Trial Outcomes & Findings for Nirogacestat in Ovarian Granulosa Cell Tumors (NCT NCT05348356)

Last Updated: 2026-08-25

Results Overview

The percentage of participants with complete response (CR) + partial response (PR) assessed using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 criteria.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

53 participants

Primary outcome timeframe

2.5 years

Results posted on

2026-08-25

Participant Flow

Participant milestones

Participant milestones
Measure	Nirogacestat Open-Label Nirogacestat 150 mg tablet by mouth, twice daily.
Overall Study STARTED	53
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	52

Reasons for withdrawal

Reasons for withdrawal
Measure	Nirogacestat Open-Label Nirogacestat 150 mg tablet by mouth, twice daily.
Overall Study Death	1
Overall Study Lack of Efficacy	38
Overall Study Withdrawal by Subject	4
Overall Study Physician Decision	8
Overall Study Clinical Progression	1

Baseline Characteristics

Nirogacestat in Ovarian Granulosa Cell Tumors

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Nirogacestat Open-Label n=53 Participants Nirogacestat 150 mg tablet by mouth, twice daily.
Age, Continuous	55.1 years STANDARD_DEVIATION 12.85 • n=31 Participants
Age, Customized <45 years	13 Participants n=31 Participants
Age, Customized >=45 years	40 Participants n=31 Participants
Sex: Female, Male Female	53 Participants n=31 Participants
Sex: Female, Male Male	0 Participants n=31 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants n=31 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	43 Participants n=31 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	4 Participants n=31 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=31 Participants
Race (NIH/OMB) Asian	3 Participants n=31 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=31 Participants
Race (NIH/OMB) Black or African American	4 Participants n=31 Participants
Race (NIH/OMB) White	37 Participants n=31 Participants
Race (NIH/OMB) More than one race	2 Participants n=31 Participants
Race (NIH/OMB) Unknown or Not Reported	7 Participants n=31 Participants
Region of Enrollment United States	50 Participants n=31 Participants
Region of Enrollment Poland	3 Participants n=31 Participants
Baseline Weight	82.48 kilogram STANDARD_DEVIATION 19.672 • n=31 Participants
Baseline Height	1.632 meter STANDARD_DEVIATION 0.0611 • n=31 Participants

PRIMARY outcome

Timeframe: 2.5 years

Population: Full Analysis Set which consisted of all participant who received at least 1 dose of nirogacestat.

The percentage of participants with complete response (CR) + partial response (PR) assessed using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 criteria.

Outcome measures

Outcome measures
Measure	Nirogacestat Open-Label n=53 Participants Nirogacestat 150 mg tablet by mouth, twice daily.
Objective Response Rate (ORR)	0 percentage of participants

SECONDARY outcome

Timeframe: First day of cycle 7 (approximately 6 months after the first dose of study treatment). Each cycle is 28 days.

Population: Full Analysis Set which consisted of all participant who received at least 1 dose of nirogacestat.

The number of participants without progression according to RECIST v1.1 or death at 6 months.

Outcome measures

Outcome measures
Measure	Nirogacestat Open-Label n=53 Participants Nirogacestat 150 mg tablet by mouth, twice daily.
Progression Free Survival at 6 Months (PFS-6)	22 Participants

SECONDARY outcome

Timeframe: 2 years after first dose of study treatment

Population: Full Analysis Set which consisted of all participant who received at least 1 dose of nirogacestat.

The number of participants who have not died of any cause by Year 2.

Outcome measures

Outcome measures
Measure	Nirogacestat Open-Label n=53 Participants Nirogacestat 150 mg tablet by mouth, twice daily.
Overall Survival at Year 2	47 Participants

SECONDARY outcome

Timeframe: First day of every other cycle (each cycle is 28 days) for the first year, and then every 3 cycles thereafter until study completion (estimated to be an average of 2.5 years).

Population: The DOR was to be evaluated in participants with CR or PR. Since no participants had CR or PR, therefore, no participants were evaluable for DOR.

