Brain (Nervous System) CancersPhase 2EViDENCE-BTNCT06804655
Pharmacoscopy for Patients With Refractory Primary Brain Tumors
A Phase 2 study testing Pharmacoscopy 1.0 in people with Brain (Nervous System) Cancers and Glioblastoma, run by University of Zurich at 3 sites in Switzerland.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Brain (Nervous System) Cancers, Glioblastoma, Glioma
- What is being tested?
- Pharmacoscopy 1.0
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 3 sitesSwitzerland
- Study dates
- 11 Sep 2026to 15 Sep 2028
- Phase
- Phase 2
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Switzerland3 sites, 3 recruiting
- Not yet recruiting
University Hospital Basel
Basel, Basel-Landschaft, 4021
Gregor Hutter, Prof. Dr. med., +41612657124, Gregor.Hutter@usb.ch
- Not yet recruiting
Cantonal Hospital St Gallen
Sankt Gallen, Canton of St. Gallen, 9007
Marian Neidert, PD Dr. med., +41714941199, marian.neidert@kssg.ch
- Recruiting
University Hospital Zurich
Zurich, Canton of Zurich, 8091
Emilie Le Rhun, PD Dr med, +41442553899, EViDENCE-BT@usz.ch
About this study
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
- Being tested
- Pharmacoscopy 1.0
- Keywords
- pharmacoscopy, drug testing, refractory tumor, drug repurposing, personalized medicine
- Medical subject headings
- Neurologic Manifestations, Neoplasms, Glioblastoma, Glioma, Ependymoma, Medulloblastoma, Meningioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pharmacoscopy 1.0Device
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Device: Pharmacoscopy 1.0 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 2 months
Secondary outcomes (9)
To explore the efficacy of pharmacoscopy-guided treatment - 1
(1) Response rate
Measured: 12 months
To explore the efficacy of pharmacoscopy guided treatment - 2
duration of response
Measured: 12 months
To explore the efficacy of pharmascopy guided treatment - 3
progression-free survival
Measured: 12 months
To explore the efficacy of pharmascopy guided treatment - 4
overall survival
Measured: 24 months
To confirm the safety and tolerability of treatment selected based on pharmacoscopy - 1
Neurological function (NANO scale, Nayak et al. 2017)
Measured: 12 months
To confirm the safety and tolerability of treatment selected based on pharmacoscopy -2
Karnofsky performance status (score 0-100)
Measured: 12 months
To confirm the safety and tolerability of treatment selected based on pharmacoscopy - 3
Steroid use (dose in mg equivalent dexamethasone)
Measured: 12 months
Quality of life -1
EORTC QLQ C30 auto-questionnaire
Measured: 12 months
Quality of life - 2
EORTC BN20 autoquestionnaire
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2025
- Study start11 Sep 2026
- Primary completion15 Sep 2028 (estimated)
- Study completion15 Sep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Brain (Nervous System) CancersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Zurich
- Sponsor type
- University, hospital or other
- Collaborators
- University Hospital, Zürich, University Hospital, Basel, Switzerland, Cantonal Hospital of St. Gallen
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06804655
- Sponsor's protocol ID
- EViDENCE-BT
- Acronym
- EViDENCE-BT
- Status last verified
- Sep 2026
- First submitted
- 17 Dec 2024
- Last updated
- 21 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
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.
PubMed 39304781