Trial Outcomes & Findings for Onapristone and Anastrozole for the Treatment of Refractory Hormone Receptor Positive Endometrial Cancer (NCT NCT04719273)

NCT ID: NCT04719273

Last Updated: 2026-07-17

Results Overview

Defined by the percentage of patients with tumor response (complete response \[CR\] or partial response \[PR\]) as defined by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

14 participants

Primary outcome timeframe

Up to 1 year post-treatment, up to 36 months total

Results posted on

2026-07-17

Participant Flow

Participants were recruited between January, 2021 and March, 2023, a total of 18 patients were screened across two sites, with 14 ultimately enrolled.

18 patients were screened for eligibility based on protocol defined inclusion/exclusion criteria. Eligibility was determined by histologically confirmed diagnosis of endometrial cancer with ER and/or PR expression ≥1% by IHC on archival tissue taken within the prior 3 years or new biopsy if no archival tissue is available, measurable disease as defined by RECIST v.1.1., and ECOG Performance status 0-1. Data from 14 patients were included in the FAS.

Participant milestones

Participant milestones
Measure	Treatment (Onapristone, Anastrozole) Patients receive onapristone PO BID and anastrozole PO QD on days 1-28. Treatment repeats every 28 days for up to 24 cycles (24 months) until November 30, 2023 or in the absence of disease progression or unacceptable toxicity. Extended-release Onapristone: Given PO Anastrozole: Given PO Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies Estrogen Receptor Positive (Positive Estrogen Receptor; ESR Positive; ESR1 Positive; ER Positive; Estrogen Receptor Alpha Positive): Immunohistochemistry (IHC):Integral : Tissue Progesterone Receptor Positive ( PGR Positive; PR Positive): Immunohistochemistry (IHC)
Overall Study STARTED	14
Overall Study COMPLETED	13
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Treatment (Onapristone, Anastrozole) Patients receive onapristone PO BID and anastrozole PO QD on days 1-28. Treatment repeats every 28 days for up to 24 cycles (24 months) until November 30, 2023 or in the absence of disease progression or unacceptable toxicity. Extended-release Onapristone: Given PO Anastrozole: Given PO Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies Estrogen Receptor Positive (Positive Estrogen Receptor; ESR Positive; ESR1 Positive; ER Positive; Estrogen Receptor Alpha Positive): Immunohistochemistry (IHC):Integral : Tissue Progesterone Receptor Positive ( PGR Positive; PR Positive): Immunohistochemistry (IHC)
Overall Study Adverse Event	1

Baseline Characteristics

Onapristone and Anastrozole for the Treatment of Refractory Hormone Receptor Positive Endometrial Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Onapristone, Anastrozole) n=14 Participants Patients receive onapristone PO BID and anastrozole PO QD on days 1-28. Treatment repeats every 28 days for up to 24 cycles (24 months) until November 30, 2023 or in the absence of disease progression or unacceptable toxicity. Extended-release Onapristone: Given PO Anastrozole: Given PO Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies Estrogen Receptor Positive (Positive Estrogen Receptor; ESR Positive; ESR1 Positive; ER Positive; Estrogen Receptor Alpha Positive): Immunohistochemistry (IHC):Integral : Tissue Progesterone Receptor Positive ( PGR Positive; PR Positive): Immunohistochemistry (IHC)
Age, Continuous	67 years n=20 Participants
Sex: Female, Male Female	14 Participants n=20 Participants
Sex: Female, Male Male	0 Participants n=20 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=20 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	14 Participants n=20 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=20 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=20 Participants
Race (NIH/OMB) Asian	0 Participants n=20 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=20 Participants
Race (NIH/OMB) Black or African American	4 Participants n=20 Participants
Race (NIH/OMB) White	10 Participants n=20 Participants
Race (NIH/OMB) More than one race	0 Participants n=20 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=20 Participants
Region of Enrollment United States	14 participants n=20 Participants