The time from response (Complete Response \[CR\] + Partial Response \[PR\] using RECIST v.1.) to disease progression and/or death, in participants with CR or PR.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline and at Cycle 2 Day 1 (cycle length is 28 days).

Population: Participants evaluable for outcome of change from baseline at Cycle 2 Day 1 in the FOSI score.

Participant reported ovarian cancer symptoms as measured by Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI). The FOSI is an 8-question evaluation scored on a Likert scale of 0-4 from not at all to very much. Metrics assessed include lack of energy, vomiting, pain, nausea, swelling in stomach area, concern condition will get worse, quality of life assessment and stomach cramps. Higher score means worse outcome. The responses to the statements except for statement 7 (I am content with the quality of my life right now) were reversed and the sum of non-missing responses is calculated. The FOSI score is the sum multiplied by 8, then divided by the number of responded statements. The FOSI score was missing if a participants had 5 or more missing values in the 8 individual symptom scores at a visit.

Outcome measures

Outcome measures
Measure	Nirogacestat Open-Label n=49 Participants Nirogacestat 150 mg tablet by mouth, twice daily.
Change From Baseline at Cycle 2 Day 1 in the Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI) Score.	-1.1 score on a scale Standard Deviation 3.86

SECONDARY outcome

Timeframe: Baseline and at Cycle 3 Day 1 (cycle length is 28 days).

Population: Participants evaluable for outcome of change from baseline at Cycle 3 Day 1 in the FOSI score.

Outcome measures

Outcome measures
Measure	Nirogacestat Open-Label n=36 Participants Nirogacestat 150 mg tablet by mouth, twice daily.
Change From Baseline at Cycle 3 Day 1 Treatment in the Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI) Score	-1.2 score on a scale Standard Deviation 4.00

SECONDARY outcome

Timeframe: Baseline and at Cycle 4 Day 1 (cycle length is 28 days).

Population: Participants evaluable for the outcome of change from baseline at Cycle 4 Day 1 in the FOSI score.

Outcome measures

Outcome measures
Measure	Nirogacestat Open-Label n=33 Participants Nirogacestat 150 mg tablet by mouth, twice daily.
Change From Baseline at Cycle 4 Day 1 in the Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI) Score	-1.5 score on a scale Standard Deviation 3.08

SECONDARY outcome

Timeframe: Baseline and at Cycle 5 Day 1 (cycle length is 28 days).

Population: Participants evaluable for the outcome of change from baseline at Cycle 5 Day 1 in the FOSI score.

Outcome measures

Outcome measures
Measure	Nirogacestat Open-Label n=25 Participants Nirogacestat 150 mg tablet by mouth, twice daily.
Change From Baseline at Cycle 5 Day 1 in the Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI) Score	-1.3 score on a scale Standard Deviation 3.36

SECONDARY outcome

Timeframe: Baseline and at end of treatment (estimated to be an average of 5.5 months [minimum of 0 month and maximum of 33 months]).

Population: Participants evaluable for the outcome of change from baseline in the FOSI score at end of treatment.

Outcome measures

Outcome measures
Measure	Nirogacestat Open-Label n=48 Participants Nirogacestat 150 mg tablet by mouth, twice daily.
Change From Baseline at End of Treatment in the Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI) Score	-2.2 score on a scale Standard Deviation 4.48

SECONDARY outcome

Timeframe: Baseline and at safety follow-up (up to a maximum of 34 months).

Population: Participants evaluable for the outcome of change from baseline in the FOSI score at safety follow-up.

