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Anderson PM, Trucco MM, Tarapore RS, et al. Phase 2 Study of ONC201 in Neuroendocrine Tumors including Pheochromocytoma-Paraganglioma and Desmoplastic Small Round Cell Tumor. Clin Cancer Res. 2022;doi: 10.1158/1078-0432.CCR-21-4030.
Anderson, P. M., Trucco, M. M., Tarapore, R. S., Zahler, S., Thomas, S., Gortz, J., Mian, O., Stoignew, M., Prabhu, V., Morrow, S., & Allen, J. E. (2022). Phase 2 Study of ONC201 in Neuroendocrine Tumors including Pheochromocytoma-Paraganglioma and Desmoplastic Small Round Cell Tumor. Clinical cancer research : an official journal of the American Association for Cancer Research, . https://doi.org/10.1158/1078-0432.CCR-21-4030
Anderson, Peter M, et al. "Phase 2 Study of ONC201 in Neuroendocrine Tumors including Pheochromocytoma-Paraganglioma and Desmoplastic Small Round Cell Tumor." Clinical cancer research : an official journal of the American Association for Cancer Research vol. (2022). doi: https://doi.org/10.1158/1078-0432.CCR-21-4030
Anderson PM, Trucco MM, Tarapore RS, Zahler S, Thomas S, Gortz J, Mian O, Stoignew M, Prabhu V, Morrow S, Allen JE. Phase 2 Study of ONC201 in Neuroendocrine Tumors including Pheochromocytoma-Paraganglioma and Desmoplastic Small Round Cell Tumor. Clin Cancer Res. 2022 Jan 12; doi: 10.1158/1078-0432.CCR-21-4030. Epub 2022 Jan 12. PMID: 35022321.
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Clin Cancer Res. 2022 Jan 12; doi: 10.1158/1078-0432.CCR-21-4030. Epub 2022 Jan 12.

Phase 2 Study of ONC201 in Neuroendocrine Tumors including Pheochromocytoma-Paraganglioma and Desmoplastic Small Round Cell Tumor.

Clinical cancer research : an official journal of the American Association for Cancer Research

Peter M Anderson, Matteo M Trucco, Rohinton S Tarapore, Stacey Zahler, Stefanie Thomas, Janette Gortz, Omar Mian, Martin Stoignew, Varun Prabhu, Sara Morrow, Joshua E Allen

Affiliations

  1. Oncology, Cleveland Clinic [email protected].
  2. Pediatric Hematology/Oncology/BMT, Cleveland Clinic.
  3. Oncoceutics Inc.
  4. Oncology, Cleveland Clinic.
  5. Oncology, Cleveland Clinic Children's.
  6. Translational Hematology & Oncology Research (THOR) and Radiation Oncology, Cleveland Clinic.
  7. Oncoceutics (United States).
  8. R, Oncoceutics Inc.
  9. Clinical Pharmacology & Translational Medicine, Oncoceutics (United States).
  10. R&D, Oncoceutics Inc.

PMID: 35022321 DOI: 10.1158/1078-0432.CCR-21-4030

Abstract

PURPOSE: Tumor dopamine-like DRD2 receptor expression is higher in pheochromocytoma-paraganglioma (PC-PG) compared to other cancers. ONC201 is a bitopic DRD2 antagonist with pre-clinical ONC201 activity in DSRCT.

EXPERIMENTAL DESIGN: Patients (N=30) with neuroendocrine tumors were treated on this investigator-initiated trial (NCT03034200). ONC201 dose and schedule was 625 mg PO weekly in cohorts A (PC-PG) +B (other neuroendocrine tumors) and 625 mg orally on 2 consecutive days each week in cohort C which included 5 responding patients. The primary endpoint was radiographic response measured using RECIST criteria. Secondary endpoints included progression-free survival, overall survival, and safety.

RESULTS: In Arm A (n=10; all PC-PG) 50% (5/10) exhibited a partial-response (PR) and 2 additional patients had stable-disease (SD) >3 months. Median duration of therapy for Arm A patients was 9 months (range: 1.5 months-33 months) with 5 patients treated >1 year. In Arm B (n=12) there were 1 PR (DSRCT) and 2 SD (DSRCT; neuroblastoma) >3 months. Median duration of therapy in Arm A was 18 months (range: 1-33 months) and arm B was 3 months (range 1.5-33 months). Arm C PC-PG (N=8) showed 1 PR and 7 SD at 3 months with median duration of therapy>10 months. There was no decline in Karnofsky Performance Status (KPS) at week 12 for 28/30 patients and no dose modification due to treatment-related adverse events.

CONCLUSIONS: Oral ONC201 was well tolerated in patients with metastatic neuroendocrine tumors and associated with clinical benefit including tumor responses, particularly in some DSRCT patients and the majority of PC-PG patients.

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