PRIMARY outcome

Timeframe: Up to 1 year post-treatment, up to 36 months total

Outcome measures

Outcome measures
Measure	Treatment (Onapristone, Anastrozole) n=14 Participants Patients receive onapristone PO BID and anastrozole PO QD on days 1-28. Treatment repeats every 28 days for up to 24 cycles (24 months) until November 30, 2023 or in the absence of disease progression or unacceptable toxicity. Extended-release Onapristone: Given PO Anastrozole: Given PO Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies Estrogen Receptor Positive (Positive Estrogen Receptor; ESR Positive; ESR1 Positive; ER Positive; Estrogen Receptor Alpha Positive): Immunohistochemistry (IHC):Integral : Tissue Progesterone Receptor Positive ( PGR Positive; PR Positive): Immunohistochemistry (IHC)
Objective Response Rate (ORR)	3 Participants

PRIMARY outcome

Timeframe: From treatment until disease progression or death, up to 25 months

Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.

Outcome measures

Outcome measures
Measure	Treatment (Onapristone, Anastrozole) n=14 Participants Patients receive onapristone PO BID and anastrozole PO QD on days 1-28. Treatment repeats every 28 days for up to 24 cycles (24 months) until November 30, 2023 or in the absence of disease progression or unacceptable toxicity. Extended-release Onapristone: Given PO Anastrozole: Given PO Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies Estrogen Receptor Positive (Positive Estrogen Receptor; ESR Positive; ESR1 Positive; ER Positive; Estrogen Receptor Alpha Positive): Immunohistochemistry (IHC):Integral : Tissue Progesterone Receptor Positive ( PGR Positive; PR Positive): Immunohistochemistry (IHC)
Progression-Free Survival (PFS)	14.2 Months Interval 9.6 to The reported median PFS is 14.2 months with a 95% confidence interval of 9.6 months to infinite

SECONDARY outcome

Timeframe: Up to 1 year post-treatment

Defined as best overall response of CR, PR, or stable disease lasting for \>= 24 weeks, per RECIST 1.1.

Outcome measures

Outcome measures
Measure	Treatment (Onapristone, Anastrozole) n=14 Participants Patients receive onapristone PO BID and anastrozole PO QD on days 1-28. Treatment repeats every 28 days for up to 24 cycles (24 months) until November 30, 2023 or in the absence of disease progression or unacceptable toxicity. Extended-release Onapristone: Given PO Anastrozole: Given PO Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies Estrogen Receptor Positive (Positive Estrogen Receptor; ESR Positive; ESR1 Positive; ER Positive; Estrogen Receptor Alpha Positive): Immunohistochemistry (IHC):Integral : Tissue Progesterone Receptor Positive ( PGR Positive; PR Positive): Immunohistochemistry (IHC)
Disease Control Rate	13 Participants

SECONDARY outcome

Timeframe: up to 1 yr post treatment

Population: Only participants who achieved a complete response or partial response were included in this analysis. Eleven participants did not achieve either a complete or partial response and were therefore excluded from the analysis.

Time to Response defined as time from randomization to first documented response (CR or PR) in months.

Outcome measures

Outcome measures
Measure	Treatment (Onapristone, Anastrozole) n=3 Participants Patients receive onapristone PO BID and anastrozole PO QD on days 1-28. Treatment repeats every 28 days for up to 24 cycles (24 months) until November 30, 2023 or in the absence of disease progression or unacceptable toxicity. Extended-release Onapristone: Given PO Anastrozole: Given PO Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies Estrogen Receptor Positive (Positive Estrogen Receptor; ESR Positive; ESR1 Positive; ER Positive; Estrogen Receptor Alpha Positive): Immunohistochemistry (IHC):Integral : Tissue Progesterone Receptor Positive ( PGR Positive; PR Positive): Immunohistochemistry (IHC)
Time to Response	4.14 months Interval 1.61 to 6.26

SECONDARY outcome

Timeframe: From the first date of documented response to progression or death due to endometrial cancer, assessed up to 1 year post-treatment

Duration of Response (DOR) is the time from the first documented achievement of a Partial Response (PR) or Complete Response (CR) until the date of confirmed disease Progression (PD) or death due to endometrial cancer.

