NO_LONGER_AVAILABLENot Applicable

Expanded Access to OKN-007 for Patients With Diffuse Midline Glioma, H3 K27-altered

United States

Plain-language 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.

Who can participate

Age range13 Months – 18 Years

SexALL

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Inclusion criteria

✓. 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

Exclusion criteria

✕. 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.

Trial details

NCT IDNCT05518838

SponsorOblato, Inc.

Sponsor typeINDUSTRY

Study typeEXPANDED_ACCESS

View on ClinicalTrials.gov More Diffuse Midline Glioma, H3 K27M-Mutant trials