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

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

Unknown
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Expanded Access
Eligible Ages 13 Months - 18 Years
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

1. Age from > 12 months to ≤ 18 years of age at the time of enrollment. 2. 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. 3. Patient has completed definitive radiation therapy as part of standard-of-care treatment or was unable to undergo radiation. 4. 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. 5. Patients must have adequate bone marrow, liver, and renal function: 6. For females of childbearing potential, negative urinary or serum pregnancy test. 7. 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. 8. 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. 9. 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.

Exclusion Criteria:

1. 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. 2. 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. 3. Patients completing radiotherapy treatment less than 14 days prior to planned study treatment initiation. 4. 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. 5. 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. 6. Known hypersensitivity to OKN-007 or any component in its formulation. 7. Patients taking prohibited medications as described in the current Investigator's Brochure. 8. Patient is pregnant or actively breastfeeding.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT05518838
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Oblato, Inc.
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

N/A
Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Available
Countries United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Diffuse Midline Glioma, H3 K27M-Mutant, Diffuse Intrinsic Pontine Glioma

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Arkansas Children's Research Institute, Little Rock, Arkansas

Status

Available

Address

Arkansas Children's Research Institute

Little Rock, Arkansas, 72202

Site Contact

Catherine Redinger

RedingerCatherineL@uams.edu

501-364-4290

Nicklaus Children's Hospital, Miami, Florida

Status

Available

Address

Nicklaus Children's Hospital

Miami, Florida, 33155

Site Contact

Damaris Rodriguez

Damaris.Rodriguez@Nicklaushealth.org

786-624-4908

Arnold Palmer Hospital for Children, Orlando, Florida

Status

Available

Address

Arnold Palmer Hospital for Children

Orlando, Florida, 32806

Site Contact

Paige Dubberly

paige.dubberly@orlandohealth.com

321-841-7561

Johns Hopkins All Children's Hospital, Saint Petersburg, Florida

Status

Available

Address

Johns Hopkins All Children's Hospital

Saint Petersburg, Florida, 33701

Site Contact

Stacie Stapleton

Stacie.Stapleton@jhmi.edu

727-767-4176

Children's Hospital and Medical Center, Omaha, Nebraska

Status

Available

Address

Children's Hospital and Medical Center

Omaha, Nebraska, 68114

Site Contact

Josie Kugler

jokugler@unmc.edu

402-639-0209

Columbia, South Carolina

Status

Available

Address

Prisma Health Midlands Pediatric Hematology/Oncology

Columbia, South Carolina, 29203

Site Contact

Melissa Henson

Melissa.Henson@Prismahealth.org

803-434-3685

Norfolk, Virginia

Status

Available

Address

Children's Hospital of The King's Daughters

Norfolk, Virginia, 23507

Site Contact

Melissa Mark, MD

melissa.mark@chkd.org

+1-609-734-4329

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