Clinical trial NCT06804655: Pharmacoscopy for Patients with Refractory Primary Brain Tumors
Official title: Pharmacoscopy for Patients with Refractory Primary Brain Tumors
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
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
Lead sponsor: University of Zurich
Sponsor class: OTHER
Collaborators: name: University Hospital, Zürich
name: University Hospital, Basel, Switzerland
name: Cantonal Hospital St. Gallen, Switzerland
First posted: 2025-02-03
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

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.


Eligibility criteria: 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.

Primary outcomes:
measure: To demonstrate the overall feasibility of pharmacoscopy-guided treatment for patients with refractory primary brain tumors
timeFrame: 2 months
Secondary outcomes:
measure: To explore the efficacy of pharmacoscopy-guided treatment - 1
timeFrame: 12 months
measure: To explore the efficacy of pharmacoscopy guided treatment - 2
timeFrame: 12 months
measure: To explore the efficacy of pharmascopy guided treatment - 3
timeFrame: 12 months
measure: To explore the efficacy of pharmascopy guided treatment - 4
timeFrame: 24 months
measure: To confirm the safety and tolerability of treatment selected based on pharmacoscopy - 1
timeFrame: 12 months
measure: To confirm the safety and tolerability of treatment selected based on pharmacoscopy -2
timeFrame: 12 months
measure: To confirm the safety and tolerability of treatment selected based on pharmacoscopy - 3
timeFrame: 12 months
measure: Quality of life -1
timeFrame: 12 months
measure: Quality of life - 2
timeFrame: 12 months
Other outcomes:
measure: Translational research
timeFrame: 12 months
Locations:
city: Basel
country: Switzerland
facility: University Hospital Basel
state: Basel Land
city: St Gallen
country: Switzerland
facility: Cantonal Hospital St Gallen
city: Zurich
country: Switzerland
facility: University Hospital Zurich
Study officials:
References:
citation: 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.