Pharmacoscopy for Patients With Refractory Primary Brain Tumors

NCT06804655 | Not Yet Recruiting Phase 2

• 1 other identifier: EViDENCE-BT
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 3

Brief Summary

Advanced technology of ex vivo drug profiling referred to as pharmacoscopy may allow to identify novel drugs for the treatment of glioblastoma and other refractory brain tumors at an individual patient level. This personalized therapeutic approach was developed and validated in pre-clinical glioma models. With the current research proposal, we seek to establish feasibility for a clinical interventional trial for patients with refractory primary brain tumors that is based on pharmacoscopy-guided selection of treatment. The study is supported by an unrestricted grant from Anti Cancer Fund.

Conditions

Brain (Nervous System) Cancers; Glioblastoma; Glioma; Ependymoma; Medulloblastoma; Meningioma; Rare Primary Brain Tumors; Rare CNS Primary Tumors

Keywords

pharmacoscopy; drug testing; refractory tumor; drug repurposing; personalized medicine

Outcome Measures

Primary Outcomes (1)

• To demonstrate the overall feasibility of pharmacoscopy-guided treatment for patients with refractory primary brain tumors

  The feasibility is defined as: 80% success rate of generating a pharmacoscopy result within 10 days of surgery (32/40 patients), 50% success rate of placing patients on selected drug based on pharmacoscopy (16/32 patients).

  2 months

Secondary Outcomes (9)

• To explore the efficacy of pharmacoscopy-guided treatment - 1

  12 months

• To explore the efficacy of pharmacoscopy guided treatment - 2

  12 months

• To explore the efficacy of pharmascopy guided treatment - 3

  12 months

• To explore the efficacy of pharmascopy guided treatment - 4

  24 months

• To confirm the safety and tolerability of treatment selected based on pharmacoscopy - 1

  12 months

Other Outcomes (1)

• Translational research

  12 months

Study Arms (1)

All patients will have their tumor analyzed by pharmacoscopy.

EXPERIMENTAL

Therapeutic decisions will be taken considering the results of the pharmacoscopy analysis, and also standard histopathological and molecular analysis, including next generation sequencing, molecular profiling analysis as available, characteristics of the patient and previous treatments received.

Device: Pharmacoscopy 1.0

Interventions

Pharmacoscopy 1.0 DEVICE

The intervention is the submission of freshly obtained surgical material to ex vivo drug profiling which we term pharmacoscopy (PCY). The performance study is interventional as the test results shall influence patient management decisions and/or may be used to guide treatment.

All patients will have their tumor analyzed by pharmacoscopy.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 18 years or older on day of signing informed consent, female or male.
• Refractory glioblastoma, isocitrate dehydrogenase (IDH)-mutant astrocytoma or oligodendroglioma, histone-mutant glioma, ependymoma, medulloblastoma, meningioma or other rare primary brain tumor with a histological confirmation according to the WHO classification 2021. Primary tumors can be located at the cerebral or spinal level. Primary brain tumors with metastases outside of the brain may also be considered.
• Karnofsky performance status of 60 or more
• Life expectancy \>12 weeks.
• Limited systemic therapeutic options as per treating physician judgement. The number of previous lines of therapies is not limited.
• Surgery clinically indicated. A histological confirmation of the diagnosis of a recurrent brain tumor will be required before any treatment can be initiated.
• Adequate bone marrow, renal and hepatic function
• Ability to understand the requirements of the study, provide written informed consent and authorization of use and disclosure of protected health information, and agree to abide by the study restrictions and return for the required assessments.
• Written informed consent for study participation must be signed and dated by the patient and the investigator prior to any study-related intervention.

You may not qualify if:

• Inability to undergo brain or spine MRI.
• Concurrent treatment with other systemic tumor-directed pharmacotherapies.
• Intent to be treated with radiotherapy.
• Any investigational antitumor therapy other than those under investigation in this study.
• Judgment by the investigator that the patient should not participate in the study because the patient is unlikely to comply with study procedures, restrictions and requirements.
• Intention to become pregnant during the course of the study or pregnancy. Women of childbearing potential, including women who had their last menstruation in the last 2 years, must have a negative urinary or serum pregnancy test.
• Women who are breast feeding and who do not agree to discontinue nursing prior to the first study treatment and for the period defined in the protocol.
• Sexually active men and women of childbearing potential who are not willing to use an effective contraceptive method during the study.

Sponsors & Collaborators

• University of Zurich: lead
• University Hospital, Zürich: collaborator
• University Hospital, Basel, Switzerland: collaborator
• Cantonal Hospital of St. Gallen: collaborator

Study Sites (3)

University Hospital Basel

Basel, Basel-Landschaft, 4021, Switzerland

Location

Cantonal Hospital St Gallen

Sankt Gallen, Canton of St. Gallen, 9007, Switzerland

Location

University Hospital Zurich

Zurich, Canton of Zurich, 8091, Switzerland

Location

Related Publications (1)

• Lee S, Weiss T, Buhler M, Mena J, Lottenbach Z, Wegmann R, Sun M, Bihl M, Augustynek B, Baumann SP, Goetze S, van Drogen A, Pedrioli PGA, Penton D, Festl Y, Buck A, Kirschenbaum D, Zeitlberger AM, Neidert MC, Vasella F, Rushing EJ, Wollscheid B, Hediger MA, Weller M, Snijder B. High-throughput identification of repurposable neuroactive drugs with potent anti-glioblastoma activity. Nat Med. 2024 Nov;30(11):3196-3208. doi: 10.1038/s41591-024-03224-y. Epub 2024 Sep 20.

  PMID: 39304781 BACKGROUND

MeSH Terms

Conditions

Neurologic Manifestations; Neoplasms; Glioblastoma; Glioma; Ependymoma; Medulloblastoma; Meningioma

Condition Hierarchy (Ancestors)

Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Astrocytoma; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Neuroectodermal Tumors, Primitive; Neoplasms, Vascular Tissue; Meningeal Neoplasms; Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site

Central Study Contacts

Michael Weller, Prof. Dr. med.

CONTACT

+41442555500; EViDENCE-BT@usz.ch

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 17, 2024
• First Posted: February 3, 2025
• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): September 15, 2028
• Study Completion (Estimated): September 15, 2028
• Last Updated: May 6, 2026

Record last verified: 2026-04

Locations

CH (3)