Open-label Study Investigating of OKN-007 Combined With Temozolomide in Patients With Recurrent Glioblastoma

NCT04388475 | Completed Phase 2 FDA Drug

• 1 other identifier: OKN-007-IV-RMG-201
• Study Type: interventional
• Target: 57
• Locations: 1 country
• Sites: 13

Brief Summary

This is a phase II open-label study investigating the efficacy, safety and pharmacokinetic(PK) properties of OKN-007 combined with temozolomide(TMZ) in patients with recurrent glioblastoma(GBM). All patients will have been previously treated with the standard-of-care treatment which includes surgical resection, radiation and chemotherapy, and in some cases treatment for recurrent disease. Patients with unequivocal recurrence (first or greater) established by MRI and meeting inclusion and exclusion criteria, will be eligible for OKN-007 treatment on this protocol.

Conditions

Recurrent Malignant Glioma; Brain Glioblastoma

Outcome Measures

Primary Outcomes (2)

• Incidents of Adverse Events during the subjects are taking OKN-007 with Temozolomide

  Evaluate incidents of Adverse Events during the subjects are taking OKN-007 with Temozolomide. Adverse events will be graded according to Common Terminology Criteria for Adverse Events (CTCAE, version 5.0).

  Through study completion up to 24 months

• Overall Survival (OS) rate

  Proportion of subjects who are alive after six months of starting treatment. OS is defined as the time from first treatment dose until date of death due to any cause.

  6 months

Secondary Outcomes (8)

• Overall Response Rate (ORR%)

  24 months

• Progression Free Survival (PFS) rate

  6 months

• Cmax of OKN-007 in blood plasma

  Day 1 and Day 5 in Cycle 1 and 2 (28 Day Cycle)

• AUC of OKN-007 in blood plasma

  Day 1 and Day 5 in Cycle 1 and 2 (28 Day Cycle)

• Tmax of OKN-007 in blood plasma

  Day 1 and Day 5 in Cycle 1 and 2 (28 Day Cycle)

Study Arms (1)

All patients

EXPERIMENTAL

All patients enrolled in this study

Drug: OKN-007; Drug: Temozolomide (TMZ)

Interventions

OKN-007 DRUG

Drug: OKN-007 (400 mg OKN-007/mL in a phosphate buffer) Administered via IV infusion, at a dose level of 60 mg/kg, given three times a week for 12 weeks, two times a week for a further 12 weeks and once per week until disease progression or up to two years.

Also known as: NXY-059, HPN-07

All patients

Temozolomide (TMZ) DRUG

Administered via oral, at a dose level of 150 mg/m2, once daily on Days 1-5 of each 28 day cycle in Cycle 1. If this dose level is tolerated, then in Cycle 2 (and subsequent cycles), at a dose level of 200 mg/m2, once daily on Days 1-5 of each 28 day cycle.

Also known as: Temodar

All patients

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Confirmed Glioblastoma based on histopathology or molecular profile analysis (WHO Grade IV), following primary treatment with TMZ and radiotherapy (minimum of 50 Gy) and at least two cycles of maintenance TMZ (5 days of a 28 day cycle) as first-line or second-line treatment with another treatment regimen, excluding bevacizumab.
• Patients must have medical records available documenting O6-methylguanine-DNA methyltransferase (MGMT) promoter methylation status analysis or must have tumor tissue samples available from prior GBM surgery or open biopsy for MGMT status determination.
• For patients with unresected recurrent tumor, unequivocal radiographic evidence of tumor progression by MRI. These patients must have at least one measurable lesion.
• Patients with recent resection of recurrent viable tumor are eligible following post-operative MRI perfusion scan with or without measurable lesions.
• No more than two prior lines of therapy for glioblastoma. Any second-line therapy is acceptable, excluding bevacizumab as second line.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
• Full recovery (≤ grade 1) from the toxic effects.
• Adequate renal, liver and bone marrow function:
• Hemoglobin \>9.0 g/dL
• Leukocytes \>3,000/mcL
• Absolute neutrophil count \>1,500/mcL
• Platelets \>100,000/mcL
• Total bilirubin ≤ 1.5 × upper limit of normal (ULN)
• AST (SGOT) / ALT (SGPT) ≤2.5 × ULN
• Creatinine clearance ≥ 60 mL/min

You may not qualify if:

• Early discontinuation of TMZ in prior line due to treatment related Adverse events (AEs).
• Second primary malignancy expected to require treatment within a 6 month period (except adequately treated basal cell carcinoma of the skin).
• Have received treatment within the last 28 days with a drug that has not received regulatory approval for any indication at the time of study entry.
• Have received chemotherapeutic agents (including temozolomide) within 28 days or within 5 half-lives for non-cytotoxic agents (whichever is shorter) of study entry
• Serious concomitant systemic disorders
• Patients with abnormal sodium, potassium, or creatinine levels ≥ grade 2.
• Patients with prothrombin time/partial thromboplastin time (PT/PTT) or International normalized ratio (INR) above the ULN.
• Inability to comply with protocol or study procedures.
• Patients who have received bevacizumab for recurrent glioblastoma or are planning to initiate treatment with bevacizumab for tumor necrosis. (Past treatment with bevacizumab for tumor necrosis is acceptable).
• Patients receiving or planning to initiate treatment with the tumor treating fields device (Optune®) (Optune® prior to enrollment is permitted).

Sponsors & Collaborators

• Oblato, Inc.: lead

Study Sites (13)

University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

Location

St. Joseph's Hospital and Medical Center

Phoenix, Arizona, 85013, United States

Location

Providence Saint John's Health Center - John Wayne Cancer Institute

Santa Monica, California, 90404, United States

Location

Swedish Medical Center

Englewood, Colorado, 80113, United States

Location

AdventHealth Orlando

Orlando, Florida, 32804, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

Norton Healthcare

Louisville, Kentucky, 40241, United States

Location

Henry Ford Health System

Detroit, Michigan, 48202, United States

Location

Wake Forest Baptist Comprehensive Cancer Center

Winston-Salem, North Carolina, 27157, United States

Location

The University of Toledo

Toledo, Ohio, 43606, United States

Location

The University of Oklahoma

Oklahoma City, Oklahoma, 73117, United States

Location

Lifespan Office of Research

Providence, Rhode Island, 02903, United States

Location

St. Joseph Hospital of Orange

Seattle, Washington, 35143, United States

Location

MeSH Terms

Conditions

Glioma

Interventions

OKN 007; disufenton sodium; Temozolomide

Condition Hierarchy (Ancestors)

Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Dacarbazine; Triazenes; Organic Chemicals; Imidazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 6, 2020
• First Posted: May 14, 2020
• Study Start: June 12, 2020
• Primary Completion: May 8, 2024
• Study Completion: March 5, 2025
• Last Updated: August 12, 2025

Record last verified: 2025-08

Locations

US (13)