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ONC201 in Recurrent/Refractory Metastatic Breast Cancer and Advanced Endometrial Carcinoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03394027
Recruitment Status : Completed
First Posted : January 9, 2018
Results First Posted : January 12, 2022
Last Update Posted : January 12, 2022
Sponsor:
Information provided by (Responsible Party):
Alexandra Zimmer, M.D., National Cancer Institute (NCI)

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Triple Negative Breast Cancer
Endometrial Cancer
Hormone Receptor Positive, HER2 Negative Breast Cancer
Intervention Drug: ONC201
Enrollment 30
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Cohort 1-Hormone Receptor (HR) + Breast Cancer (Male and Female) Cohort 2-Triple Negative Breast Cancer (Male and Female) Cohort 3-Endometrial Cancer (Female Only)
Hide Arm/Group Description 625mg ONC201 by mouth every 7 days 625mg ONC201 by mouth every 7 days 625mg ONC201 by mouth every 7 days
Period Title: Overall Study
Started 10 6 14
Completed 7 5 10
Not Completed 3 1 4
Reason Not Completed
Ineligible             3             1             3
Participant declined to participate before treatment started             0             0             1
Arm/Group Title Cohort 1-Hormone Receptor (HR) + Breast Cancer (Male and Female) Cohort 2-Triple Negative Breast Cancer (Male and Female) Cohort 3-Endometrial Cancer (Female Only) Total
Hide Arm/Group Description 625mg ONC201 by mouth every 7 days 625mg ONC201 by mouth every 7 days 625mg ONC201 by mouth every 7 days Total of all reporting groups
Overall Number of Baseline Participants 10 6 14 30
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 6 participants 14 participants 30 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
8
  80.0%
6
 100.0%
8
  57.1%
22
  73.3%
>=65 years
2
  20.0%
0
   0.0%
6
  42.9%
8
  26.7%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 10 participants 6 participants 14 participants 30 participants
60.14  (6.25) 53.88  (8.37) 63.65  (8.99) 60.53  (8.62)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 6 participants 14 participants 30 participants
Female
10
 100.0%
6
 100.0%
14
 100.0%
30
 100.0%
Male
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 6 participants 14 participants 30 participants
Hispanic or Latino
1
  10.0%
0
   0.0%
0
   0.0%
1
   3.3%
Not Hispanic or Latino
8
  80.0%
5
  83.3%
13
  92.9%
26
  86.7%
Unknown or Not Reported
1
  10.0%
1
  16.7%
1
   7.1%
3
  10.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 6 participants 14 participants 30 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
1
   7.1%
1
   3.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
1
  16.7%
0
   0.0%
1
   3.3%
Black or African American
1
  10.0%
3
  50.0%
2
  14.3%
6
  20.0%
White
8
  80.0%
2
  33.3%
11
  78.6%
21
  70.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
1
  10.0%
0
   0.0%
0
   0.0%
1
   3.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 10 participants 6 participants 14 participants 30 participants
10 6 14 30
1.Primary Outcome
Title Cohort 1 - Progression-free Survival (PFS)
Hide Description PFS in participants with refractory, metastatic hormone receptor positive breast cancer. PFS is defined as the duration of time from start of treatment to time of progression or death, whichever occurs first assessed by the Response Evaluation Criteria in Solid Tumors (RECIST). Progression is at least a 20% increase in the sum of the diameters of target lesions. And the appearance of one or more new lesions.
Time Frame Every 8 weeks, while on trial treatment
Hide Outcome Measure Data
Hide Analysis Population Description
7/10 participants were analyzed because 3 participants were ineligible.
Arm/Group Title Cohort 1-Hormone Receptor (HR) + Breast Cancer (Male and Female)
Hide Arm/Group Description:
625mg ONC201 by mouth every 7 days.
Overall Number of Participants Analyzed 7
Median (Full Range)
Unit of Measure: Months
1.9
(0.6 to 4)
2.Primary Outcome
Title Cohort 2 - Overall Response Rate (ORR) (Complete Response (CR) + Partial Response (PR) by the Response Evaluation Criteria in Solid Tumors (RECIST)
Hide Description Overall response (Complete response (CR) + Partial Response (PR) of ONC201 in participants with metastatic triple negative breast cancer was measured by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete response is disappearance of all target lesions. Partial Response is at least a 30% decrease in the sum of the diameters of target lesions.
