Clinical trial · Interventional
Pharmacoscopy for Patients With Refractory Primary Brain Tumors
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 26, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260926-000001
Summary
Brief summary (as posted)
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
Conditions (8)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Brain (Nervous System) Cancers | — | UNRESOLVED | — |
| Ependymoma | Ependymoma | ONTOLOGY_EXACT | 0.90 |
| Glioblastoma | Glioblastoma | CURATED_BROADER | 0.80 |
| Glioma | Glioma | ONTOLOGY_EXACT | 0.98 |
| Medulloblastoma | Medulloblastoma | CURATED_BROADER | 0.80 |
| Meningioma | Meningioma | ONTOLOGY_EXACT | 0.90 |
| Rare CNS Primary Tumors | — | UNRESOLVED | — |
| Rare Primary Brain Tumors | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Pharmacoscopy 1.0 | Device | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- All patients will have their tumor analyzed by pharmacoscopy.
- description
- 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.
- interventionNames
- Device: Pharmacoscopy 1.0
Primary outcomes (1)
- measure
- To demonstrate the overall feasibility of pharmacoscopy-guided treatment for patients with refractory primary brain tumors
- timeFrame
- 2 months
- description
- 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).
Secondary outcomes (9)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: 1. Age 18 years or older on day of signing informed consent, female or male. 2. 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. 3. Karnofsky performance status of 60 or more 4. Life expectancy \>12 weeks. 5. Limited systemic therapeutic options as per treating physician judgement. The number of previous lines of therapies is not limited. 6. Surgery clinically indicated. A histological confirmation of the diagnosis of a recurrent brain tumor will be required before any treatment can be initiated. 7. Adequate bone marrow, renal and hepatic function 8. 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. 9. Written informed consent for study participation must be signed and dated by the patient and the investigator prior to any study-related intervention. Exclusion Criteria: 1. Inability to undergo brain or spine MRI. 2. Concurrent treatment with other systemic tumor-directed pharmacotherapies. 3. Intent to be treated with radiotherapy. 4. Any investigational antitumor therapy other than those under investigation in this study. 5. 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. 6. 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. 7. 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. 8. Sexually active men and women of childbearing potential who are not willing to use an effective contraceptive method during the study.
References
Publications (1)
- BACKGROUNDLee 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