Table 2. Open in new tab Dordaviprone... | Oxford Academic

Table 2.

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Dordaviprone Clinical Trials across Non-CNS Cancers

Identifier	Conditions	Date	Phase	Intervention	Description	Published Results
NCT02250781	Solid Tumors	12 Jan 2015 to 25 Oct 2018.	Phase 1	ONC201	Testing the side effects and best dosing for ONC201 in treating patients with advanced solid tumors.	Completed. No results posted.
NCT02324621	Unspecified Adult Solid Tumor	20 Feb 2015 to 23 Oct 2018.	Phase 1	ONC201	To evaluate long-term efficacy of ONC201 in patients with solid tumors that have metastasized for patients who have previously benefitted from the drug.	Completed. Prolonged stable disease for >6 months was observed in 23.8% (5/21) of patients.
NCT02420795	Central Nervous System Lymphoma Gastric Mantle Cell Lymphoma Recurrent Mantle Cell Lymphoma Recurrent Non-Hodgkin Lymphoma Refractory Mantle Cell Lymphoma Refractory Non-Hodgkin Lymphoma Splenic Mantle Cell Lymphoma	3 Nov 2015 to 16 Nov 2020.	Phase 1/2	ONC201	Testing safety and efficacy of ONC201 in treating patients with non-Hodgkin’s lymphoma that is recurrent or refractory.	Terminated (Per PI request). Median progression-free survival on 625 mg ONC201 was a median of 5 weeks.
NCT02392572	Recurrent Acute Lymphoblastic Leukemia Recurrent Acute Myeloid Leukemia Recurrent Myelodysplastic Syndrome Refractory Acute Lymphoblastic Leukemia Refractory Acute Myeloid Leukemia Refractory Myelodysplastic Syndrome	3 Nov 2015 to 30 Nov 2023.	Phase 1/2	ONC201	Testing the safety and dosing of ONC201 in patients with relapsed/refractory acute leukemia or high-risk myelodysplastic syndrome.	Recruiting. No results posted.
NCT02609230	Advanced Solid Tumors Multiple Myeloma	5 Nov 2015 to 26 Mar 2020.	Phase 1	ONC201	Testing the safety and MTD of ONC201 in patients with advanced solid tumors or multiple myeloma.	Completed. No results posted.
NCT02863991	Multiple Myeloma	1 Jan 2016 to 31 Dec 2022.	Phase 1/2	ONC201	Open-label study of ONC201 in combination with dexamethasone for patients with relapsed/refractory multiple myeloma.	Active, not recruiting.
NCT03099499	Endometrial Cancer	8 June 2017 to Sept 2023.	Phase 2	ONC201	Testing the clinical benefit of ONC201 in women with recurrent or metastatic endometrial cancers, especially those with alterations in the PI3K/mTOR pathway.	Suspended due to slow accrual.
NCT03034200	Recurrent Neuroendocrine Tumor Metastatic Neuroendocrine Tumor	2 Aug 2017 to 30 Nov 2022.	Phase 2	ONC201	Testing whether ONC201 has efficacy in tumor and metastases reduction in PC–PG (pheochromocytoma–paraganglioma) and other neuroendocrine tumors.	Completed. The trial was separated into 3 arms; cohorts A (PC-PG) and B (other neuroendocrine tumors) were treated once a week with ONC201, while dosage was twice a week in cohort C. Of the 10 PC-PG patients in cohort A, 5 exhibited a partial response, with 2 additional patients having stable disease for more than 3 months. In arm B, there was 1 partial responder and 2 with stable disease for more than 3 months, from 12 total patients. Patients enrolled in arm C with PC-PG (n=8) showed 1 partial responder and 7 with stable disease at 3 months.
NCT03394027	Triple Negative Breast Cancer Endometrial Cancer Hormone Receptor Positive, HER2 Negative Breast Cancer	17 Jan 2018 to 7 Oct 2021.	Phase 2	ONC201	Testing ONC201 in each disease cohort to see if there is lasting efficacy in shrinking tumors.	No objective responses were achieved, however, 18% (4/22) of patients had prolonged stable disease (>9 cycles).