Time Frame Every 8 weeks, while on trial treatment
Hide Outcome Measure Data
Hide Analysis Population Description
5/6 participants were analyzed because 1 participant was ineligible.
Arm/Group Title Cohort 2-Triple Negative Breast Cancer (Male and Female)
Hide Arm/Group Description:
625mg ONC201 by mouth every 7 days.
Overall Number of Participants Analyzed 5
Measure Type: Count of Participants
Unit of Measure: Participants
Complete Response
0
   0.0%
Partial Response
0
   0.0%
3.Primary Outcome
Title Cohort 3 - Overall Response Rate (ORR) (Complete Response (CR) + Partial Response (PR) by the Response Evaluation Criteria in Solid Tumors (RECIST)
Hide Description Overall response (Complete response (CR) + Partial Response (PR) of ONC201 in participants with advanced or metastatic endometrial cancer was measured by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete response is disappearance of all target lesions. Partial Response is at least a 30% decrease in the sum of the diameters of target lesions.
Time Frame Every 8 weeks, while on trial treatment
Hide Outcome Measure Data
Hide Analysis Population Description
10/14 participants were analyzed because 3 participants were ineligible, and 1 participant declined to participate before treatment started.
Arm/Group Title Cohort 3-Endometrial Cancer (Female Only)
Hide Arm/Group Description:
625mg ONC201 by mouth every 7 days.
Overall Number of Participants Analyzed 10
Measure Type: Count of Participants
Unit of Measure: Participants
Complete Response
0
   0.0%
Partial Response
0
   0.0%
4.Secondary Outcome
Title Number of Serious and Non-serious Adverse Events Grade ≥1 in Cohorts 1, 2, and 3
Hide Description Serious and non-serious adverse events were assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. Grade 1 is mild, Grade 2 is moderate, Grade 3 is severe, , Grade 4 is life-threatening, and Grade 5 is death related to adverse event.
Time Frame Date treatment consent signed to date off study, approximately 13 months and 28 days for cohort 1, 11 months and 12 days for cohort 2, and 34 months and 29 days for cohort 3.
Hide Outcome Measure Data
Hide Analysis Population Description
7/10 participants were analyzed in cohort 1 because 3 participants were ineligible. 5/6 participants were analyzed in cohort 2 because 1 participant was ineligible. And 10/14 participants were analyzed in cohort 3 because 3 participants were ineligible, and 1 participant declined to participate before treatment started. Serious adverse events are denoted by an *.
Arm/Group Title Cohort 1 - Grade 1 Cohort 1 - Grade 2 Cohort 1 - Grade 3 Cohort 1 - Grade 4 Cohort 1 - Grade 5 Cohort 2 - Grade 1 Cohort 2 - Grade 2 Cohort 2 - Grade 3 Cohort 2 - Grade 4 Cohort 2 - Grade 5 Cohort 3 - Grade 1 Cohort 3 - Grade 2 Cohort 3 - Grade 3 Cohort 3 - Grade 4 Cohort 3 - Grade 5
Hide Arm/Group Description:
Grade 1 is mild.
Grade 2 is moderate.
Grade 3 is severe.
Grade 4 is life-threatening.
Grade 5 is death related to adverse event.
Grade 1 is mild.
Grade 2 is moderate.
Grade 3 is severe.
Grade 4 is life-threatening.
Grade 5 is death related to adverse event.
Grade 1 is mild.
Grade 2 is moderate.
Grade 3 is severe.
Grade 4 is life-threatening.
Grade 5 is death related to adverse event.
