This is an intermediate-size expanded access protocol to provide ONC201 to patients with H3 K27M-mutant and/or midline gliomas who cannot access ONC201 through clinical trials.
Drug: ONC201
ONC201 is an oral, small molecule selective antagonist of DRD2
Inclusion Criteria: 1. Patient meets one or more of the criteria below: Arm A 1. Central nervous system tumor that is positive for the H3 K27M mutation (performed in a laboratory with CLIA or equivalent certification); OR 2. Central nervous system tumor involving the thalamus, hypothalamus, basal ganglia, brainstem, cerebellum, cerebellar peduncle, midline cortex, corpus callosum, pineal region, optic tract, or optic chiasm. Arm B 3. Diffuse intrinsic pontine glioma (DIPG), defined as tumors with a pontine epicenter and diffuse involvement of the pons. H3 K27M status does not have to be known or positive for this arm. 2. Arm A: Patient must have unequivocal radiographic evidence of progressive disease on as defined by RANO criteria or have documented recurrent glioma on diagnostic biopsy. Arm B: Patient isnot required to have radiographic or clinical evidence of progressive disease. 3. Arm A: Patient must be at least 90 days from completion of radiotherapy. Arm B: Patient must be at least 14 days from completion of radiotherapy. 4. Patient must be at least 2 years of age. 5. Patient must weigh at least 10kg. 6. From the projected start of scheduled study treatment, the following time periods must have elapsed from prior anti-cancer treatments: 5 half-lives from any investigational agent, 4 weeks from cytotoxic therapy (except 23 days for temozolomide and 6 weeks from nitrosoureas), 6 weeks from anti-cancer antibodies (except 21 days for bevacizumab), 4 weeks (or 5 half-lives, whichever is shorter) from other anti-tumor therapies, and 1 week from devices used to treat cancer. 7. Brain MRI within 28 days prior to start of study drug. 8. Adequate organ and marrow function as defined below: 1. Absolute neutrophil count ≥1,000/mm3 without growth factor use ≤ 7 days prior to treatment (cycle 1 day 1, C1D1) 2. Hemoglobin >8.0 mg/dL without red blood cell transfusion ≤ 3 days prior to C1D1 3. Total serum bilirubin 480 milliseconds) using Fridericia's QT correction formula on two ECGs separated by at least 2 days. 7. A history of Torsades de Pointes or heart failure, hypokalemia, or family history of prolonged QT Syndrome 8. Concomitant use of medication(s) known to prolong the QT/QTc interval.
BMDACC at Banner University Medical Center Phoenix
Phoenix, Arizona, 85006
Investigator: Devon Coleman
Investigator: Na Tosha Gatson, MD
Children's Hospital of Orange County
Orange, California, 92868
Investigator: Mariko Sato
Investigator: Mariko Sato
Rady Children's Hospital
San Diego, California, 92123
Investigator: Megan Paul, MD
Providence Saint John's Health Center
Santa Monica, California, 90404
Investigator: Akanksha Sharma, MD
Children's Hospital of Colorado
Aurora, Colorado, 80045
Investigator: Ashley Mettetal
Contact: 720-777-5305
Investigator: Kathleen Dorris, MD
Children's National Medical Center
Washington, District of Columbia, 20010
Investigator: Lindsay Kilburn, MD, MS
Contact: 202-476-3643
Miami Cancer Institute
Miami, Florida, 33176
Investigator: Yazmin Odia, MD, MS
Contact: 786-596-2000
Investigator: Yazmin Odia, MD, MS
Children's Healthcare of Atlanta, Emory University School of Medicine
Atlanta, Georgia, 30322
Investigator: Dolly Aguilera, MD
Contact: 404-785-1112
Kapi'olani Medical Center for Women and Children
Honolulu, Hawaii, 96826
Investigator: Andrea Siu, MPH
Contact: 808-535-7169
Investigator: Kelley Hutchins, DO
Lurie Children's Hospital
Chicago, Illinois, 60661
Investigator: Angela Waanders, MD, MPH
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
Investigator: David Dickens, MD
University of Michigan
Ann Arbor, Michigan, 48109
Investigator: Misty Gravelin
Contact: 734-763-0592
Investigator: Carl Koschmann, MD, MS
University of Minnesota
Minneapolis, Minnesota, 55455
Investigator: Clark Chen, MD, PhD
Washington University in St. Louis
Saint Louis, Missouri, 63110
Investigator: Andrew Cluster, MD
Investigator: Alison Barnwell
Investigator: Andrew Cluster, MD
University of Nebraska Medical Center - Adults Only
Omaha, Nebraska, 68198
Investigator: Nicole Shonka
Overlook Medical Center/ Atlantic Health System
Summit, New Jersey, 07901
Investigator: Robert Aiken, MD
Albany Medical Center
Albany, New York, 12208
Investigator: Lauren Weintraub, MD
New York University Langone - Active, Not Enrolling
New York, New York, 10016
Investigator: Anna Yaffe
Investigator: Sharon Gardner, MD
University of Rochester
Rochester, New York, 14627
Investigator: David Korones, MD
Contact: 585-275-2981
Investigator: Jennifer Serventi, PA
Contact: 585-275-5863
University of Texas Southwestern
Dallas, Texas, 75390
Investigator: Alison Patterson
Contact: 214-456-2726
Investigator: Daniel Bowers, MD
MD Anderson Cancer Center
Houston, Texas, 77030
Investigator: Teresa Hannah
Investigator: Nazanin Majd
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507
Investigator: Melissa Mark, MD
Tarapore, PhD
1-919-806-1074
clinicaltrials@chimerix.com