Trial Outcomes & Findings for A Study of the Efficacy and Safety of Pexidartinib in Adult Subjects With TGCT (NCT NCT04488822)

Last Updated: 2025-09-09

Results Overview

For the assessment of tumor response, participants were classified into the best of the following tumor response categories based on Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1) by centrally reviewed MRI scan. Complete response (CR), disappearance of all target lesions and normalization of tumor marker level; partial response (PR), at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters; and overall response rate (ORR), defined as CR+PR, are presented.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE3

Target enrollment

40 participants

Primary outcome timeframe

At Week 25 postdose

Results posted on

2025-09-09

Participant Flow

A total of 40 participants who met all inclusion criteria and no exclusion criteria were enrolled and treated at clinic centers in China and Taiwan. Primary results reported are from baseline up to data cut-off date of 27 Oct 2021. The results presented are based on primary analysis. Data collection is still on-going and additional results will be provided after study completion.

Participant milestones

Participant milestones
Measure	Pexidartinib Participants who received pexidartinib 400mg twice daily (BID) for a total daily dose of 800 mg (2 capsules in the morning and 2 capsules in the evening).
Overall Study STARTED	40
Overall Study COMPLETED	31
Overall Study NOT COMPLETED	9

Reasons for withdrawal

Reasons for withdrawal
Measure	Pexidartinib Participants who received pexidartinib 400mg twice daily (BID) for a total daily dose of 800 mg (2 capsules in the morning and 2 capsules in the evening).
Overall Study Adverse Event	8
Overall Study Withdrawal by Subject	1

Baseline Characteristics

A Study of the Efficacy and Safety of Pexidartinib in Adult Subjects With TGCT

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Pexidartinib n=40 Participants Participants who received pexidartinib 400mg twice daily (BID) for a total daily dose of 800 mg (2 capsules in the morning and 2 capsules in the evening).
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	39 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Age, Continuous	40.9 Years STANDARD_DEVIATION 12.05 • n=5 Participants
Sex: Female, Male Female	27 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	40 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment China	31 participants n=5 Participants
Region of Enrollment Taiwan	9 participants n=5 Participants

PRIMARY outcome

Timeframe: At Week 25 postdose

Population: ORR based on RECIST 1.1 was assessed in the Full Analysis Set.

Outcome measures

Outcome measures
Measure	Pexidartinib n=40 Participants Participants who received pexidartinib 400mg twice daily (BID) for a total daily dose of 800 mg (2 capsules in the morning and 2 capsules in the evening).
Overall Response Rate (ORR) of Pexidartinib Based on RECIST 1.1 of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25 CR	0 Participants
Overall Response Rate (ORR) of Pexidartinib Based on RECIST 1.1 of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25 ORR	9 Participants
Overall Response Rate (ORR) of Pexidartinib Based on RECIST 1.1 of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25 PR	9 Participants

SECONDARY outcome

Timeframe: At Week 25 postdose

Population: ORR based on TVS was assessed in the Full Analysis Set.

For the assessment of tumor response, participants were classified into the best of the following tumor response categories based on Tumor Volume Score (TVS) by centrally reviewed MRI scan. Tumor Volume Score (TVS) is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor. Complete response (CR), disappearance of lesion; partial response (PR), at least 50% decrease in volume score relative to baseline; overall response rate (ORR), defined as CR+PR, are presented.

Outcome measures

Outcome measures
Measure	Pexidartinib n=40 Participants Participants who received pexidartinib 400mg twice daily (BID) for a total daily dose of 800 mg (2 capsules in the morning and 2 capsules in the evening).
Overall Response Rate (ORR) of Pexidartinib Based on TVS of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25 CR	0 Participants
Overall Response Rate (ORR) of Pexidartinib Based on TVS of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25 PR	19 Participants
Overall Response Rate (ORR) of Pexidartinib Based on TVS of Asian Participants With Symptomatic Tenosynovial Giant Cell Tumor (TGCT) at Week 25 ORR	19 Participants

SECONDARY outcome

Timeframe: At Week 25 postdose

Population: Mean Percent Change From Baseline for Range of Motion (ROM) was assessed in Full Analysis Set; however, participant assessments falling outside the visit window were excluded from the change from baseline analysis.

Range of motion (ROM) of the joint was assessed by a qualified, independent, and blinded or third-party assessors at the clinical site. The relative ROM was calculated (expressed in percent) as follows: Relative ROM = 100 x (absolute ROM measured)/(reference ROM standard). The change from baseline in relative ROM at Week 25 was calculated as the difference in relative ROM at Week 25 visit minus relative ROM at Screening visit.