Outcome measures

Outcome measures
Measure	Nirogacestat Open-Label n=40 Participants Nirogacestat 150 mg tablet by mouth, twice daily.
Change From Baseline at Safety Follow-up in the Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI) Score	-0.8 score on a scale Standard Deviation 3.75

Adverse Events

Nirogacestat Open-Label

Serious events: 12 serious events

Other events: 52 other events

Deaths: 7 deaths

Serious adverse events

Serious adverse events
Measure	Nirogacestat Open-Label n=53 participants at risk Nirogacestat 150 mg by mouth, twice daily Nirogacestat oral tablet: Nirogacestat tablet Nirogacestat: nirogacestat oral tablet
Gastrointestinal disorders Abdominal pain	7.5% 4/53 • Number of events 4 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Renal and urinary disorders Acute kidney injury	3.8% 2/53 • Number of events 2 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Cardiac disorders Acute myocardial infarction	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Cardiac disorders Angina pectoris	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Cardiac disorders Cardio-respiratory arrest	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Gastrointestinal disorders Colitis	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Metabolism and nutrition disorders Dehydration	3.8% 2/53 • Number of events 2 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Gastrointestinal disorders Diarrhoea	3.8% 2/53 • Number of events 2 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Respiratory, thoracic and mediastinal disorders Dyspnoea	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Investigations Electrocardiogram QT prolonged	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Nervous system disorders Encephalopathy	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Infections and infestations Gastroenteritis astroviral	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Skin and subcutaneous tissue disorders Hidradenitis	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Metabolism and nutrition disorders Hypokalaemia	3.8% 2/53 • Number of events 3 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Metabolism and nutrition disorders Hypomagnesaemia	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Metabolism and nutrition disorders Hypophagia	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Metabolism and nutrition disorders Hypophosphataemia	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
General disorders Influenza like illness	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Blood and lymphatic system disorders Leukocytosis	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Gastrointestinal disorders Nausea	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Gastrointestinal disorders Pancreatitis	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Respiratory, thoracic and mediastinal disorders Pulmonary oedema	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Infections and infestations Sepsis	1.9% 1/53 • Number of events 1 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Gastrointestinal disorders Small intestinal obstruction	3.8% 2/53 • Number of events 2 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.