Overall Number of Participants Analyzed 7 7 7 7 7 5 5 5 5 5 10 10 10 10 10
Measure Type: Number
Unit of Measure: Adverse events
Abdominal distension 0 0 0 0 0 1 0 0 0 0 0 0 0 0 0
Abdominal pain 0 0 0 0 0 2 0 0 0 0 0 0 0 0 0
*Abdominal pain 0 1 0 0 0 0 1 0 0 0 0 0 0 0 0
Alanine aminotransferase increased 2 0 1 0 0 1 1 0 0 0 1 2 0 0 0
Alkaline phosphatase increased 0 2 1 0 0 1 1 1 0 0 2 2 0 0 0
Anemia 2 0 0 0 0 1 5 3 0 0 2 2 0 0 0
*Anemia 0 0 0 0 0 1 1 1 0 0 0 0 0 0 0
Anorexia 4 1 0 0 0 0 0 0 0 0 0 1 0 0 0
Arthralgia 0 0 0 0 0 0 0 0 0 0 1 0 0 0 0
Ascites 0 1 0 0 0 0 0 0 0 0 0 0 0 0 0
Aspartate aminotransferase increased 0 1 3 1 0 0 1 1 1 0 3 1 0 0 0
*Atrial flutter 0 0 0 0 0 0 0 0 0 0 0 0 1 0 0
Back pain 0 0 0 0 0 0 0 0 0 0 0 1 0 0 0
Blood bilirubin increased 0 0 0 0 0 1 1 1 0 0 0 0 0 0 0
Breast pain 0 0 0 0 0 1 1 0 0 0 0 0 0 0 0
*Breast pain 0 0 0 0 0 0 0 1 0 0 0 0 0 0 0
*Cardiac arrest 0 0 0 0 0 0 0 0 0 0 0 0 0 0 1
Confusion 0 0 0 0 0 1 0 0 0 0 0 0 0 0 0
Constipation 0 0 3 0 0 1 0 0 0 0 1 0 0 0 0
Cough 1 0 0 0 0 1 0 0 0 0 1 0 0 0 0
Creatinine increased 0 0 0 0 0 1 0 0 0 0 0 2 0 0 0
Dehydration 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0
*Dehydration 0 0 0 0 0 0 0 0 0 0 0 0 1 0 0
Diarrhea 2 0 0 0 0 0 0 0 0 0 3 0 0 0 0
*Diarrhea 0 0 0 0 0 0 0 0 0 0 0 1 0 0 0
Dizziness 1 0 0 0 0 0 0 0 0 0 5 0 0 0 0
*Dysarthria 0 1 0 0 0 0 0 0 0 0 0 0 0 0 0
Dysgeusia 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0
Dyspepsia 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0
Dyspnea 0 1 0 0 0 0 0 0 0 0 1 1 0 0 0
*Dyspnea 0 0 0 0 0 0 0 1 0 0 0 0 0 0 0
Edema limbs 1 0 0 0 0 0 0 0 0 0 0 1 0 0 0
Epistaxis 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0
Facial pain 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0
Fall 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0
*Fall 0 0 1 0 0 0 0 0 0 0 0 0 0 0 0
Fatigue 2 1 0 0 0 1 0 0 0 0 5 2 0 0 0
*Fatigue 0 0 1 0 0 0 0 0 0 0 0 0 0 0 0
Fever 3 0 0 0 0 1 0 0 0 0 0 0 0 0 0
*Fever 0 0 0 0 0 1 0 0 0 0 0 0 0 0 0
Flu like symptoms 0 1 0 0 0 0 0 0 0 0 1 0 0 0 0
Flushing 0 0 0 0 0 1 0 0 0 0 0 0 0 0 0
Gastroesophageal reflux disease 0 0 0 0 0 0 0 0 0 0 1 0 0 0 0
Headache 0 0 0 0 0 0 0 0 0 0 2 0 0 0 0
Hot flashes 0 0 0 0 0 1 0 0 0 0 0 0 0 0 0
Hyperhidrosis 1 0 0 0 0 1 0 0 0 0 0 0 0 0 0
Hypermagnesemia 1 0 0 0 0 0 0 1 0 0 0 0 0 0 0
Hypoalbuminemia 0 1 0 0 0 1 1 1 0 0 0 0 0 0 0
Hypocalcemia 0 0 0 0 0 1 0 0 0 0 0 0 0 0 0
Hypomagnesemia 0 0 0 0 0 2 0 0 0 0 0 0 0 0 0
Hypophosphatemia 0 0 0 0 0 0 0 0 0 0 1 1 0 0 0
Hypotension 0 0 0 0 0 1 0 1 0 0 0 0 0 0 0
Insomnia 0 0 0 0 0 1 0 0 0 0 0 0 0 0 0
Laryngeal hemorrhage 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0
Lethargy 1 0 0 0 0 0 0 0 0 0 1 0 0 0 0
Lymphedema 0 0 0 0 0 0 0 0 0 0 1 0 0 0 0
