Trial Outcomes & Findings for ONC201 in Recurrent or Metastatic Type II Endometrial Cancer Endometrial Cancer (NCT NCT03485729)

Last Updated: 2024-12-24

Results Overview

Progression-free survival rate at 2 months was defined as the percentage of participants who exhibited progression-free survival for \>8 weeks (\>56 days) following treatment initiation; only Arm B participants were analyzed for this outcome.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

27 participants

Primary outcome timeframe

2 months (8 weeks); from treatment initiation to 2 months (8 weeks) following treatment initiation

Results posted on

2024-12-24

Participant Flow

Participant milestones

Participant milestones
Measure	Arm A Patients received 625 mg dordaviprone (ONC201) once weekly on Days 1, 8, and 15 of each 3-week cycle. Patients were required to undergo a biopsy of their tumor one day after their second dose of dordaviprone (ONC201) on Cycle 1, Day 9.	Arm B Patients received 625 mg of dordaviprone (ONC201) once weekly on Days 1, 8, and 15 of each 3-week cycle. Patients were not required to undergo a biopsy of their tumor.	Arm C Patients received 625 mg dordaviprone (ONC201) twice weekly on consecutive days on Days 1, 2, 8, 9, 15, and 16 of each 3-week cycle. Patients were required to undergo a biopsy of their tumor one day after their fourth dose of dordaviprone (ONC201) on Cycle 1, Day 10.
Overall Study STARTED	10	14	3
Overall Study COMPLETED	0	0	1
Overall Study NOT COMPLETED	10	14	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Arm A Patients received 625 mg dordaviprone (ONC201) once weekly on Days 1, 8, and 15 of each 3-week cycle. Patients were required to undergo a biopsy of their tumor one day after their second dose of dordaviprone (ONC201) on Cycle 1, Day 9.	Arm B Patients received 625 mg of dordaviprone (ONC201) once weekly on Days 1, 8, and 15 of each 3-week cycle. Patients were not required to undergo a biopsy of their tumor.	Arm C Patients received 625 mg dordaviprone (ONC201) twice weekly on consecutive days on Days 1, 2, 8, 9, 15, and 16 of each 3-week cycle. Patients were required to undergo a biopsy of their tumor one day after their fourth dose of dordaviprone (ONC201) on Cycle 1, Day 10.
Overall Study Withdrawal by Subject	0	0	1
Overall Study Disease progression	8	13	1
Overall Study Hospice care	1	0	0
Overall Study Poor performance status	1	0	0
Overall Study Illness	0	1	0

Baseline Characteristics

ONC201 in Recurrent or Metastatic Type II Endometrial Cancer Endometrial Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Arm A n=10 Participants Patients received 625 mg dordaviprone (ONC201) once weekly on Days 1, 8, and 15 of each 3-week cycle. Patients were required to undergo a biopsy of their tumor one day after their second dose of dordaviprone (ONC201) on Cycle 1, Day 9.	Arm B n=14 Participants Patients received 625 mg of dordaviprone (ONC201) once weekly on Days 1, 8, and 15 of each 3-week cycle. Patients were not required to undergo a biopsy of their tumor.	Arm C n=3 Participants Patients received 625 mg dordaviprone (ONC201) twice weekly on consecutive days on Days 1, 2, 8, 9, 15, and 16 of each 3-week cycle. Patients were required to undergo a biopsy of their tumor one day after their fourth dose of dordaviprone (ONC201) on Cycle 1, Day 10.	Total n=27 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=5 Participants	5 Participants n=7 Participants	0 Participants n=5 Participants	11 Participants n=4 Participants
Age, Categorical >=65 years	4 Participants n=5 Participants	9 Participants n=7 Participants	3 Participants n=5 Participants	16 Participants n=4 Participants
Sex: Female, Male Female	10 Participants n=5 Participants	14 Participants n=7 Participants	3 Participants n=5 Participants	27 Participants n=4 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=5 Participants	13 Participants n=7 Participants	3 Participants n=5 Participants	24 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	4 Participants n=5 Participants	4 Participants n=7 Participants	1 Participants n=5 Participants	9 Participants n=4 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants	8 Participants n=7 Participants	2 Participants n=5 Participants	15 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 Participants
Histologically confirmed metastatic or recurrent Type II endometrial cancer	10 Participants n=5 Participants	14 Participants n=7 Participants	3 Participants n=5 Participants	27 Participants n=4 Participants

PRIMARY outcome

Timeframe: 2 months (8 weeks); from treatment initiation to 2 months (8 weeks) following treatment initiation

Population: Note that only Arm B participants were planned to be assessed for progression-free survival at 2 months following treatment initiation. Participants in Arm A and Arm C were required to undergo a biopsy of their tumor during the study; participants in Arm B were not. Therefore, including participants in Arm A and Arm C may have represented a biased subset of the overall target population since some sites of metastases may not have been amenable to biopsy.

