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This is an intermediate-size expanded access protocol to provide ONC201 to patients with diffuse intrinsic pontine gliomas who cannot access ONC201 through clinical trials.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ONC201 | Drug | ONC201 is an oral, small molecule selective antagonist of DRD2 |
Inclusion Criteria:
Patient with DIPG who failed at least one line of prior therapy such as focal radiation therapy
Patient must be > than 6 months and < 18 years of age.
Confirmed evidence of disease progression from immediately prior therapy or refractory to the immediately prior treatment.
Karnofsky/Lansky performance status ≥ 50.
Adequate organ and marrow function as defined below:
Absolute neutrophil count ≥1,000/mm3 without growth factor use ≤ 7 days prior to treatment (cycle 1 day 1, C1D1)
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Hemoglobin>8.0 mg/dL without red blood cell transfusion ≤ 3 days prior to C1D1
Total serum bilirubin<1.5 X upper limit of normal (ULN)
AST (SGOT)/ALT (SGPT)≤2 X ULN;; ≤ 5 X ULN if there is liver involvement secondary to tumor
Serum creatinine≤ 1.5 X ULN (OR creatinine clearance ≥ 60 mL/min/1.73 m2)
Ability to understand and the willingness to sign a written informed consent document by the patient or their legal guardian.
If patient is of child--bearing age, female patients must be surgically sterile or be postmenopausal, or must agree to use effective contraception during the period of the trial and for at least 90 days after completion of treatment. Male patients must be surgically sterile or must agree to use effective contraception during the period of the trial and for at least 90 days after completion of treatment. The decision of effective contraception will be based on the judgment of the principal investigator or a designated associate.
Patient must be able to swallow capsules and retain orally administered medication.
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California San Francisco | San Francisco | California | 94143 | United States | ||
| Children's Hospital Colorado |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38335473 | Derived | Arrillaga-Romany I, Gardner SL, Odia Y, Aguilera D, Allen JE, Batchelor T, Butowski N, Chen C, Cloughesy T, Cluster A, de Groot J, Dixit KS, Graber JJ, Haggiagi AM, Harrison RA, Kheradpour A, Kilburn LB, Kurz SC, Lu G, MacDonald TJ, Mehta M, Melemed AS, Nghiemphu PL, Ramage SC, Shonka N, Sumrall A, Tarapore RS, Taylor L, Umemura Y, Wen PY. ONC201 (Dordaviprone) in Recurrent H3 K27M-Mutant Diffuse Midline Glioma. J Clin Oncol. 2024 May 1;42(13):1542-1552. doi: 10.1200/JCO.23.01134. Epub 2024 Feb 9. |
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| ID | Term |
|---|---|
| D005910 | Glioma |
| ID | Term |
|---|---|
| D018302 | Neoplasms, Neuroepithelial |
| D017599 | Neuroectodermal Tumors |
| D009373 | Neoplasms, Germ Cell and Embryonal |
| D009370 | Neoplasms by Histologic Type |
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| ID | Term |
|---|---|
| C585684 | TIC10 compound |
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| Aurora |
| Colorado |
| 80045 |
| United States |
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | United States |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30342 | United States |
| University of Michigan | Ann Arbor | Michigan | 48109 | United States |
| University of Nebraska Medical Center | Omaha | Nebraska | 68198 | United States |
| Albany Medical Center | Albany | New York | 12208 | United States |
| NYU Langone Health | New York | New York | 10016 | United States |
| Stephen Hassenfeld Children's Center for Cancer and Blood Disorders (NYU) | New York | New York | 10016 | United States |
| University of Rochester Medical Center | Rochester | New York | 14642 | United States |
| D009369 | Neoplasms |
| D009375 | Neoplasms, Glandular and Epithelial |
| D009380 | Neoplasms, Nerve Tissue |