Lymphocyte count decreased 2 2 1 0 0 1 1 0 0 0 2 2 2 0 0
Malaise 1 0 0 0 0 0 0 0 0 0 1 0 0 0 0
Memory impairment 0 0 0 0 0 1 0 0 0 0 0 0 0 0 0
Mucosal infection 0 0 0 0 0 0 0 0 0 0 0 1 0 0 0
Myalgia 1 0 0 0 0 1 0 0 0 0 1 0 0 0 0
Nausea 3 1 0 0 0 3 0 0 0 0 2 0 0 0 0
*Nausea 0 0 0 0 0 0 0 1 0 0 0 0 0 0 0
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify 0 0 0 0 1 0 0 0 0 0 0 0 0 0 0
*Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify 0 0 0 0 1 0 0 0 0 1 0 0 0 0 1
Nervous system disorders - Other, specify 0 0 0 0 0 1 0 0 0 0 0 0 0 0 0
Non-cardiac chest pain 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0
Pain 1 1 0 0 0 0 0 0 0 0 0 0 0 0 0
*Pain 0 0 1 0 0 0 0 0 0 0 0 0 1 0 0
Pain in extremity 0 1 0 0 0 0 0 0 0 0 0 0 0 0 0
Paresthesia 0 0 0 0 0 0 0 0 0 0 1 0 0 0 0
*Peripheral sensory neuropathy 0 1 0 0 0 0 0 0 0 0 0 0 0 0 0
Platelet count decreased 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0
Pleural effusion 0 1 0 0 0 0 0 0 0 0 0 1 0 0 0
*Pleural effusion 0 0 0 0 0 0 1 0 0 0 0 0 0 0 0
Proteinuria 1 1 0 0 0 0 0 0 0 0 1 0 0 0 0
*Pneumonitis 0 0 0 0 0 0 1 0 0 0 0 0 0 0 0
Pruritus 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0
Rash maculo-papular 0 0 0 0 0 0 0 0 0 0 1 0 0 0 0
Rhinorrhea 0 0 0 0 0 0 0 0 0 0 1 0 0 0 0
*Sepsis 0 0 0 0 0 0 0 1 0 0 0 0 0 1 0
Sinus tachycardia 0 0 0 0 0 0 1 0 0 0 2 0 0 0 0
*Sinus tachycardia 0 0 0 0 0 0 0 0 0 0 1 0 0 0 0
*Stroke 0 0 0 0 0 0 0 0 0 0 0 0 1 0 0
*Thromboembolic event 0 0 0 0 0 0 0 0 0 0 0 1 1 0 0
*Transient ischemic attacks 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0
*Tremor 0 1 0 0 0 0 0 0 0 0 0 0 0 0 0
*Tumor hemorrhage 0 0 0 0 0 0 1 1 0 0 0 0 0 0 0
Upper respiratory infection 0 1 0 0 0 0 0 0 0 0 0 0 0 0 0
Urinary tract infection 0 0 1 0 0 0 0 0 0 0 0 0 0 0 0
*Urinary tract infection 0 0 0 0 0 0 0 0 0 0 0 0 1 0 0
Vaginal discharge 0 0 0 0 0 0 0 0 0 0 1 0 0 0 0
Vaginal hemorrhage 0 0 0 0 0 0 0 0 0 0 1 0 0 0 0
Vascular access complication 0 0 0 0 0 0 0 0 0 0 0 0 1 0 0
Vomiting 3 0 0 0 0 1 0 0 0 0 0 0 0 0 0
*Vomiting 0 0 0 0 0 1 0 0 0 0 0 0 0 0 0
Weight loss 0 1 0 0 0 0 0 0 0 0 1 0 0 0 0
Wheezing 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0
White blood cell decreased 1 0 0 0 0 0 0 0 0 0 0 0 0 0 0
5.Secondary Outcome
Title Cohort 1 - Overall Response Rate (Complete Response (CR) + Partial Response (PR) by the Response Evaluation Criteria in Solid Tumors (RECIST)
Hide Description Overall response (Complete response (CR) + Partial Response (PR) of ONC201 in participants with refractory, metastatic hormone receptor positive breast cancer was measured by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete response is disappearance of all target lesions. Partial Response is at least a 30% decrease in the sum of the diameters of target lesions.
