Expanded Access to OKN-007 for Patients With Diffuse Midline Glioma, H3 K27-altered
- Conditions
- Diffuse Midline Glioma, H3 K27M-MutantDiffuse Intrinsic Pontine Glioma
- Registration Number
- NCT05518838
- Lead Sponsor
- Oblato, Inc.
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NO_LONGER_AVAILABLE
- Sex
- All
- Target Recruitment
- Not specified
- 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.
- 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.
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (9)
Nicklaus Children's Hospital
🇺🇸Miami, Florida, United States
Arkansas Children's Research Institute
🇺🇸Little Rock, Arkansas, United States
Arnold Palmer Hospital for Children
🇺🇸Orlando, Florida, United States
Children's Hospital and Medical Center
🇺🇸Omaha, Nebraska, United States
Johns Hopkins All Children's Hospital
🇺🇸Saint Petersburg, Florida, United States
Prisma Health Midlands Pediatric Hematology/Oncology
🇺🇸Columbia, South Carolina, United States
Children's Health
🇺🇸Dallas, Texas, United States
Children's Hospital of The King's Daughters
🇺🇸Norfolk, Virginia, United States
Carilion Roanoke Memorial Hospital
🇺🇸Roanoke, Virginia, United States