NCT05518838

Brief Summary

To provide OKN-007 for compassionate use in patients with diffuse midline glioma, H3 K27-altered (DMG), including diffuse intrinsic pontine glioma (DIPG), pediatric and young adult patients with high-grade diffuse midline glioma will be treated under this Intermediate-sized expanded access treatment protocol.

Trial Health

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Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Geographic Reach
1 country

9 active sites

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 24, 2022

Completed
5 days until next milestone

First Posted

Study publicly available on registry

August 29, 2022

Completed
Last Updated

November 8, 2024

Status Verified

November 1, 2024

First QC Date

August 24, 2022

Last Update Submit

November 6, 2024

Conditions

Interventions

OKN-007 is an intravenously administrated drug being developed as a novel anti-cancer therapy against gliomas.

Eligibility Criteria

Age13 Months - 18 Years
Sexall
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Age from \> 12 months to ≤ 18 years of age at the time of enrollment.
  • Diagnosis of diffuse midline glioma, H3 K27-altered according to the WHO 2021 Classification of Tumors of the Central Nervous System diagnostic criteria including diffuse intrinsic pontine glioma (DIPG). In the absence of a pathologically confirmed diagnosis, a grade IV glioma involving the thalamus, hypothalamus, brainstem, cerebellum, midbrain, or spinal cord, or with a pontine epicenter and diffuse involvement of the pons.
  • Patient has completed definitive radiation therapy as part of standard-of-care treatment or was unable to undergo radiation.
  • Karnofsky performance status ≥ 50 for patients ≥ 16 years of age and Lansky performance status ≥ 50 for patients \< 16 years of age; patients who are unable to walk because of paralysis, but who are upright in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
  • Patients must have adequate bone marrow, liver, and renal function:
  • For females of childbearing potential, negative urinary or serum pregnancy test.
  • Patients must be willing to highly effective contraception for both male and female patients throughout the treatment and for at least 4 months after last treatment administration.
  • Toxicities related to any prior treatments are either stable, stable on supportive therapy, resolved, or in the opinion of the treating physician, clinically non-significant
  • Ability to understand a written informed consent document, and the willingness to sign it. Assent will be obtained when appropriate based on the patient's age.

You may not qualify if:

  • Patient is already participating in or qualifies for and is able to enroll in a clinical trial of OKN-007 and other ongoing clinical trials.
  • Patient has received systemic therapy with an investigational agent within 5 half-lives or 14 days prior to starting OKN-007 treatment, whichever is shorter.
  • Patients completing radiotherapy treatment less than 14 days prior to planned study treatment initiation.
  • Current evidence of uncontrolled, significant intercurrent illness that would, in the treating physician's judgment, contraindicate the patient's treatment with OKN-007 due to safety concerns.
  • Patients who, in the opinion of the treating physician, have not fully recovered from recent major surgery to a sufficient extent to tolerate treatment with OKN-007.
  • Known hypersensitivity to OKN-007 or any component in its formulation.
  • Patients taking prohibited medications as described in the current Investigator's Brochure.
  • Patient is pregnant or actively breastfeeding.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

Arkansas Children's Research Institute

Little Rock, Arkansas, 72202, United States

Location

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

Arnold Palmer Hospital for Children

Orlando, Florida, 32806, United States

Location

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States

Location

Children's Hospital and Medical Center

Omaha, Nebraska, 68114, United States

Location

Prisma Health Midlands Pediatric Hematology/Oncology

Columbia, South Carolina, 29203, United States

Location

Children's Health

Dallas, Texas, 75235, United States

Location

Children's Hospital of The King's Daughters

Norfolk, Virginia, 23507, United States

Location

Carilion Roanoke Memorial Hospital

Roanoke, Virginia, 24014, United States

Location

MeSH Terms

Conditions

Diffuse Intrinsic Pontine Glioma

Interventions

OKN 007

Condition Hierarchy (Ancestors)

GliomaNeoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasmsNeoplasms, Glandular and EpithelialNeoplasms, Nerve TissueBrain Stem NeoplasmsInfratentorial NeoplasmsBrain NeoplasmsCentral Nervous System NeoplasmsNervous System NeoplasmsNeoplasms by SiteBrain DiseasesCentral Nervous System DiseasesNervous System Diseases

Study Design

Study Type
expanded access
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 24, 2022

First Posted

August 29, 2022

Last Updated

November 8, 2024

Record last verified: 2024-11

Locations