Pharmacoscopy for Patients with Refractory Primary Brain Tumors

Phase 2

Not yet recruiting

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

• Registration Number: NCT06804655
• Lead Sponsor: University of Zurich

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-12-17
• Status Verified: 2025-01
• Study First Submitted QC: 2025-01-27
• Last Update Submitted: 2025-01-27
• Study First Posted: 2025-02-03
• Last Update Posted: 2025-02-03
• Study Start: 2025-04-01
• Primary Completion: 2026-01-15
• Study Completion: 2026-08-15

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 40

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
To demonstrate the overall feasibility of pharmacoscopy-guided treatment for patients with refractory primary brain tumors	2 months	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 Outcome Measures

Name	Time	Method
To explore the efficacy of pharmacoscopy-guided treatment - 1	12 months	(1) Response rate
To explore the efficacy of pharmacoscopy guided treatment - 2	12 months	duration of response
To explore the efficacy of pharmascopy guided treatment - 3	12 months	progression-free survival
To explore the efficacy of pharmascopy guided treatment - 4	24 months	overall survival
To confirm the safety and tolerability of treatment selected based on pharmacoscopy - 1	12 months	Neurological function (NANO scale, Nayak et al. 2017)
To confirm the safety and tolerability of treatment selected based on pharmacoscopy -2	12 months	Karnofsky performance status (score 0-100)
To confirm the safety and tolerability of treatment selected based on pharmacoscopy - 3	12 months	Steroid use (dose in mg equivalent dexamethasone)
Quality of life -1	12 months	EORTC QLQ C30 auto-questionnaire
Quality of life - 2	12 months	EORTC BN20 autoquestionnaire

Trial Locations

Locations (3)

University Hospital Zurich; 🇨🇭 Zurich, Switzerland

University Hospital Basel; 🇨🇭 Basel, Basel Land, Switzerland

Cantonal Hospital St Gallen; 🇨🇭 St Gallen, Switzerland