NCT03485729	Endometrial Cancer Recurrent	21 Mar 2018 to 31 Dec 2022.	Phase 2	ONC201	Two-stage, non-randomized, open label, 2-arm trial of ONC201 in women with metastatic or recurrent Type II endometrial cancer, who have already failed at least 1 prior chemotherapy regimen.	Active, not recruiting. No results posted.
NCT03492138	Multiple Myeloma	26 Mar 2018 to 13 Jan 2020.	Phase 1/2	ONC201 Ixazomib Dexamethasone	Single arm, open-label study for patients with relapsed/refractory multiple myeloma testing ONC201, ixazomib, and dexamethasone.	Terminated due to low enrollment. No results posted.
NCT03733119	Triple Negative Breast Cancer	13 Nov 2018 to 13 Feb 2021.	Phase 2	ONC201 Methionine-Restricted Diet	Combining ONC201 and a methionine-restricted diet for patients with metastatic or unresectable triple-negative breast cancer (TNBC)	Terminated. The trial was terminated due to slow accrual of patients and statistical analysis was not completed.
NCT03932643	Acute Myeloid Leukemia Myelodysplastic Syndromes	30 July 2019 to July 2025.	Phase 1	ONC201	Pilot study of 20 patients with AML/MDS to determine the safety and preliminary efficacy of ONC201 as a maintenance therapy.	Recruiting. No results posted.
NCT03791398	Metastatic Colorectal Cancer	15 Nov 2019 to 5 Aug 2021.	Phase 1b/2	ONC201 625 mg Nivolumab	Single arm, open label, pharmacokinetic, pharmacodynamics, and efficacy study of ONC201 in combination with Opdivo (Nivolumab) in adult patients with metastatic colorectal cancer.	Terminated. 13 patients were enrolled, and all died of disease progression. Median extension of survival was 58 days. The study was terminated due to lack of efficacy.
NCT04055649	Malignant Ovarian Epithelial Tumor Platinum-Resistant Fallopian Tube Carcinoma Platinum-Resistant Ovarian Carcinoma Platinum-Resistant Primary Peritoneal Carcinoma Recurrent Fallopian Tube Carcinoma Recurrent Ovarian Carcinoma Recurrent Primary Peritoneal Carcinoma Refractory Fallopian Tube Carcinoma Refractory Ovarian Carcinoma Refractory Primary Peritoneal Carcinoma	21 Jan 2020 to 27 April 2024.	Phase 2	ONC201 Paclitaxel	An efficacy trial for combining ONC201 and paclitaxel for patients with platinum-resistant epithelial ovarian, fallopian tube, and recurrent/refractory primary peritoneal cancer.	Recruiting. No results posted.
NCT05542407	Endometrial Cancer Metastasis	15 Apr 2023 to 15 Jan 2025.	Phase 1	Atezolizumab ONC201	Testing the combination of ONC201 and atezolizumab in patients in obese and non-obese metastatic/recurrent endometrial cancer.	Not yet recruiting.
NCT05630794	Colorectal adenomatous polyp Colorectal carcinoma Familial adenomatous polyposis Multiple adenomatous polyps	13 May 2023 to 1 Feb 2025.	Phase 1	Dordaviprone (ONC201)	Testing ONC201 in preventing colorectal cancer in preventing colorectal cancer in patients with familial adenomatous polyposis (FAP) or multiple polyps.	Not yet recruiting. No results posted.

Identifier	Conditions	Date	Phase	Intervention	Description	Published Results
NCT02250781	Solid Tumors	12 Jan 2015 to 25 Oct 2018.	Phase 1	ONC201	Testing the side effects and best dosing for ONC201 in treating patients with advanced solid tumors.	Completed. No results posted.
NCT02324621	Unspecified Adult Solid Tumor	20 Feb 2015 to 23 Oct 2018.	Phase 1	ONC201	To evaluate long-term efficacy of ONC201 in patients with solid tumors that have metastasized for patients who have previously benefitted from the drug.	Completed. Prolonged stable disease for >6 months was observed in 23.8% (5/21) of patients.