Time Frame Every 8 weeks, while on trial treatment
Hide Outcome Measure Data
Hide Analysis Population Description
7/10 participants were analyzed because 3 participants were ineligible.
Arm/Group Title Cohort 1-Hormone Receptor (HR) + Breast Cancer (Male and Female)
Hide Arm/Group Description:
625mg ONC201 by mouth every 7 days.
Overall Number of Participants Analyzed 7
Measure Type: Count of Participants
Unit of Measure: Participants
Complete Response
0
   0.0%
Partial Response
0
   0.0%
6.Secondary Outcome
Title Number of Participants in Cohorts 1, 2, and 3 With Clinical Benefit
Hide Description Clinical benefit is Complete Response (CR) + Partial Response (PR) + Stable Disease (SD) measured by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete response is disappearance of all target lesions. Partial Response is at least a 30% decrease in the sum of the diameters of target lesions. Stable Disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (i.e., at least a 20% increase in the sum of the diameters of target lesions).
Time Frame Every 8 weeks, while on treatment
Hide Outcome Measure Data
Hide Analysis Population Description
7/10 participants were analyzed in cohort 1 because 3 participants were ineligible. 5/6 participants were analyzed in cohort 2 because 1 participant was ineligible. And 10/14 participants were analyzed in cohort 3 because 3 participants were ineligible, and 1 participant declined to participate before treatment started.
Arm/Group Title Cohort 1-Hormone Receptor (HR) + Breast Cancer (Male and Female) Cohort 2-Triple Negative Breast Cancer (Male and Female) Cohort 3-Endometrial Cancer (Female Only)
Hide Arm/Group Description:
625mg ONC201 by mouth every 7 days.
625mg ONC201 by mouth every 7 days.
625mg ONC201 by mouth every 7 days.
Overall Number of Participants Analyzed 7 5 10
Measure Type: Count of Participants
Unit of Measure: Participants
Complete Response
0
   0.0%
0
   0.0%
0
   0.0%
Partial Response
0
   0.0%
0
   0.0%
0
   0.0%
Stable Disease
1
  14.3%
1
  20.0%
2
  20.0%
7.Secondary Outcome
Title Cohorts 2 and 3 - Progression-free Survival (PFS)
Hide Description PFS in participants with triple negative breast cancer and endometrial cancer. PFS is defined as the duration of time from start of treatment to time of progression or death, whichever occurs first assessed by the Response Evaluation Criteria in Solid Tumors (RECIST). Progression is at least a 20% increase in the sum of the diameters of target lesions. And the appearance of one or more new lesions.
Time Frame Every 8 weeks, while on treatment
Hide Outcome Measure Data
Hide Analysis Population Description
5/6 participants were analyzed in cohort 2 because 1 participant was ineligible. And 10/14 participants were analyzed in cohort 3 because 3 participants were ineligible, and 1 participant declined to participate before treatment started.
Arm/Group Title Cohort 2-Triple Negative Breast Cancer (Male and Female) Cohort 3-Endometrial Cancer (Female Only)
Hide Arm/Group Description:
625mg ONC201 by mouth every 7 days.
625mg ONC201 by mouth every 7 days.
Overall Number of Participants Analyzed 5 10
Median (Full Range)
Unit of Measure: Months
1.2
(0.5 to 1.8)
2
(0.6 to 3)
8.Other Pre-specified Outcome
Title Here is the Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0).
Hide Description Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.