Outcome measures

Outcome measures
Measure	Treatment (Onapristone, Anastrozole) n=1 Participants Patients receive onapristone PO BID and anastrozole PO QD on days 1-28. Treatment repeats every 28 days for up to 24 cycles (24 months) until November 30, 2023 or in the absence of disease progression or unacceptable toxicity. Extended-release Onapristone: Given PO Anastrozole: Given PO Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies Estrogen Receptor Positive (Positive Estrogen Receptor; ESR Positive; ESR1 Positive; ER Positive; Estrogen Receptor Alpha Positive): Immunohistochemistry (IHC):Integral : Tissue Progesterone Receptor Positive ( PGR Positive; PR Positive): Immunohistochemistry (IHC)
Duration of Response in Participants With Complete Response	16.25 months

SECONDARY outcome

Timeframe: From the first date of documented response to progression or death due to endometrial cancer, assessed up to 1 year post-treatment

Outcome measures

Outcome measures
Measure	Treatment (Onapristone, Anastrozole) n=1 Participants Patients receive onapristone PO BID and anastrozole PO QD on days 1-28. Treatment repeats every 28 days for up to 24 cycles (24 months) until November 30, 2023 or in the absence of disease progression or unacceptable toxicity. Extended-release Onapristone: Given PO Anastrozole: Given PO Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies Estrogen Receptor Positive (Positive Estrogen Receptor; ESR Positive; ESR1 Positive; ER Positive; Estrogen Receptor Alpha Positive): Immunohistochemistry (IHC):Integral : Tissue Progesterone Receptor Positive ( PGR Positive; PR Positive): Immunohistochemistry (IHC)
Duration of Response in Participants With Partial Response	25 months

SECONDARY outcome

Timeframe: Up to 25 months

Population: only 12 of the 14 patients completed the ESAS questionnaires and were included in the analysis. Two participants did not complete the questionnaires.

Quality of life and pain scores are defined by the Edmonton Symptom Assessment System (ESAS) using nine subjective patient measures of well-being including pain, tiredness, nausea, depression, anxiety, drowsiness, appetite, well-being, shortness of breath. Numerical Rating Scale (NRS): Each symptom, including Pain and Well-being (which reflects Quality of Life), is rated on a 0 to 10 scale. Pain Score: 0 = No pain, 10 = Worst possible pain. Quality of Life / Well-being Score: 0 = Best possible feeling of well-being (i.e., best QoL), 10 = Worst possible feeling of well-being (i.e., worst QoL). ESAS scores were collected over the course of treatment and follow-up. Results represent the mean score for each symptom averaged across all available assessments for each participant. Higher scores reflect greater symptom severity. Scores are reported as means with standard deviations.

Outcome measures

Outcome measures
Measure	Treatment (Onapristone, Anastrozole) n=12 Participants Patients receive onapristone PO BID and anastrozole PO QD on days 1-28. Treatment repeats every 28 days for up to 24 cycles (24 months) until November 30, 2023 or in the absence of disease progression or unacceptable toxicity. Extended-release Onapristone: Given PO Anastrozole: Given PO Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies Estrogen Receptor Positive (Positive Estrogen Receptor; ESR Positive; ESR1 Positive; ER Positive; Estrogen Receptor Alpha Positive): Immunohistochemistry (IHC):Integral : Tissue Progesterone Receptor Positive ( PGR Positive; PR Positive): Immunohistochemistry (IHC)
Quality of Life Assessed by the Edmonton Symptom Assessment System Anxiety	1.3 scores on a scale (0-10) Standard Deviation 1.1
Quality of Life Assessed by the Edmonton Symptom Assessment System Change in Appetite	2.4 scores on a scale (0-10) Standard Deviation 1.9
Quality of Life Assessed by the Edmonton Symptom Assessment System Depression	1.1 scores on a scale (0-10) Standard Deviation 1.1
Quality of Life Assessed by the Edmonton Symptom Assessment System Drowsiness	1.5 scores on a scale (0-10) Standard Deviation 1.6
Quality of Life Assessed by the Edmonton Symptom Assessment System Nausea	1.0 scores on a scale (0-10) Standard Deviation 1.5
Quality of Life Assessed by the Edmonton Symptom Assessment System Pain	2.2 scores on a scale (0-10) Standard Deviation 2.2
Quality of Life Assessed by the Edmonton Symptom Assessment System Shortness of Breath	0.9 scores on a scale (0-10) Standard Deviation 1.5
Quality of Life Assessed by the Edmonton Symptom Assessment System Tiredness	3.2 scores on a scale (0-10) Standard Deviation 2.6
Quality of Life Assessed by the Edmonton Symptom Assessment System Wellbeing	2.5 scores on a scale (0-10) Standard Deviation 1.9

