

Upon joining the study, informed consent must be provided. For those under the legal age, consent is required from a parent or legal representative.

Tumor tissue and blood samples are collected for central pathology review.

2 Enrollment and randomization

Eligibility is confirmed through clinical assessments, including MRI scans to evaluate the extent of tumor resection and absence of metastasis.

Baseline health assessments are conducted, including liver, kidney, and blood function tests.

For women of childbearing potential, a pregnancy test is required, and effective birth control methods must be used during and after the study.

3 Treatment phase

Participants are assigned to either the experimental group receiving sonidegib or the standard therapy group.

The experimental group receives Odomzo 200 mg hard capsules for oral use, which contains the active substance sonidegib.

Standard therapy may include medications such as cisplatin (concentrate for solution for infusion), lomustine (oral use), and vincristine (solution for injection).

4 Monitoring and follow-up

Regular monitoring of health status and treatment response is conducted through clinical evaluations and imaging studies.

Participants report any side effects or changes in health, which are assessed according to standardized criteria.

5 Post-treatment observation

After completing the treatment phase, participants continue to be monitored for progression-free survival and overall survival.

Health-related quality of life and neurocognitive function are evaluated using specific tests and questionnaires.

Who Can Join the Study?

  • Before joining the study, you or your legal representative must give written permission, following specific guidelines and rules.
  • Your health condition should be stable within two weeks before joining the study, with a score between 50 and 100 on a scale called the Karnofsky scale, which measures your ability to perform daily activities.
  • Your NANO-score, which assesses neurological function, should be between 0 and 9, even if you have full cerebellar symptoms, which affect balance and coordination.
  • You should have a standard-risk medulloblastoma, confirmed by MRI scans before and after surgery, showing that most of the tumor has been removed and there are no signs of spread to the central nervous system (CNS).
  • There should be no signs of the cancer spreading outside the CNS.
  • You must have fully recovered from surgery or any complications like bleeding or infections.
  • Baseline MRI scans of the brain and spine are required, with specific timing for when these scans should be done.
  • Your liver, kidney, and blood functions should be normal within two weeks before joining the study, with specific blood count and chemical levels.
  • If you are a woman who can have children, you must have a negative pregnancy test within seven days before joining the study.
  • Women who can have children must use two methods of birth control during the study and for a certain period after the last treatment, depending on the treatment received.
  • Men, even those who have had a vasectomy, must use a condom during treatment and for 12 months after the last treatment. Men should not donate semen during this time.
  • Tumor tissue and blood samples from surgery must be available for review.
  • Women who are breastfeeding must stop nursing before starting the study treatment and until 20 months after the last treatment.
  • You must have a newly diagnosed, confirmed type of brain tumor called medulloblastoma.
  • The tumor must be of a specific molecular subtype, such as SHH-activated or WNT-activated, and not have spread beyond certain areas.
  • The tumor must be of a specific histologic subtype, like classic or large cell/anaplastic medulloblastoma.
  • You must be a post-pubertal patient under 18 years old or an adult with SHH-activated medulloblastoma.
  • Adults aged 18 and above with WNT-activated or other specific types of medulloblastoma can participate.
  • If you are under 18, you must have completed puberty.
  • If you are under 18, your bone age must be at least 15 years for females and 17 years for males, confirmed by radiology.

Who Cannot Join the Study?

  • Patients who have not been diagnosed with medulloblastoma, a type of brain tumor, cannot participate.
  • Patients who are not in the SHH-activated subgroup, which is a specific category of medulloblastoma, are excluded.
  • Patients who are not post-pubertal, meaning they have not gone through puberty, are not eligible.
  • Patients who do not have a standard risk level of medulloblastoma, which refers to a specific level of disease severity, cannot join the study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Universitaetsklinikum Heidelberg AöR Heidelberg Germany

Other Sites

Site Name City Country Status
Universitaetsklinikum Regensburg AöR Regensburg Germany
Universitaetsklinikum Tuebingen AöR Tuebingen Germany
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Updfuycvqj Mdywyyk Cjmgql Hblrsvglhmfnvuymj Hamburg Germany
Uyzrgzwzqvjtqtmgqfzmj Eoypv Ada Essen Germany
Gwpngv Uegrnpkjmp Fkltgfvja Frankfurt Germany
Krhawwji dcv Ubypbvroraos Mcnvjlie Aoc Munich Germany

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Germany Germany
Not recruiting
10.11.2022

Trial locations

Investigated drugs:

Sonidegib is an experimental medication being tested in this clinical trial. It is used as part of a personalized intensity-modulated therapy for patients with newly diagnosed medulloblastoma, specifically in the SHH-activated subgroup. The goal of using sonidegib is to improve progression-free survival, which means extending the time patients live without the cancer getting worse.

Investigated diseases:

Medulloblastoma – Medulloblastoma is a type of brain tumor that originates in the cerebellum, the part of the brain responsible for coordination and balance. It is most commonly diagnosed in children but can also occur in adults. The disease is characterized by rapid growth and can spread to other parts of the brain and spinal cord. Medulloblastomas are classified into different subgroups based on genetic and molecular features, such as SHH-activated, WNT, and Groups 3 and 4. These subgroups can influence the behavior and progression of the tumor. The progression of medulloblastoma can vary, with some subtypes being more aggressive than others.

Trial ID:
2023-506193-12-00
Protocol code:
1634
NCT ID:
NCT04402073
Trial Phase:
Therapeutic exploratory (Phase II)

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