NCT02420795	Central Nervous System Lymphoma Gastric Mantle Cell Lymphoma Recurrent Mantle Cell Lymphoma Recurrent Non-Hodgkin Lymphoma Refractory Mantle Cell Lymphoma Refractory Non-Hodgkin Lymphoma Splenic Mantle Cell Lymphoma	3 Nov 2015 to 16 Nov 2020.	Phase 1/2	ONC201	Testing safety and efficacy of ONC201 in treating patients with non-Hodgkin’s lymphoma that is recurrent or refractory.	Terminated (Per PI request). Median progression-free survival on 625 mg ONC201 was a median of 5 weeks.
NCT02392572	Recurrent Acute Lymphoblastic Leukemia Recurrent Acute Myeloid Leukemia Recurrent Myelodysplastic Syndrome Refractory Acute Lymphoblastic Leukemia Refractory Acute Myeloid Leukemia Refractory Myelodysplastic Syndrome	3 Nov 2015 to 30 Nov 2023.	Phase 1/2	ONC201	Testing the safety and dosing of ONC201 in patients with relapsed/refractory acute leukemia or high-risk myelodysplastic syndrome.	Recruiting. No results posted.
NCT02609230	Advanced Solid Tumors Multiple Myeloma	5 Nov 2015 to 26 Mar 2020.	Phase 1	ONC201	Testing the safety and MTD of ONC201 in patients with advanced solid tumors or multiple myeloma.	Completed. No results posted.
NCT02863991	Multiple Myeloma	1 Jan 2016 to 31 Dec 2022.	Phase 1/2	ONC201	Open-label study of ONC201 in combination with dexamethasone for patients with relapsed/refractory multiple myeloma.	Active, not recruiting.
NCT03099499	Endometrial Cancer	8 June 2017 to Sept 2023.	Phase 2	ONC201	Testing the clinical benefit of ONC201 in women with recurrent or metastatic endometrial cancers, especially those with alterations in the PI3K/mTOR pathway.	Suspended due to slow accrual.
NCT03034200	Recurrent Neuroendocrine Tumor Metastatic Neuroendocrine Tumor	2 Aug 2017 to 30 Nov 2022.	Phase 2	ONC201	Testing whether ONC201 has efficacy in tumor and metastases reduction in PC–PG (pheochromocytoma–paraganglioma) and other neuroendocrine tumors.	Completed. The trial was separated into 3 arms; cohorts A (PC-PG) and B (other neuroendocrine tumors) were treated once a week with ONC201, while dosage was twice a week in cohort C. Of the 10 PC-PG patients in cohort A, 5 exhibited a partial response, with 2 additional patients having stable disease for more than 3 months. In arm B, there was 1 partial responder and 2 with stable disease for more than 3 months, from 12 total patients. Patients enrolled in arm C with PC-PG (n=8) showed 1 partial responder and 7 with stable disease at 3 months.
NCT03394027	Triple Negative Breast Cancer Endometrial Cancer Hormone Receptor Positive, HER2 Negative Breast Cancer	17 Jan 2018 to 7 Oct 2021.	Phase 2	ONC201	Testing ONC201 in each disease cohort to see if there is lasting efficacy in shrinking tumors.	No objective responses were achieved, however, 18% (4/22) of patients had prolonged stable disease (>9 cycles).
NCT03485729	Endometrial Cancer Recurrent	21 Mar 2018 to 31 Dec 2022.	Phase 2	ONC201	Two-stage, non-randomized, open label, 2-arm trial of ONC201 in women with metastatic or recurrent Type II endometrial cancer, who have already failed at least 1 prior chemotherapy regimen.	Active, not recruiting. No results posted.
NCT03492138	Multiple Myeloma	26 Mar 2018 to 13 Jan 2020.	Phase 1/2	ONC201 Ixazomib Dexamethasone	Single arm, open-label study for patients with relapsed/refractory multiple myeloma testing ONC201, ixazomib, and dexamethasone.	Terminated due to low enrollment. No results posted.
NCT03733119	Triple Negative Breast Cancer	13 Nov 2018 to 13 Feb 2021.	Phase 2	ONC201 Methionine-Restricted Diet	Combining ONC201 and a methionine-restricted diet for patients with metastatic or unresectable triple-negative breast cancer (TNBC)	Terminated. The trial was terminated due to slow accrual of patients and statistical analysis was not completed.
NCT03932643	Acute Myeloid Leukemia Myelodysplastic Syndromes	30 July 2019 to July 2025.	Phase 1	ONC201	Pilot study of 20 patients with AML/MDS to determine the safety and preliminary efficacy of ONC201 as a maintenance therapy.	Recruiting. No results posted.