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 1 year post-treatment

Assessed by immunohistochemistry and represented as a percentage prior to trial initiation and at progression. For analysis, ER/PR expression values were summarized according to each participant's best overall tumor response category-Complete Response (CR), Partial Response (PR), Stable Disease (SD), or Progressive Disease (PD)-as defined by RECIST criteria. Higher receptor expression is generally associated with more favorable treatment response, whereas lower expression is associated with poorer outcomes.

Outcome measures

Outcome measures
Measure	Treatment (Onapristone, Anastrozole) n=14 Participants Patients receive onapristone PO BID and anastrozole PO QD on days 1-28. Treatment repeats every 28 days for up to 24 cycles (24 months) until November 30, 2023 or in the absence of disease progression or unacceptable toxicity. Extended-release Onapristone: Given PO Anastrozole: Given PO Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies Estrogen Receptor Positive (Positive Estrogen Receptor; ESR Positive; ESR1 Positive; ER Positive; Estrogen Receptor Alpha Positive): Immunohistochemistry (IHC):Integral : Tissue Progesterone Receptor Positive ( PGR Positive; PR Positive): Immunohistochemistry (IHC)
Estrogen Receptor and Progesterone Receptor Expression Stable Disease (SD)	10 Participants
Estrogen Receptor and Progesterone Receptor Expression Complete Response (CR)	1 Participants
Estrogen Receptor and Progesterone Receptor Expression Progressive Disease (PD)	1 Participants
Estrogen Receptor and Progesterone Receptor Expression Partial Response (PR)	2 Participants

Adverse Events

Treatment (Onapristone, Anastrozole)

Serious events: 3 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Onapristone, Anastrozole) n=14 participants at risk Patients receive onapristone PO BID and anastrozole PO QD on days 1-28. Treatment repeats every 28 days for up to 24 cycles (24 months) until November 30, 2023 or in the absence of disease progression or unacceptable toxicity. Extended-release Onapristone: Given PO Anastrozole: Given PO Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies Estrogen Receptor Positive (Positive Estrogen Receptor; ESR Positive; ESR1 Positive; ER Positive; Estrogen Receptor Alpha Positive): Immunohistochemistry (IHC):Integral : Tissue Progesterone Receptor Positive ( PGR Positive; PR Positive): Immunohistochemistry (IHC)
Gastrointestinal disorders Small intestinal obstruction	7.1% 1/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Gastrointestinal disorders Dysphagia	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Infections and infestations Abdominal Infection	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Vascular disorders Thromboembolic event	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Nervous system disorders Dizziness	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.