Outcome measures

Outcome measures
Measure	Arm B n=14 Participants Patients received 625 mg of dordaviprone (ONC201) once weekly on Days 1, 8, and 15 of each 3-week cycle. Patients were not required to undergo a biopsy of their tumor.
Progression-Free Survival Rate at 2 Months	3 Participants

Adverse Events

Arm A

Serious events: 7 serious events

Other events: 10 other events

Deaths: 5 deaths

Arm B

Serious events: 4 serious events

Other events: 14 other events

Deaths: 4 deaths

Arm C

Serious events: 3 serious events

Other events: 3 other events

Deaths: 2 deaths

Serious adverse events

Other adverse events

Additional Information

Chief Medical Officer

Chimerix, Inc.

Phone: 919-806-1074

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Within 12 months of the completion of the Study at all sites, if no publication of the overall multi-center results has been made, institutions are entitled to publish their locally obtained results, provided the Sponsor is given the opportunity to review and comment. Institution publications may be delayed up to an additional 60 days to allow the Sponsor to seek patent protection.
Publication restrictions are in place

Restriction type: OTHER

Measure	Arm A n=10 participants at risk Patients received 625 mg dordaviprone (ONC201) once weekly on Days 1, 8, and 15 of each 3-week cycle. Patients were required to undergo a biopsy of their tumor one day after their second dose of dordaviprone (ONC201) on Cycle 1, Day 9.	Arm B n=14 participants at risk Patients received 625 mg of dordaviprone (ONC201) once weekly on Days 1, 8, and 15 of each 3-week cycle. Patients were not required to undergo a biopsy of their tumor.	Arm C n=3 participants at risk Patients received 625 mg dordaviprone (ONC201) twice weekly on consecutive days on Days 1, 2, 8, 9, 15, and 16 of each 3-week cycle. Patients were required to undergo a biopsy of their tumor one day after their fourth dose of dordaviprone (ONC201) on Cycle 1, Day 10.
Blood and lymphatic system disorders Anaemia	10.0% 1/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Cardiac disorders Atrial fibrillation	0.00% 0/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	33.3% 1/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Cardiac disorders Cardiac failure	0.00% 0/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	33.3% 1/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Cardiac disorders Pericardial effusion	0.00% 0/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	7.1% 1/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Gastrointestinal disorders Abdominal pain	10.0% 1/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Gastrointestinal disorders Ascites	0.00% 0/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	7.1% 1/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	33.3% 1/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Gastrointestinal disorders Gastric haemorrhage	10.0% 1/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Gastrointestinal disorders Rectal haemorrhage	10.0% 1/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Gastrointestinal disorders Small intestinal obstruction	0.00% 0/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	7.1% 1/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Gastrointestinal disorders Vomiting	10.0% 1/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
General disorders Disease progression	10.0% 1/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	33.3% 1/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
General disorders Oedema peripheral	0.00% 0/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	33.3% 1/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
General disorders Pyrexia	0.00% 0/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	7.1% 1/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Infections and infestations Bacteraemia	0.00% 0/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	7.1% 1/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Infections and infestations Lung abscess	0.00% 0/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	7.1% 1/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Metabolism and nutrition disorders Decreased appetite	10.0% 1/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Metabolism and nutrition disorders Hyponatraemia	0.00% 0/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	33.3% 1/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Musculoskeletal and connective tissue disorders Flank pain	10.0% 1/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Psychiatric disorders Mental status changes	0.00% 0/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	33.3% 1/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Renal and urinary disorders Acute kidney injury	20.0% 2/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Renal and urinary disorders Hydronephrosis	10.0% 1/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Reproductive system and breast disorders Pelvic pain	0.00% 0/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	33.3% 1/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Respiratory, thoracic and mediastinal disorders Dyspnoea	10.0% 1/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	7.1% 1/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	66.7% 2/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Respiratory, thoracic and mediastinal disorders Pleural effusion	30.0% 3/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	14.3% 2/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.00% 0/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	33.3% 1/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Vascular disorders Deep vein thrombosis	0.00% 0/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	33.3% 1/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Vascular disorders Embolism	10.0% 1/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.
Vascular disorders Lymphoedema	10.0% 1/10 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/14 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.	0.00% 0/3 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months) Note: The progression of cancer under study was not considered an adverse event.