Perillyl Alcohol for Glioblastoma

Recruiting at 8 trial locations

Overseen By Chloe Richmond

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Neonc Technologies, Inc.

Must not be taking: Bevacizumab, Gliadel, others

Disqualifiers: Large tumor, Multifocal tumor, others

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

Compensation: Varies

Number of Visits: Unknown

49 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing NEO100, a purified form of perillyl alcohol, to treat aggressive brain tumors in patients whose cancer has returned or not responded to other treatments. The treatment is given through the nose regularly and may help slow down tumor growth. Perillyl alcohol (POH) is a naturally occurring substance with properties that may inhibit tumor growth, and NEO100 is a highly purified version of POH currently being tested for glioblastoma treatment.

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications. However, you must be on a stable dose of steroids for at least five days before joining, and if you have seizures, your anti-epileptic medication must be stable for 14 days prior to consent.

What data supports the effectiveness of the drug Perillyl alcohol for treating glioblastoma?

Research shows that Perillyl alcohol (POH) can kill glioblastoma cells and make them more sensitive to radiation and chemotherapy. It has been effective in both lab studies and early clinical trials, especially when given through the nose, and has shown promise in treating tumors that resist other treatments. ^{1 2 3 4 5}

Is perillyl alcohol safe for humans?

Perillyl alcohol (POH) has been tested in clinical trials and was generally well tolerated when given intranasally, with fewer side effects compared to oral administration, which caused significant gastrointestinal issues. The intranasal delivery of POH has shown promise in treating gliomas with manageable safety profiles. ^{1 2 3 4 5}

How is the drug Perillyl Alcohol unique for treating glioblastoma?

Perillyl Alcohol is unique for treating glioblastoma because it is administered intranasally, which helps bypass the blood-brain barrier, a common challenge in treating brain tumors. It also acts as a radiosensitizer, enhancing the effects of radiation therapy, and is effective against temozolomide-resistant gliomas, making it a promising option for difficult-to-treat cases. ^{1 2 3 5 6}

Research Team

Thomas Chen, MD, PhD

Principal Investigator

NeOnc Technologies

Vincent F Simmon, PhD

Principal Investigator

NeOnc Technologies

Patrick Walters

Principal Investigator

NeOnc Technologies

Eligibility Criteria

This trial is for patients with Grade IV glioma that has progressed after radiation or temozolomide treatment. They must have a life expectancy of at least three months, be in fair to good physical condition (ECOG 0-2 or KPS ≥60), and not have had recent surgery or chemotherapy. Participants need stable steroid use before consent, adequate organ function, and agree to contraception.

Inclusion Criteria

Must have the ability to understand, and the willingness to sign, a written informed consent.

Must have an expected survival of at least three months.

I agree to use birth control during the study.

Exclusion Criteria

I finished chemo-radiation within the last 3 months, unless my cancer has grown or spread.

I had surgery within the last week.

I have received chemotherapy in the last 28 days.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 1 Treatment

Intranasal delivery of NEO100 (perillyl alcohol) four times a day, escalation up to four different doses to determine maximum tolerated dose

28-day treatment cycles

Phase 2A Treatment

Intranasal delivery of NEO100 (perillyl alcohol) four times a day at maximum tolerated dose

28-day treatment cycles

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Perillyl alcohol

Trial Overview NEO100 (perillyl alcohol) is being tested through nasal administration for its safety and effectiveness against recurrent Grade IV glioma. The study includes two phases: Phase 1 determines the maximum tolerated dose via a '3+3' design; Phase 2a treats patients with this dose. Up to six cycles of NEO100 are given daily over a period of four weeks each.

Participant Groups

Group I: NEO100 Phase 2A Experimental Treatment 1 Intervention

Intranasal delivery of NEO100 (perillyl alcohol) four times a day. Treatment of total of 25 patients at maximum tolerated dose.

Group II: NEO100 Phase 1 Experimental Treatment 1 Intervention

Intranasal delivery of NEO100 (perillyl alcohol) four times a day, escalation up to four different doses to determine maximum tolerated dose.

Who Is Running the Clinical Trial?

Neonc Technologies, Inc.

Lead Sponsor

Trials

4

Recruited

230+

Findings from Research

Intranasal delivery of NEO100, a purified form of perillyl alcohol, was well tolerated in a Phase I trial with 12 patients suffering from recurrent glioblastoma, with no severe adverse events reported.

The treatment showed promising survival outcomes, with a 33% progression-free survival at 6 months and a 55% overall survival at 12 months, suggesting that this novel approach may be beneficial for patients with recurrent GBM.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phase I trial of intranasal NEO100, highly purified perillyl alcohol, in adult patients with recurrent glioblastoma. Schönthal, AH., Peereboom, DM., Wagle, N., et al.[2022]

Perillyl alcohol (POH) is effective against both temozolomide-sensitive and temozolomide-resistant glioma cells, showing cytotoxic effects through mechanisms involving endoplasmic reticulum stress and inhibition of survival pathways.

Intranasal administration of POH was well tolerated and led to decreased tumor growth and increased survival in animal models with intracranial tumors, suggesting a promising route for treatment of resistant gliomas.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Perillyl alcohol for the treatment of temozolomide-resistant gliomas. Cho, HY., Wang, W., Jhaveri, N., et al.[2021]

In a study of 67 patients with recurrent malignant gliomas treated with intranasal perillyl alcohol, those with tumors located in the basal ganglia had significantly longer survival compared to those with lobar gliomas.

The presence of peritumoral brain edema (PTBE) was associated with poor therapeutic response and shorter survival, indicating that tumor size and edema may influence treatment outcomes and the behavior of glioma cells.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Correlation of tumor topography and peritumoral edema of recurrent malignant gliomas with therapeutic response to intranasal administration of perillyl alcohol. Da Fonseca, CO., Silva, JT., Lins, IR., et al.[2021]

References

1. Greece pubmed.ncbi.nlm.nih.gov

Perillyl alcohol induces apoptosis in human glioblastoma multiforme cells. [2016]

2. England pubmed.ncbi.nlm.nih.gov

Phase I trial of intranasal NEO100, highly purified perillyl alcohol, in adult patients with recurrent glioblastoma. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Perillyl alcohol as a radio-/chemosensitizer in malignant glioma. [2021]

4. Greece pubmed.ncbi.nlm.nih.gov

Long-term outcome in patients with recurrent malignant glioma treated with Perillyl alcohol inhalation. [2016]

5. United States pubmed.ncbi.nlm.nih.gov

Perillyl alcohol for the treatment of temozolomide-resistant gliomas. [2021]

6. United States pubmed.ncbi.nlm.nih.gov

Correlation of tumor topography and peritumoral edema of recurrent malignant gliomas with therapeutic response to intranasal administration of perillyl alcohol. [2021]