Oral OKN-007 for Brain Tumor

Recruiting at 3 trial locations

Overseen By Shinwook Kang

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Oblato, Inc.

Must not be taking: Bevacizumab, Chemotherapeutics

Disqualifiers: Prior malignancy, Cardiac disease, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 24 months

16 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a phase 1 open-label, multicenter study to investigate tolerability, safety and PK properties of oral OKN-007 in patients with recurrent high-grade glioma.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, you must not have received certain treatments like chemotherapeutic agents within 28 days before starting the study.

What data supports the effectiveness of the drug OKN-007 for brain tumors?

Research shows that OKN-007 has been effective in reducing tumor size and increasing survival in animal models of glioblastoma, a type of brain tumor. It also helps normalize blood vessels in tumors, which can improve treatment outcomes. ^{1 2 3 4 5}

Is OKN-007 safe for humans?

The research articles provided do not contain specific safety data for OKN-007 in humans, as they focus on its effects in animal models. ^{1 3 6 7 8}

How is the drug OKN-007 unique for treating brain tumors?

OKN-007 is unique because it targets osteopontin (OPN), a protein that is overexpressed in brain tumors like glioblastoma, and its reduction has shown anti-tumor effects. This approach is different from standard treatments as it focuses on reducing tumor growth by interfering with specific proteins involved in tumor progression. ^{9 10 11 12 13}

Eligibility Criteria

Adults with recurrent high-grade gliomas (WHO Grade 3 or 4) who have had no more than two prior treatments, including radiotherapy and chemotherapy. Participants must be in good physical condition (ECOG <2), not pregnant, willing to use contraception, and able to swallow oral medications. They should have adequate organ function and a life expectancy of at least three months.

Inclusion Criteria

I am HIV positive, on treatment, and my viral load has been undetectable for the last 6 months.

Have provided written informed consent

My brain tumor has grown or come back, confirmed by an MRI.

Exclusion Criteria

Serious concomitant systemic disorders, for example, abnormal electrocardiogram (ECG) indicative of cardiac disease (patients with Fridericia-corrected QT interval [QTcF] >480 msec

I am not pregnant or breastfeeding.

Patients with abnormal sodium, potassium, or creatinine levels grade ≥2

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral OKN-007 daily in 28-day cycles with dose escalation across cohorts

Up to 24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• OKN-007

Trial Overview The trial is testing different doses of an oral drug called OKN-007 taken either twice or three times daily for safety and how the body processes it. It's open-label, meaning everyone knows what treatment they're getting, and involves multiple centers where patients are being studied.

Participant Groups

Group I: Mid-dose OKN-007, three times a day (TID) Experimental Treatment 1 Intervention

Dose Escalation Cohort 3

Group II: Low-dose OKN-007, two times a day (BID) Experimental Treatment 1 Intervention

Dose Escalation Cohort 1

Group III: Low-dose OKN-007, three times a day (TID) Experimental Treatment 1 Intervention

Dose Escalation Cohort 2

Group IV: High-dose OKN-007, three times a day (TID) Experimental Treatment 1 Intervention

Dose Escalation Cohort 4

Who Is Running the Clinical Trial?

Oblato, Inc.

Lead Sponsor

Trials

5

Recruited

100+

Findings from Research

A study analyzing 202 adverse events (AEs) reported by patients on health forums found that musculoskeletal disorders were reported more frequently than in the French pharmacovigilance database (FPVD), suggesting patients may experience and discuss these issues more openly online.

In contrast, skin disorders were reported less frequently on patient websites compared to the FPVD, and overall, the AEs shared by patients were less serious than those recorded in the FPVD, indicating a potential difference in the severity of AEs perceived by patients versus those documented in formal reporting systems.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Undesirable effects related to oral antineoplastic drugs: comparison between patients' internet narratives and a national pharmacovigilance database. Pages, A., Bondon-Guitton, E., Montastruc, JL., et al.[2021]

References

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

OKN-007 decreases tumor necrosis and tumor cell proliferation and increases apoptosis in a preclinical F98 rat glioma model. [2018]

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

A tale of two multi-focal therapies for glioblastoma: An antibody targeting ELTD1 and nitrone-based OKN-007. [2022]

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

Association of decreased levels of lipopolysaccharide-binding protein with OKN-007-induced regression of tumor growth in an F98 rat glioma model. [2020]

4. Japan pubmed.ncbi.nlm.nih.gov

[Phase II study of recombinant leukocyte A interferon (Ro 22-8181) in malignant brain tumors]. [2007]

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

KRN8602 (MX2-hydrochloride): an active new agent for the treatment of recurrent high-grade glioma. [2022]

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

OKN-007 decreases free radical levels in a preclinical F98 rat glioma model. [2018]

7. England pubmed.ncbi.nlm.nih.gov

Regression of glioma tumor growth in F98 and U87 rat glioma models by the Nitrone OKN-007. [2021]

8. New Zealand pubmed.ncbi.nlm.nih.gov

Undesirable effects related to oral antineoplastic drugs: comparison between patients' internet narratives and a national pharmacovigilance database. [2021]

9. England pubmed.ncbi.nlm.nih.gov

Osteopontin as a candidate of therapeutic application for the acute brain injury. [2021]

10. China pubmed.ncbi.nlm.nih.gov

[Expressions and significances of CD147, OPN and MMP-2 in oral squamous cell carcinoma]. [2017]

11. Switzerland pubmed.ncbi.nlm.nih.gov

Intranasal Delivery of RGD-Containing Osteopontin Heptamer Peptide Confers Neuroprotection in the Ischemic Brain and Augments Microglia M2 Polarization. [2021]

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

Expression of osteopontin in human glioma. Its correlation with the malignancy. [2022]

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

Selective osteopontin knockdown exerts anti-tumoral activity in a human glioblastoma model. [2018]