Other adverse events

Other adverse events
Measure	Treatment (Onapristone, Anastrozole) n=14 participants at risk Patients receive onapristone PO BID and anastrozole PO QD on days 1-28. Treatment repeats every 28 days for up to 24 cycles (24 months) until November 30, 2023 or in the absence of disease progression or unacceptable toxicity. Extended-release Onapristone: Given PO Anastrozole: Given PO Quality-of-Life Assessment: Ancillary studies Questionnaire Administration: Ancillary studies Estrogen Receptor Positive (Positive Estrogen Receptor; ESR Positive; ESR1 Positive; ER Positive; Estrogen Receptor Alpha Positive): Immunohistochemistry (IHC):Integral : Tissue Progesterone Receptor Positive ( PGR Positive; PR Positive): Immunohistochemistry (IHC)
Gastrointestinal disorders Abdominal Pain	28.6% 4/14 • Number of events 4 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Investigations Alanine aminotransferase increased	78.6% 11/14 • Number of events 16 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Investigations Alkaline phosphatase increased	42.9% 6/14 • Number of events 11 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Skin and subcutaneous tissue disorders Alopecia	14.3% 2/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Blood and lymphatic system disorders Anemia	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Psychiatric disorders Anxiety	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Musculoskeletal and connective tissue disorders Arthralgia	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Investigations Aspartate aminotransferase increased	21.4% 3/14 • Number of events 3 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Musculoskeletal and connective tissue disorders Back pain	7.1% 1/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Reproductive system and breast disorders Vaginal hemorrhage	7.1% 1/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Immune system disorders Autoimmune disorder	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Investigations Cholesterol high	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Gastrointestinal disorders Colitis	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Psychiatric disorders Confusion	14.3% 2/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Gastrointestinal disorders Constipation	14.3% 2/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Infections and infestations Infections and infestations - Other, specify	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Respiratory, thoracic and mediastinal disorders Cough	21.4% 3/14 • Number of events 3 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Psychiatric disorders Depression	14.3% 2/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Gastrointestinal disorders Diarrhea	21.4% 3/14 • Number of events 3 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Nervous system disorders Dizziness	14.3% 2/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Nervous system disorders Dysarthria	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Respiratory, thoracic and mediastinal disorders Dyspnea	21.4% 3/14 • Number of events 3 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Gastrointestinal disorders Dysphagia	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
General disorders Edema limbs	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
General disorders Localized edema	14.3% 2/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Eye disorders Eye disorders - Other, specify	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Injury, poisoning and procedural complications Fall	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
General disorders Fatigue	35.7% 5/14 • Number of events 5 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Vascular disorders Flushing	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
General disorders Gair disturbance	14.3% 2/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Nervous system disorders Headache	14.3% 2/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Vascular disorders Hot flashes	50.0% 7/14 • Number of events 7 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Metabolism and nutrition disorders Hyperglycemina	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Metabolism and nutrition disorders Hyperkalemia	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Endocrine disorders Hypothyroidism	14.3% 2/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Psychiatric disorders Insomnia	14.3% 2/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Musculoskeletal and connective tissue disorders Pain in extremity	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Musculoskeletal and connective tissue disorders Muscle cramp	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Musculoskeletal and connective tissue disorders Neck pain	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Musculoskeletal and connective tissue disorders Myalgia	14.3% 2/14 • Number of events 3 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Gastrointestinal disorders Nausea	42.9% 6/14 • Number of events 7 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Musculoskeletal and connective tissue disorders Osteoporosis	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Reproductive system and breast disorders Pelvic pain	14.3% 2/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Nervous system disorders Paresthesia	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Investigations Platelet count decreased	14.3% 2/14 • Number of events 6 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Skin and subcutaneous tissue disorders Rash acneform	14.3% 2/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Skin and subcutaneous tissue disorders Rash maculo-papular	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Renal and urinary disorders Renal and urinary disorders - Other, specify	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Vascular disorders Thromboembolic event	14.3% 2/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Infections and infestations Urinary tract infection	21.4% 3/14 • Number of events 6 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Renal and urinary disorders Urine discoloration	7.1% 1/14 • Number of events 2 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Gastrointestinal disorders Vomiting	21.4% 3/14 • Number of events 3 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Hepatobiliary disorders Elevated liver enzymes	28.6% 4/14 • Number of events 15 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Gastrointestinal disorders Small Bowel Obstruction	7.1% 1/14 • Number of events 1 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.
Metabolism and nutrition disorders Weight loss	21.4% 3/14 • Number of events 3 • Adverse events and serious adverse events will be followed starting after first administration of study medication until 28 days after the last administration of study medication. Ongoing AEs at the time of end of treatment should be followed until study drug-related toxicities resolve, return to baseline, or are deemed irreversible, whichever is longer, up to 36 months. At each study visit, the investigator (or designee) will inquire about the occurrence of AE/SAEs since the last visit.

Additional Information

Tommy Buchanan, MD

Thomas Jefferson University

Phone: 215-481-4000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place