NCT03791398	Metastatic Colorectal Cancer	15 Nov 2019 to 5 Aug 2021.	Phase 1b/2	ONC201 625 mg Nivolumab	Single arm, open label, pharmacokinetic, pharmacodynamics, and efficacy study of ONC201 in combination with Opdivo (Nivolumab) in adult patients with metastatic colorectal cancer.	Terminated. 13 patients were enrolled, and all died of disease progression. Median extension of survival was 58 days. The study was terminated due to lack of efficacy.
NCT04055649	Malignant Ovarian Epithelial Tumor Platinum-Resistant Fallopian Tube Carcinoma Platinum-Resistant Ovarian Carcinoma Platinum-Resistant Primary Peritoneal Carcinoma Recurrent Fallopian Tube Carcinoma Recurrent Ovarian Carcinoma Recurrent Primary Peritoneal Carcinoma Refractory Fallopian Tube Carcinoma Refractory Ovarian Carcinoma Refractory Primary Peritoneal Carcinoma	21 Jan 2020 to 27 April 2024.	Phase 2	ONC201 Paclitaxel	An efficacy trial for combining ONC201 and paclitaxel for patients with platinum-resistant epithelial ovarian, fallopian tube, and recurrent/refractory primary peritoneal cancer.	Recruiting. No results posted.
NCT05542407	Endometrial Cancer Metastasis	15 Apr 2023 to 15 Jan 2025.	Phase 1	Atezolizumab ONC201	Testing the combination of ONC201 and atezolizumab in patients in obese and non-obese metastatic/recurrent endometrial cancer.	Not yet recruiting.
NCT05630794	Colorectal adenomatous polyp Colorectal carcinoma Familial adenomatous polyposis Multiple adenomatous polyps	13 May 2023 to 1 Feb 2025.	Phase 1	Dordaviprone (ONC201)	Testing ONC201 in preventing colorectal cancer in preventing colorectal cancer in patients with familial adenomatous polyposis (FAP) or multiple polyps.	Not yet recruiting. No results posted.

Table 2.

Open in new tab

Dordaviprone Clinical Trials across Non-CNS Cancers

Identifier	Conditions	Date	Phase	Intervention	Description	Published Results
NCT02250781	Solid Tumors	12 Jan 2015 to 25 Oct 2018.	Phase 1	ONC201	Testing the side effects and best dosing for ONC201 in treating patients with advanced solid tumors.	Completed. No results posted.
NCT02324621	Unspecified Adult Solid Tumor	20 Feb 2015 to 23 Oct 2018.	Phase 1	ONC201	To evaluate long-term efficacy of ONC201 in patients with solid tumors that have metastasized for patients who have previously benefitted from the drug.	Completed. Prolonged stable disease for >6 months was observed in 23.8% (5/21) of patients.
NCT02420795	Central Nervous System Lymphoma Gastric Mantle Cell Lymphoma Recurrent Mantle Cell Lymphoma Recurrent Non-Hodgkin Lymphoma Refractory Mantle Cell Lymphoma Refractory Non-Hodgkin Lymphoma Splenic Mantle Cell Lymphoma	3 Nov 2015 to 16 Nov 2020.	Phase 1/2	ONC201	Testing safety and efficacy of ONC201 in treating patients with non-Hodgkin’s lymphoma that is recurrent or refractory.	Terminated (Per PI request). Median progression-free survival on 625 mg ONC201 was a median of 5 weeks.
NCT02392572	Recurrent Acute Lymphoblastic Leukemia Recurrent Acute Myeloid Leukemia Recurrent Myelodysplastic Syndrome Refractory Acute Lymphoblastic Leukemia Refractory Acute Myeloid Leukemia Refractory Myelodysplastic Syndrome	3 Nov 2015 to 30 Nov 2023.	Phase 1/2	ONC201	Testing the safety and dosing of ONC201 in patients with relapsed/refractory acute leukemia or high-risk myelodysplastic syndrome.	Recruiting. No results posted.
NCT02609230	Advanced Solid Tumors Multiple Myeloma	5 Nov 2015 to 26 Mar 2020.	Phase 1	ONC201	Testing the safety and MTD of ONC201 in patients with advanced solid tumors or multiple myeloma.	Completed. No results posted.