Time Frame Date treatment consent signed to date off study, approximately 13 months and 28 days for cohort 1, 11 months and 12 days for cohort 2, and 34 months and 29 days for cohort 3.
Hide Outcome Measure Data
Hide Analysis Population Description
7/10 participants were analyzed in cohort 1 because 3 participants were ineligible. 5/6 participants were analyzed in cohort 2 because 1 participant was ineligible. And 10/14 participants were analyzed in cohort 3 because 3 participants were ineligible, and 1 participant declined to participate before treatment started.
Arm/Group Title Cohort 1-Hormone Receptor (HR) + Breast Cancer (Male and Female) Cohort 2-Triple Negative Breast Cancer (Male and Female) Cohort 3-Endometrial Cancer (Female Only)
Hide Arm/Group Description:
625mg ONC201 by mouth every 7 days.
625mg ONC201 by mouth every 7 days.
625mg ONC201 by mouth every 7 days.
Overall Number of Participants Analyzed 7 5 10
Measure Type: Count of Participants
Unit of Measure: Participants
7
 100.0%
5
 100.0%
10
 100.0%
Time Frame Date treatment consent signed to date off study, approximately 13 months and 28 days for cohort 1, 11 months and 12 days for cohort 2, and 34 months and 29 days for cohort 3.
Adverse Event Reporting Description 7/10 participants were analyzed in cohort 1 because 3 participants were ineligible. 5/6 participants were analyzed in cohort 2 because 1 participant was ineligible. And 10/14 participants were analyzed in cohort 3 because 3 participants were ineligible, and 1 participant declined to participate before treatment started.
 
Arm/Group Title Cohort 1-Hormone Receptor (HR) + Breast Cancer (Male and Female) Cohort 2-Triple Negative Breast Cancer (Male and Female) Cohort 3-Endometrial Cancer (Female Only)
Hide Arm/Group Description 625mg ONC201 by mouth every 7 days 625mg ONC201 by mouth every 7 days 625mg ONC201 by mouth every 7 days
All-Cause Mortality
Cohort 1-Hormone Receptor (HR) + Breast Cancer (Male and Female) Cohort 2-Triple Negative Breast Cancer (Male and Female) Cohort 3-Endometrial Cancer (Female Only)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/7 (28.57%)      1/5 (20.00%)      2/10 (20.00%)    
Hide Serious Adverse Events
Cohort 1-Hormone Receptor (HR) + Breast Cancer (Male and Female) Cohort 2-Triple Negative Breast Cancer (Male and Female) Cohort 3-Endometrial Cancer (Female Only)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   4/7 (57.14%)      3/5 (60.00%)      5/10 (50.00%)    
Blood and lymphatic system disorders       
Anemia  1  0/7 (0.00%)  0 1/5 (20.00%)  3 0/10 (0.00%)  0
Cardiac disorders       
Atrial flutter  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Cardiac arrest  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Sinus tachycardia  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Gastrointestinal disorders       
Abdominal pain  1  1/7 (14.29%)  1 1/5 (20.00%)  1 0/10 (0.00%)  0
Diarrhea  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Nausea  1  0/7 (0.00%)  0 1/5 (20.00%)  1 0/10 (0.00%)  0
Vomiting  1  0/7 (0.00%)  0 1/5 (20.00%)  1 0/10 (0.00%)  0
General disorders       
Fatigue  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Fever  1  0/7 (0.00%)  0 1/5 (20.00%)  1 0/10 (0.00%)  0
Pain  1  1/7 (14.29%)  1 0/5 (0.00%)  0 1/10 (10.00%)  1
Infections and infestations       
Sepsis  1  0/7 (0.00%)  0 1/5 (20.00%)  1 1/10 (10.00%)  1
Urinary tract infection  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Injury, poisoning and procedural complications       
Fall  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Metabolism and nutrition disorders       
Dehydration  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)       