Outcome measures

Outcome measures
Measure	Pexidartinib n=31 Participants Participants who received pexidartinib 400mg twice daily (BID) for a total daily dose of 800 mg (2 capsules in the morning and 2 capsules in the evening).
Mean Percent Change From Baseline for Range of Motion (ROM) Score in Participants Receiving Pexidartinib	23.05 Percent Change Standard Deviation 3.207

SECONDARY outcome

Timeframe: At Week 25 postdose

Population: Mean Change From PROMIS was assessed in the Full Analysis Set; however, participant assessments falling outside the visit window were excluded from the change from baseline analysis.

The Patient-reported Outcomes Measurement Information System (PROMIS) physical function scale was used to assess the physical function of the upper and lower limbs. The scale ranges from 1 defined as 'unable to do' or 'cannot do' to 5 defined as 'without any difficulty' or 'not at all', where higher scores represent better outcomes.

Outcome measures

Outcome measures
Measure	Pexidartinib n=31 Participants Participants who received pexidartinib 400mg twice daily (BID) for a total daily dose of 800 mg (2 capsules in the morning and 2 capsules in the evening).
Mean Change From Baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score in Participants Receiving Pexidartinib	0.46 Score on a scale Standard Deviation 1.384

SECONDARY outcome

Timeframe: From baseline until disease progression or death (whichever occurs first), up to approximately 29 months

Best overall response (CR or PR) of pexidartinib based on RECIST 1.1 and TVS are summarized. Tumor categories for RECIST v1.1 were as follows: complete response (CR), disappearance of all target lesions; partial response (PR), at least a 30% decrease in the sum of diameters of target lesions; stable disease (SD), neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease; progressive disease (PD), at least a 20% increase in the sum of diameters of target lesions; and not evaluable (NE). Tumor categories for TVS were as follows: complete response (CR), disappearance of all target lesions; partial response (PR), at least a 50% decrease in volume score relative to baseline; stable disease (SD), does not meet any of the prior criteria based on score during study; progressive disease (PD), at least a 30% increase in in volume relative to lowest score during the study whether at baseline or some other visit; and not evaluable (NE).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Time from the date of first documented response until documented disease progression or death from any cause (whichever occurs first), up to approximately 29 months

Duration of response (DOR) is defined as the date of the first recorded response to the first date of documented disease progression.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to approximately 29 months

A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Week 3: Cycle 1 Day 1 at pre-dose, 0.5h, 1h, 2h, 4h, and 6h post-dose (each cycle is 28 days)

Population: AUC(0-6h) was assessed in the Pharmacokinetic Analysis Set.

AUC(0-6h) was calculated using standard non-compartmental analysis. AUC(0-6h) for Pexidartinib and primary metabolite, ZAAD-1006a, are presented.

Outcome measures

Outcome measures
Measure	Pexidartinib n=7 Participants Participants who received pexidartinib 400mg twice daily (BID) for a total daily dose of 800 mg (2 capsules in the morning and 2 capsules in the evening).
Area Under the Plasma Concentration Time Curve From Time 0 to 6 Hours AUC(0-6h) in Participants Receiving Pexidartinib Pexidartinib	51500 ng*h/mL Standard Deviation 28.5
Area Under the Plasma Concentration Time Curve From Time 0 to 6 Hours AUC(0-6h) in Participants Receiving Pexidartinib ZAAD-1006a	108000 ng*h/mL Standard Deviation 52.1

SECONDARY outcome

Timeframe: Week 3: Cycle 1 Day 1 at pre-dose, 0.5h, 1h, 2h, 4h, and 6h post-dose (each cycle is 28 days)

Population: Cmax was assessed in the Pharmacokinetic Analysis Set.

Cmax was calculated using standard non-compartmental analysis. Cmax for Pexidartinib and primary metabolite, ZAAD-1006a, are presented.

Outcome measures

Outcome measures
Measure	Pexidartinib n=7 Participants Participants who received pexidartinib 400mg twice daily (BID) for a total daily dose of 800 mg (2 capsules in the morning and 2 capsules in the evening).
Maximum Plasma Concentration (Cmax) in Participants Receiving Pexidartinib Pexidartinib	12400 ng/mL Standard Deviation 32.2
Maximum Plasma Concentration (Cmax) in Participants Receiving Pexidartinib ZAAD-1006a	20400 ng/mL Standard Deviation 46.3

SECONDARY outcome

Timeframe: Week 3: Cycle 1 Day 1 at pre-dose, 0.5h, 1h, 2h, 4h, and 6h post-dose (each cycle is 28 days)

Population: Tmax was assessed in the Pharmacokinetic Analysis Set.

Tmax was calculated using standard non-compartmental analysis. Tmax for Pexidartinib and primary metabolite, ZAAD-1006a, are presented.