NCT02863991	Multiple Myeloma	1 Jan 2016 to 31 Dec 2022.	Phase 1/2	ONC201	Open-label study of ONC201 in combination with dexamethasone for patients with relapsed/refractory multiple myeloma.	Active, not recruiting.
NCT03099499	Endometrial Cancer	8 June 2017 to Sept 2023.	Phase 2	ONC201	Testing the clinical benefit of ONC201 in women with recurrent or metastatic endometrial cancers, especially those with alterations in the PI3K/mTOR pathway.	Suspended due to slow accrual.
NCT03034200	Recurrent Neuroendocrine Tumor Metastatic Neuroendocrine Tumor	2 Aug 2017 to 30 Nov 2022.	Phase 2	ONC201	Testing whether ONC201 has efficacy in tumor and metastases reduction in PC–PG (pheochromocytoma–paraganglioma) and other neuroendocrine tumors.	Completed. The trial was separated into 3 arms; cohorts A (PC-PG) and B (other neuroendocrine tumors) were treated once a week with ONC201, while dosage was twice a week in cohort C. Of the 10 PC-PG patients in cohort A, 5 exhibited a partial response, with 2 additional patients having stable disease for more than 3 months. In arm B, there was 1 partial responder and 2 with stable disease for more than 3 months, from 12 total patients. Patients enrolled in arm C with PC-PG (n=8) showed 1 partial responder and 7 with stable disease at 3 months.
NCT03394027	Triple Negative Breast Cancer Endometrial Cancer Hormone Receptor Positive, HER2 Negative Breast Cancer	17 Jan 2018 to 7 Oct 2021.	Phase 2	ONC201	Testing ONC201 in each disease cohort to see if there is lasting efficacy in shrinking tumors.	No objective responses were achieved, however, 18% (4/22) of patients had prolonged stable disease (>9 cycles).
NCT03485729	Endometrial Cancer Recurrent	21 Mar 2018 to 31 Dec 2022.	Phase 2	ONC201	Two-stage, non-randomized, open label, 2-arm trial of ONC201 in women with metastatic or recurrent Type II endometrial cancer, who have already failed at least 1 prior chemotherapy regimen.	Active, not recruiting. No results posted.
NCT03492138	Multiple Myeloma	26 Mar 2018 to 13 Jan 2020.	Phase 1/2	ONC201 Ixazomib Dexamethasone	Single arm, open-label study for patients with relapsed/refractory multiple myeloma testing ONC201, ixazomib, and dexamethasone.	Terminated due to low enrollment. No results posted.
NCT03733119	Triple Negative Breast Cancer	13 Nov 2018 to 13 Feb 2021.	Phase 2	ONC201 Methionine-Restricted Diet	Combining ONC201 and a methionine-restricted diet for patients with metastatic or unresectable triple-negative breast cancer (TNBC)	Terminated. The trial was terminated due to slow accrual of patients and statistical analysis was not completed.
NCT03932643	Acute Myeloid Leukemia Myelodysplastic Syndromes	30 July 2019 to July 2025.	Phase 1	ONC201	Pilot study of 20 patients with AML/MDS to determine the safety and preliminary efficacy of ONC201 as a maintenance therapy.	Recruiting. No results posted.
NCT03791398	Metastatic Colorectal Cancer	15 Nov 2019 to 5 Aug 2021.	Phase 1b/2	ONC201 625 mg Nivolumab	Single arm, open label, pharmacokinetic, pharmacodynamics, and efficacy study of ONC201 in combination with Opdivo (Nivolumab) in adult patients with metastatic colorectal cancer.	Terminated. 13 patients were enrolled, and all died of disease progression. Median extension of survival was 58 days. The study was terminated due to lack of efficacy.
