Individualized Therapy for Glioblastoma

Overseen By Stephanie Lewis, RN

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Jennifer Clarke

Must not be taking: Chemotherapy, Radiotherapy

Disqualifiers: Multifocal tumor, Active malignancy, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: Up to 9 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new approach to treat glioblastoma, a type of brain cancer, by creating personalized treatment plans based on each patient's unique cancer genetics. The study aims to identify the best combination of existing FDA-approved drugs for individuals with recurrent glioblastoma that can be surgically removed. Eligible participants must have a glioblastoma that their surgeon believes can be significantly removed. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative approach.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, you cannot have had chemotherapy or radiotherapy within 4 weeks before joining the study, with specific time requirements for certain drugs like temozolomide and nitrosurea.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that the safety of personalized treatments for glioblastoma is still under exploration. In this trial, up to four FDA-approved drugs are tailored to each patient. Since these drugs have approval, their usual doses and safety in other conditions are known.

However, this trial tests new combinations of these drugs specifically for glioblastoma, and limited information exists on how well patients tolerate these combinations due to the novelty of the approach.

The trial is in its early stages, focusing mainly on safety, so researchers will closely monitor for any new or unexpected side effects. While personalized treatments hold promise, more research is needed to fully understand their safety in this context. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatment?

Researchers are excited about this individualized therapy for glioblastoma because it tailors treatment to each patient's unique tumor profile. Unlike the standard treatments such as surgery, radiation, and chemotherapy with drugs like temozolomide, this approach selects from a range of FDA-approved drugs based on critical molecular signaling pathways specific to the patient. This precision targeting could potentially increase the effectiveness of the treatment and reduce side effects compared to the one-size-fits-all approach of current options. By harnessing a personalized regimen, this therapy offers hope for better outcomes in a notoriously difficult-to-treat cancer.

What evidence suggests that this trial's individualized therapy could be effective for glioblastoma?

Research has shown that personalized treatments for glioblastoma might extend patient survival. This trial will explore individualized therapy, which tailors treatments to the specific genetic details of each patient's tumor. However, consistent proof that these personalized therapies significantly improve survival rates is lacking. Glioblastoma is a challenging cancer to treat, and standard treatments often result in recurrence. In this trial, personalized therapy aims to combat the cancer more effectively by using drugs selected specifically for each patient. While promising, the success of this approach in improving outcomes remains under study. ^{2 5 6 7 8}

Who Is on the Research Team?

Jennifer Clarke, MD, MPH

Principal Investigator

University of California, San Francisco

Are You a Good Fit for This Trial?

Adults over 18 with recurrent, surgically-removable glioblastoma or gliosarcoma can join this trial. They must have had prior standard GBM treatment, be in good physical condition (KPS score >=70), and expected to live at least 3 more months. Participants need proper organ function and agree to use contraception. Pregnant women, those with severe heart issues, other active cancers (except certain cases), HIV-positive individuals, or psychiatric illnesses that affect study compliance are excluded.

Inclusion Criteria

Patient must have an estimated life expectancy ≥ 3 months

I agree to use birth control during and for 3 months after the study.

Patients must understand and provide written informed consent and Health Insurance Portability and Accountability Act of 1996 (HIPAA) authorization prior to initiation of any study-specific procedures

Exclusion Criteria

Patients known to be HIV-positive

Uncontrolled concurrent illness including psychiatric illness, or situations that would limit compliance with the study requirements or the ability to willingly give written informed consent

Any other acute or chronic medical or psychiatric condition, or laboratory abnormality that could increase the risk associated with trial participation or trial drug administration or could interfere with the interpretation of trial results, and, in the judgment of the investigator, would make the patient inappropriate for entry into the trial

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Surgery and Diagnostic Testing

Surgery for tumor resection followed by diagnostic testing using UCSF 500 Cancer Gene Panel

Up to 5 weeks

1 visit (in-person) for surgery, additional visits for testing

Treatment

Initiation of individualized treatment regimen based on Tumor Board recommendations

Up to 9 months

Regular visits as per treatment protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 5 years

What Are the Treatments Tested in This Trial?

Interventions

• Individualized therapy

Trial Overview The trial is testing the feasibility of personalized therapy for adults with recurrent glioblastoma using the UCSF 500 Cancer Gene Panel from tumor tissue and blood samples. This genetic test aims to tailor targeted treatments based on individual cancer DNA changes.

How Is the Trial Designed?

Group I: Individualized therapy Experimental Treatment 1 Intervention

Study treatment for a given patient will consist of a regimen chosen from agents implicated in critical molecular signaling pathways and/or from signature-based predictions of drug efficacy. All agents are listed in the current pharmacopoeia for human use, but will differ amongst individual subjects. The study treatment will consist of up to 4 FDA approved drugs that have known dosing. This study is not only looking at 4 drugs. It is selecting up to 4 drugs per patient but the drugs chosen can be any FDA-approved drug. Therefore, it is not possible to pre-specify the medications.

Who Is Running the Clinical Trial?

Jennifer Clarke

Lead Sponsor

Trials

1

Recruited

30+

Published Research Related to This Trial

Despite aggressive treatment options for glioblastoma, including surgery and chemotherapy, the median survival remains low at 14-16 months, highlighting the need for new therapeutic strategies.

Recent data suggest that the Optune™ device, which delivers alternating electrical field therapy, may improve survival in newly diagnosed glioblastoma patients, indicating a promising new approach alongside traditional treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

What next for newly diagnosed glioblastoma? Domingo-Musibay, E., Galanis, E.[2022]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10296009/

Personalized Treatment of Glioblastoma: Current State and ... Due to inherent heterogeneity of GBM, applying patient-tailored treatment could significantly prolong survival of these patients.

2. nature.com nature.com/articles/s41467-024-51315-8

A real-world observation of patients with glioblastoma ... Current treatment outcome of patients with glioblastoma (GBM) remains poor. Following standard therapy, recurrence is universal with limited ...

3. frontiersin.org frontiersin.org/journals/medicine/articles/10.3389/fmed.2023.1166104/full

Personalised therapeutic approaches to glioblastoma There was no consistent evidence for survival efficacy of any personalised therapy. Conclusion: Personalised glioblastoma therapies remain of unproven survival ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S2950523225000387

Advances and challenges in personalized diagnosis ... This review offers a detailed analysis of progress and setbacks toward the creation and provision of individual treatments for recurrent GBM ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT03681028

Study Details | NCT03681028 | Feasibility of Individualized ... The current study will test the ability and likelihood of successfully implementing individualized combination treatment recommendations for adult patients

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10140534/

Personalised therapeutic approaches to glioblastoma Conclusion. Personalised glioblastoma therapies remain of unproven survival benefit. Evidence is inconsistent with high risk of bias. ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11111908/

Individualized survival prediction and surgery ... This was a retrospective cohort study predicting the survival outcomes of patients with GBM and identifying the patients' ITE to determine ...

8. ascopubs.org ascopubs.org/doi/abs/10.1200/JCO.2025.43.16_suppl.TPS2097

Advanced brain tumor therapy clinical trial (ATTRACT). Based on overall survival as the primary endpoint, we plan to include 240 patients (120 per group) to show with a power of 80% that we can ...