Other adverse events

Other adverse events
Measure	Nirogacestat Open-Label n=53 participants at risk Nirogacestat 150 mg by mouth, twice daily Nirogacestat oral tablet: Nirogacestat tablet Nirogacestat: nirogacestat oral tablet
Investigations Blood alkaline phosphatase increased	5.7% 3/53 • Number of events 6 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Infections and infestations Bronchitis	5.7% 3/53 • Number of events 3 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Infections and infestations COVID-19	5.7% 3/53 • Number of events 3 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Skin and subcutaneous tissue disorders Dry skin	5.7% 3/53 • Number of events 3 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Nervous system disorders Dysgeusia	5.7% 3/53 • Number of events 3 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Musculoskeletal and connective tissue disorders Flank pain	5.7% 3/53 • Number of events 9 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Gastrointestinal disorders Gastrooesophageal reflux disease	5.7% 3/53 • Number of events 3 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Renal and urinary disorders Glycosuria	5.7% 3/53 • Number of events 5 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Metabolism and nutrition disorders Hypermagnesaemia	5.7% 3/53 • Number of events 7 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Metabolism and nutrition disorders Hypoalbuminaemia	5.7% 3/53 • Number of events 5 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Metabolism and nutrition disorders Hypomagnesaemia	5.7% 3/53 • Number of events 7 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
General disorders Influenza like illness	5.7% 3/53 • Number of events 3 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Musculoskeletal and connective tissue disorders Myalgia	5.7% 3/53 • Number of events 3 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Investigations Neutrophil count decreased	5.7% 3/53 • Number of events 4 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Eye disorders Photopsia	5.7% 3/53 • Number of events 3 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Skin and subcutaneous tissue disorders Rash	5.7% 3/53 • Number of events 4 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Cardiac disorders Sinus tachycardia	5.7% 3/53 • Number of events 3 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Infections and infestations Sinusitis	5.7% 3/53 • Number of events 3 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Infections and infestations Skin infection	5.7% 3/53 • Number of events 3 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Tumour pain	5.7% 3/53 • Number of events 3 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Investigations Weight decreased	5.7% 3/53 • Number of events 4 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Investigations Alanine aminotransferase increased	7.5% 4/53 • Number of events 4 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Gastrointestinal disorders Constipation	7.5% 4/53 • Number of events 5 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Skin and subcutaneous tissue disorders Dermatitis acneiform	7.5% 4/53 • Number of events 4 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Gastrointestinal disorders Dry mouth	7.5% 4/53 • Number of events 4 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Gastrointestinal disorders Dyspepsia	7.5% 4/53 • Number of events 7 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Respiratory, thoracic and mediastinal disorders Dyspnoea	7.5% 4/53 • Number of events 5 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Gastrointestinal disorders Flatulence	7.5% 4/53 • Number of events 4 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Infections and infestations Fungal infection	7.5% 4/53 • Number of events 4 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
General disorders Oedema peripheral	7.5% 4/53 • Number of events 4 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	7.5% 4/53 • Number of events 6 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Musculoskeletal and connective tissue disorders Pain in extremity	7.5% 4/53 • Number of events 4 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Renal and urinary disorders Proteinuria	7.5% 4/53 • Number of events 11 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Infections and infestations Urinary tract infection	7.5% 4/53 • Number of events 5 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Blood and lymphatic system disorders Anaemia	9.4% 5/53 • Number of events 11 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Psychiatric disorders Anxiety	9.4% 5/53 • Number of events 6 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Nervous system disorders Headache	9.4% 5/53 • Number of events 9 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Vascular disorders Hypertension	9.4% 5/53 • Number of events 5 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Metabolism and nutrition disorders Hypocalcaemia	9.4% 5/53 • Number of events 8 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Respiratory, thoracic and mediastinal disorders Nasal congestion	9.4% 5/53 • Number of events 6 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
General disorders Pyrexia	9.4% 5/53 • Number of events 5 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Gastrointestinal disorders Stomatitis	9.4% 5/53 • Number of events 6 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Gastrointestinal disorders Abdominal distension	11.3% 6/53 • Number of events 6 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Skin and subcutaneous tissue disorders Alopecia	11.3% 6/53 • Number of events 7 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Musculoskeletal and connective tissue disorders Back pain	11.3% 6/53 • Number of events 8 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Metabolism and nutrition disorders Decreased appetite	11.3% 6/53 • Number of events 11 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Metabolism and nutrition disorders Hyperglycaemia	11.3% 6/53 • Number of events 7 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Psychiatric disorders Insomnia	11.3% 6/53 • Number of events 8 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Skin and subcutaneous tissue disorders Pruritus	11.3% 6/53 • Number of events 6 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Musculoskeletal and connective tissue disorders Arthralgia	13.2% 7/53 • Number of events 11 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Investigations Aspartate aminotransferase increased	13.2% 7/53 • Number of events 7 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Respiratory, thoracic and mediastinal disorders Epistaxis	15.1% 8/53 • Number of events 8 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Metabolism and nutrition disorders Hypokalaemia	18.9% 10/53 • Number of events 25 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Gastrointestinal disorders Abdominal pain	20.8% 11/53 • Number of events 12 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Metabolism and nutrition disorders Hypophosphataemia	28.3% 15/53 • Number of events 32 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Respiratory, thoracic and mediastinal disorders Cough	32.1% 17/53 • Number of events 25 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Skin and subcutaneous tissue disorders Rash maculo-papular	32.1% 17/53 • Number of events 23 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Gastrointestinal disorders Vomiting	37.7% 20/53 • Number of events 28 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
General disorders Fatigue	39.6% 21/53 • Number of events 30 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Gastrointestinal disorders Nausea	56.6% 30/53 • Number of events 42 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.
Gastrointestinal disorders Diarrhoea	71.7% 38/53 • Number of events 68 • All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment (an average of 5.5 months [minimum of 0 month and maximum of 33 months] and up to 34 months). All SAEs and AEs were collected from the time of signing ICF until 30 days after the last dose of study treatment.

Additional Information

Communication Center

Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany

Phone: :+49-6151-72-5200

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place