NCT04055649	Malignant Ovarian Epithelial Tumor Platinum-Resistant Fallopian Tube Carcinoma Platinum-Resistant Ovarian Carcinoma Platinum-Resistant Primary Peritoneal Carcinoma Recurrent Fallopian Tube Carcinoma Recurrent Ovarian Carcinoma Recurrent Primary Peritoneal Carcinoma Refractory Fallopian Tube Carcinoma Refractory Ovarian Carcinoma Refractory Primary Peritoneal Carcinoma	21 Jan 2020 to 27 April 2024.	Phase 2	ONC201 Paclitaxel	An efficacy trial for combining ONC201 and paclitaxel for patients with platinum-resistant epithelial ovarian, fallopian tube, and recurrent/refractory primary peritoneal cancer.	Recruiting. No results posted.
NCT05542407	Endometrial Cancer Metastasis	15 Apr 2023 to 15 Jan 2025.	Phase 1	Atezolizumab ONC201	Testing the combination of ONC201 and atezolizumab in patients in obese and non-obese metastatic/recurrent endometrial cancer.	Not yet recruiting.
NCT05630794	Colorectal adenomatous polyp Colorectal carcinoma Familial adenomatous polyposis Multiple adenomatous polyps	13 May 2023 to 1 Feb 2025.	Phase 1	Dordaviprone (ONC201)	Testing ONC201 in preventing colorectal cancer in preventing colorectal cancer in patients with familial adenomatous polyposis (FAP) or multiple polyps.	Not yet recruiting. No results posted.

Identifier	Conditions	Date	Phase	Intervention	Description	Published Results
NCT02250781	Solid Tumors	12 Jan 2015 to 25 Oct 2018.	Phase 1	ONC201	Testing the side effects and best dosing for ONC201 in treating patients with advanced solid tumors.	Completed. No results posted.
NCT02324621	Unspecified Adult Solid Tumor	20 Feb 2015 to 23 Oct 2018.	Phase 1	ONC201	To evaluate long-term efficacy of ONC201 in patients with solid tumors that have metastasized for patients who have previously benefitted from the drug.	Completed. Prolonged stable disease for >6 months was observed in 23.8% (5/21) of patients.
NCT02420795	Central Nervous System Lymphoma Gastric Mantle Cell Lymphoma Recurrent Mantle Cell Lymphoma Recurrent Non-Hodgkin Lymphoma Refractory Mantle Cell Lymphoma Refractory Non-Hodgkin Lymphoma Splenic Mantle Cell Lymphoma	3 Nov 2015 to 16 Nov 2020.	Phase 1/2	ONC201	Testing safety and efficacy of ONC201 in treating patients with non-Hodgkin’s lymphoma that is recurrent or refractory.	Terminated (Per PI request). Median progression-free survival on 625 mg ONC201 was a median of 5 weeks.
NCT02392572	Recurrent Acute Lymphoblastic Leukemia Recurrent Acute Myeloid Leukemia Recurrent Myelodysplastic Syndrome Refractory Acute Lymphoblastic Leukemia Refractory Acute Myeloid Leukemia Refractory Myelodysplastic Syndrome	3 Nov 2015 to 30 Nov 2023.	Phase 1/2	ONC201	Testing the safety and dosing of ONC201 in patients with relapsed/refractory acute leukemia or high-risk myelodysplastic syndrome.	Recruiting. No results posted.
NCT02609230	Advanced Solid Tumors Multiple Myeloma	5 Nov 2015 to 26 Mar 2020.	Phase 1	ONC201	Testing the safety and MTD of ONC201 in patients with advanced solid tumors or multiple myeloma.	Completed. No results posted.
NCT02863991	Multiple Myeloma	1 Jan 2016 to 31 Dec 2022.	Phase 1/2	ONC201	Open-label study of ONC201 in combination with dexamethasone for patients with relapsed/refractory multiple myeloma.	Active, not recruiting.
NCT03099499	Endometrial Cancer	8 June 2017 to Sept 2023.	Phase 2	ONC201	Testing the clinical benefit of ONC201 in women with recurrent or metastatic endometrial cancers, especially those with alterations in the PI3K/mTOR pathway.	Suspended due to slow accrual.
NCT03034200	Recurrent Neuroendocrine Tumor Metastatic Neuroendocrine Tumor	2 Aug 2017 to 30 Nov 2022.	Phase 2	ONC201	Testing whether ONC201 has efficacy in tumor and metastases reduction in PC–PG (pheochromocytoma–paraganglioma) and other neuroendocrine tumors.	Completed. The trial was separated into 3 arms; cohorts A (PC-PG) and B (other neuroendocrine tumors) were treated once a week with ONC201, while dosage was twice a week in cohort C. Of the 10 PC-PG patients in cohort A, 5 exhibited a partial response, with 2 additional patients having stable disease for more than 3 months. In arm B, there was 1 partial responder and 2 with stable disease for more than 3 months, from 12 total patients. Patients enrolled in arm C with PC-PG (n=8) showed 1 partial responder and 7 with stable disease at 3 months.