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, Death  1  1/7 (14.29%)  1 1/5 (20.00%)  1 1/10 (10.00%)  1
Tumor hemorrhage  1  0/7 (0.00%)  0 1/5 (20.00%)  2 0/10 (0.00%)  0
Nervous system disorders       
Dysarthria  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Peripheral sensory neuropathy  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Stroke  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Transient ischemic attacks  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Tremor  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Reproductive system and breast disorders       
Breast pain  1  0/7 (0.00%)  0 1/5 (20.00%)  1 0/10 (0.00%)  0
Respiratory, thoracic and mediastinal disorders       
Dyspnea  1  0/7 (0.00%)  0 1/5 (20.00%)  1 0/10 (0.00%)  0
Pleural effusion  1  0/7 (0.00%)  0 1/5 (20.00%)  1 0/10 (0.00%)  0
Pneumonitis  1  0/7 (0.00%)  0 1/5 (20.00%)  1 0/10 (0.00%)  0
Vascular disorders       
Thromboembolic event  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  2
1
Term from vocabulary, CTCAE (5.0)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Cohort 1-Hormone Receptor (HR) + Breast Cancer (Male and Female) Cohort 2-Triple Negative Breast Cancer (Male and Female) Cohort 3-Endometrial Cancer (Female Only)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   7/7 (100.00%)      5/5 (100.00%)      10/10 (100.00%)    
Blood and lymphatic system disorders       
Anemia  1  2/7 (28.57%)  2 3/5 (60.00%)  9 2/10 (20.00%)  4
Cardiac disorders       
Sinus tachycardia  1  0/7 (0.00%)  0 1/5 (20.00%)  1 1/10 (10.00%)  2
Gastrointestinal disorders       
Abdominal distension  1  0/7 (0.00%)  0 1/5 (20.00%)  1 0/10 (0.00%)  0
Abdominal pain  1  0/7 (0.00%)  0 2/5 (40.00%)  2 2/10 (20.00%)  2
Ascites  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Constipation  1  3/7 (42.86%)  3 1/5 (20.00%)  1 1/10 (10.00%)  1
Diarrhea  1  2/7 (28.57%)  2 0/5 (0.00%)  0 3/10 (30.00%)  3
Dyspepsia  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Gastroesophageal reflux disease  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Nausea  1  3/7 (42.86%)  4 2/5 (40.00%)  3 2/10 (20.00%)  2
Vomiting  1  3/7 (42.86%)  3 1/5 (20.00%)  1 0/10 (0.00%)  0
General disorders       
Edema limbs  1  1/7 (14.29%)  1 0/5 (0.00%)  0 1/10 (10.00%)  1
Facial pain  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Fatigue  1  3/7 (42.86%)  3 1/5 (20.00%)  1 6/10 (60.00%)  7
Fever  1  2/7 (28.57%)  3 1/5 (20.00%)  1 0/10 (0.00%)  0
Flu like symptoms  1  1/7 (14.29%)  1 0/5 (0.00%)  0 1/10 (10.00%)  1
Malaise  1  1/7 (14.29%)  1 0/5 (0.00%)  0 1/10 (10.00%)  1
Pain  1  2/7 (28.57%)  2 0/5 (0.00%)  0 1/10 (10.00%)  1
Infections and infestations       
Mucosal infection  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Upper respiratory infection  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Urinary tract infection  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Injury, poisoning and procedural complications       
Fall  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Vascular access complication  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Investigations       
Alanine aminotransferase increased  1  3/7 (42.86%)  3 1/5 (20.00%)  2 3/10 (30.00%)  3
Alkaline phosphatase increased  1  2/7 (28.57%)  3 1/5 (20.00%)  3 4/10 (40.00%)  4
Aspartate aminotransferase increased  1  4/7 (57.14%)  5 1/5 (20.00%)  3 4/10 (40.00%)  4
Blood bilirubin increased  1  2/7 (28.57%)  2 1/5 (20.00%)  3 0/10 (0.00%)  0
Creatinine increased  1  0/7 (0.00%)  0 1/5 (20.00%)  1 1/10 (10.00%)  2
Lymphocyte count decreased  1  3/7 (42.86%)  5 1/5 (20.00%)  2 4/10 (40.00%)  6
Platelet count decreased  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Weight loss  1  1/7 (14.29%)  1 0/5 (0.00%)  0 1/10 (10.00%)  1