Outcome measures

Outcome measures
Measure	Pexidartinib n=7 Participants Participants who received pexidartinib 400mg twice daily (BID) for a total daily dose of 800 mg (2 capsules in the morning and 2 capsules in the evening).
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Participants Receiving Pexidartinib Pexidartinib	2.08 hours Interval 0.87 to 2.15
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Participants Receiving Pexidartinib ZAAD-1006a	2.12 hours Interval 1.8 to 5.9

Adverse Events

Pexidartinib

Serious events: 6 serious events

Other events: 40 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Pexidartinib n=40 participants at risk Participants who received pexidartinib 400mg twice daily (BID) for a total daily dose of 800 mg (2 capsules in the morning and 2 capsules in the evening).
Hepatobiliary disorders Hepatic function abnormal	7.5% 3/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Musculoskeletal and connective tissue disorders Fistula inflammation	2.5% 1/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Musculoskeletal and connective tissue disorders Osteonecrosis	2.5% 1/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Epithelioid sarcoma	2.5% 1/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Respiratory, thoracic and mediastinal disorders Pneumothorax	2.5% 1/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).

Other adverse events

Other adverse events
Measure	Pexidartinib n=40 participants at risk Participants who received pexidartinib 400mg twice daily (BID) for a total daily dose of 800 mg (2 capsules in the morning and 2 capsules in the evening).
Skin and subcutaneous tissue disorders Hair color changes	77.5% 31/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Skin and subcutaneous tissue disorders Pruritus	47.5% 19/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Skin and subcutaneous tissue disorders Rash	42.5% 17/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Skin and subcutaneous tissue disorders Skin discoloration	17.5% 7/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Skin and subcutaneous tissue disorders Drug eruption	12.5% 5/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Skin and subcutaneous tissue disorders Hair texture abnormal	12.5% 5/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Skin and subcutaneous tissue disorders Alopecia	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Skin and subcutaneous tissue disorders Urticaria	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Alanine aminotransferase increased	60.0% 24/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Aspartate aminotransferase increased	57.5% 23/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Blood lactate dehydrogenase increased	47.5% 19/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Gamma-glutamyltransferase increased	37.5% 15/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations White blood cell count decreased	35.0% 14/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Blood alkaline phosphatase increased	30.0% 12/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Neutrophil count decreased	27.5% 11/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Blood creatine phosphokinase increased	20.0% 8/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Bilirubin conjugated increased	15.0% 6/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Blood bilirubin increased	15.0% 6/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Lymphocyte count decreased	12.5% 5/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Blood cholinesterase increased	7.5% 3/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Monocyte count decreased	7.5% 3/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Blood cholesterol increased	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Blood triglycerides increased	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations C-reactive protein increased	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations High density lipoprotein increased	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Low density lipoprotein decreased	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Total bile acids increased	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Weight decreased	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
General disorders Face oedema	42.5% 17/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Investigations Fatigue	30.0% 12/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
General disorders Oedema peripheral	10.0% 4/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
General disorders Swelling face	10.0% 4/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
General disorders Asthenia	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
General disorders Pyrexia	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Gastrointestinal disorders Nausea	15.0% 6/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Gastrointestinal disorders Diarrhoea	12.5% 5/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Gastrointestinal disorders Dry mouth	12.5% 5/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Gastrointestinal disorders Constipation	10.0% 4/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Gastrointestinal disorders Abdominal distension	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Gastrointestinal disorders Gastrointestinal disorder	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Nervous system disorders Dizziness	10.0% 4/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Nervous system disorders Dysgeusia	10.0% 4/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Nervous system disorders Headache	7.5% 3/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Nervous system disorders Hypoaesthesia	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Nervous system disorders Memory impairment	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Nervous system disorders Taste disorder	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Eye disorders Periorbital oedema	22.5% 9/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Eye disorders Eyelid oedema	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Metabolism and nutrition disorders Decreased appetite	10.0% 4/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Metabolism and nutrition disorders Hypokalaemia	10.0% 4/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Metabolism and nutrition disorders Hyperlipidaemia	7.5% 3/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Metabolism and nutrition disorders Enzyme abnormality	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Metabolism and nutrition disorders Hypophosphataemia	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Blood and lymphatic system disorders Anaemia	20.0% 8/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Blood and lymphatic system disorders Leukopenia	7.5% 3/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Infections and infestations Nasopharyngitis	12.5% 5/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Musculoskeletal and connective tissue disorders Arthralgia	10.0% 4/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Musculoskeletal and connective tissue disorders Pain in extremity	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Respiratory, thoracic and mediastinal disorders Cough	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	5.0% 2/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Vascular disorders Hypertension	12.5% 5/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).
Hepatobiliary disorders Hepatic function abnormal	7.5% 3/40 • Adverse events (AE) were collected from the date of signing the informed consent form up post-treatment visit (28 ± 7 days after the last dose of study drug), up 13 months. A Treatment-emergent adverse event (TEAE) is defined as an AE that occurs, having been absent before the first dose of study drug, or has worsened in severity or seriousness after the initiating the study drug until post-treatment visit (28 ± 7 days after the last dose of study drug).

Additional Information

Clinical Director

Daiichi Sankyo, Inc.

Phone: 908-992-6400

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place