NCT03394027	Triple Negative Breast Cancer Endometrial Cancer Hormone Receptor Positive, HER2 Negative Breast Cancer	17 Jan 2018 to 7 Oct 2021.	Phase 2	ONC201	Testing ONC201 in each disease cohort to see if there is lasting efficacy in shrinking tumors.	No objective responses were achieved, however, 18% (4/22) of patients had prolonged stable disease (>9 cycles).
NCT03485729	Endometrial Cancer Recurrent	21 Mar 2018 to 31 Dec 2022.	Phase 2	ONC201	Two-stage, non-randomized, open label, 2-arm trial of ONC201 in women with metastatic or recurrent Type II endometrial cancer, who have already failed at least 1 prior chemotherapy regimen.	Active, not recruiting. No results posted.
NCT03492138	Multiple Myeloma	26 Mar 2018 to 13 Jan 2020.	Phase 1/2	ONC201 Ixazomib Dexamethasone	Single arm, open-label study for patients with relapsed/refractory multiple myeloma testing ONC201, ixazomib, and dexamethasone.	Terminated due to low enrollment. No results posted.
NCT03733119	Triple Negative Breast Cancer	13 Nov 2018 to 13 Feb 2021.	Phase 2	ONC201 Methionine-Restricted Diet	Combining ONC201 and a methionine-restricted diet for patients with metastatic or unresectable triple-negative breast cancer (TNBC)	Terminated. The trial was terminated due to slow accrual of patients and statistical analysis was not completed.
NCT03932643	Acute Myeloid Leukemia Myelodysplastic Syndromes	30 July 2019 to July 2025.	Phase 1	ONC201	Pilot study of 20 patients with AML/MDS to determine the safety and preliminary efficacy of ONC201 as a maintenance therapy.	Recruiting. No results posted.
NCT03791398	Metastatic Colorectal Cancer	15 Nov 2019 to 5 Aug 2021.	Phase 1b/2	ONC201 625 mg Nivolumab	Single arm, open label, pharmacokinetic, pharmacodynamics, and efficacy study of ONC201 in combination with Opdivo (Nivolumab) in adult patients with metastatic colorectal cancer.	Terminated. 13 patients were enrolled, and all died of disease progression. Median extension of survival was 58 days. The study was terminated due to lack of efficacy.
NCT04055649	Malignant Ovarian Epithelial Tumor Platinum-Resistant Fallopian Tube Carcinoma Platinum-Resistant Ovarian Carcinoma Platinum-Resistant Primary Peritoneal Carcinoma Recurrent Fallopian Tube Carcinoma Recurrent Ovarian Carcinoma Recurrent Primary Peritoneal Carcinoma Refractory Fallopian Tube Carcinoma Refractory Ovarian Carcinoma Refractory Primary Peritoneal Carcinoma	21 Jan 2020 to 27 April 2024.	Phase 2	ONC201 Paclitaxel	An efficacy trial for combining ONC201 and paclitaxel for patients with platinum-resistant epithelial ovarian, fallopian tube, and recurrent/refractory primary peritoneal cancer.	Recruiting. No results posted.
NCT05542407	Endometrial Cancer Metastasis	15 Apr 2023 to 15 Jan 2025.	Phase 1	Atezolizumab ONC201	Testing the combination of ONC201 and atezolizumab in patients in obese and non-obese metastatic/recurrent endometrial cancer.	Not yet recruiting.
NCT05630794	Colorectal adenomatous polyp Colorectal carcinoma Familial adenomatous polyposis Multiple adenomatous polyps	13 May 2023 to 1 Feb 2025.	Phase 1	Dordaviprone (ONC201)	Testing ONC201 in preventing colorectal cancer in preventing colorectal cancer in patients with familial adenomatous polyposis (FAP) or multiple polyps.	Not yet recruiting. No results posted.