White blood cell decreased  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Metabolism and nutrition disorders       
Anorexia  1  4/7 (57.14%)  5 0/5 (0.00%)  0 1/10 (10.00%)  1
Dehydration  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Hypermagnesemia  1  1/7 (14.29%)  1 1/5 (20.00%)  1 0/10 (0.00%)  0
Hypoalbuminemia  1  1/7 (14.29%)  1 2/5 (40.00%)  3 0/10 (0.00%)  0
Hypocalcemia  1  0/7 (0.00%)  0 1/5 (20.00%)  1 0/10 (0.00%)  0
Hypomagnesemia  1  0/7 (0.00%)  0 1/5 (20.00%)  2 0/10 (0.00%)  0
Hypophosphatemia  1  0/7 (0.00%)  0 0/5 (0.00%)  0 2/10 (20.00%)  2
Musculoskeletal and connective tissue disorders       
Arthralgia  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Back pain  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Myalgia  1  1/7 (14.29%)  1 1/5 (20.00%)  1 1/10 (10.00%)  1
Non-cardiac chest pain  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Pain in extremity  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)       
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Nervous system disorders       
Dizziness  1  1/7 (14.29%)  1 0/5 (0.00%)  0 4/10 (40.00%)  5
Dysgeusia  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Headache  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  2
Lethargy  1  1/7 (14.29%)  1 0/5 (0.00%)  0 1/10 (10.00%)  1
Memory impairment  1  0/7 (0.00%)  0 1/5 (20.00%)  1 0/10 (0.00%)  0
Nervous system disorders - Other, Balance issues  1  0/7 (0.00%)  0 1/5 (20.00%)  1 0/10 (0.00%)  0
Paresthesia  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Psychiatric disorders       
Confusion  1  0/7 (0.00%)  0 1/5 (20.00%)  1 0/10 (0.00%)  0
Insomnia  1  0/7 (0.00%)  0 1/5 (20.00%)  1 0/10 (0.00%)  0
Renal and urinary disorders       
Proteinuria  1  2/7 (28.57%)  2 0/5 (0.00%)  0 1/10 (10.00%)  1
Reproductive system and breast disorders       
Breast pain  1  0/7 (0.00%)  0 1/5 (20.00%)  2 0/10 (0.00%)  0
Vaginal discharge  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Vaginal hemorrhage  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Respiratory, thoracic and mediastinal disorders       
Cough  1  1/7 (14.29%)  1 1/5 (20.00%)  1 1/10 (10.00%)  1
Dyspnea  1  1/7 (14.29%)  1 0/5 (0.00%)  0 2/10 (20.00%)  2
Epistaxis  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Laryngeal hemorrhage  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Pleural effusion  1  1/7 (14.29%)  1 0/5 (0.00%)  0 1/10 (10.00%)  1
Rhinorrhea  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Wheezing  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Skin and subcutaneous tissue disorders       
Hyperhidrosis  1  1/7 (14.29%)  1 1/5 (20.00%)  1 0/10 (0.00%)  0
Pruritus  1  1/7 (14.29%)  1 0/5 (0.00%)  0 0/10 (0.00%)  0
Rash maculo-papular  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
Vascular disorders       
Flushing  1  0/7 (0.00%)  0 1/5 (20.00%)  1 0/10 (0.00%)  0
Hot flashes  1  0/7 (0.00%)  0 1/5 (20.00%)  1 0/10 (0.00%)  0
Hypotension  1  0/7 (0.00%)  0 1/5 (20.00%)  2 0/10 (0.00%)  0
Lymphedema  1  0/7 (0.00%)  0 0/5 (0.00%)  0 1/10 (10.00%)  1
1
Term from vocabulary, CTCAE (5.0)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Alexandra Zimmer
Organization: National Cancer Institute
Phone: 240-760-6132
EMail: alexandra.zimmer@nih.gov
Layout table for additonal information
Responsible Party: Alexandra Zimmer, M.D., National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT03394027    
Other Study ID Numbers: 180034
18-C-0034
First Submitted: January 5, 2018
First Posted: January 9, 2018
Results First Submitted: December 17, 2021
Results First Posted: January 12, 2022
Last Update Posted: January 12, 2022