Trial Outcomes & Findings for A Phase 2 Study of Nab-sirolimus (ABI-009) in Patients With Advanced Malignant PEComa (NCT NCT02494570)

NCT ID: NCT02494570

Last Updated: 2023-04-07

Results Overview

Best ORR was assessed by Independent Radiology Review using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 Criteria. Best overall response was the best response recorded from the start of the study treatment until the end of treatment taking into account the requirement for confirmation. Per RECIST v1.1, best overall response assignment depended on the findings of both target and non-target disease and also took into consideration the appearance of new lesions. Overall response rate was defined as the percentage of patients who achieve a confirmed PR or CR per RECIST 1.1. At each timepoint, objective tumor response for target lesions were assessed as such: Complete Response (CR), disappearance of all target lesions, and pathological lymph nodes must have a reduction \<10 mm; Partial Response (PR), ≥30% decrease in the sum of the longest diameter of target lesions.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 34 participants
• Primary outcome timeframe: through study completion (up to 72 months)
• Results posted on: 2023-04-07

Participant Flow

Participant milestones

Measure	Experimental: Nab-Sirolimus Patients with malignant PEComa were treated with 100 mg/m2 nab-sirolimus (30 mins IV infusion) on Days 1 and 8 in a 21-day cycle.
Overall Study STARTED	34
Overall Study COMPLETED	34
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Patients with metastatic lesions n = 29.

Baseline characteristics by cohort

Measure	Experimental: Nab-Sirolimus n=34 Participants Patients with malignant PEComa were treated with 100 mg/m2 nab-sirolimus (30 mins IV infusion) on Days 1 and 8 in a 21-day cycle.
Age, Categorical <=18 years	0 Participants n=34 Participants
Age, Categorical Between 18 and 65 years	19 Participants n=34 Participants
Age, Categorical >=65 years	15 Participants n=34 Participants
Age, Continuous	60 years n=34 Participants
Sex: Female, Male Female	28 Participants n=34 Participants
Sex: Female, Male Male	6 Participants n=34 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=34 Participants
Race (NIH/OMB) Asian	3 Participants n=34 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=34 Participants
Race (NIH/OMB) Black or African American	3 Participants n=34 Participants
Race (NIH/OMB) White	24 Participants n=34 Participants
Race (NIH/OMB) More than one race	0 Participants n=34 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=34 Participants
Region of Enrollment United States	34 Participants n=34 Participants
ECOG Performance Score 0 (Fully active, able to carry on all pre-disease performance without restriction)	26 Participants n=34 Participants
ECOG Performance Score 1 (Restricted in physically strenuous activity but ambulatory and able to carry out light work)	8 Participants n=34 Participants
Metastatic PEComa	29 Participants n=34 Participants
Locally Advanced, Inoperable PEComa	5 Participants n=34 Participants
Prior Systemic Therapy for Advanced PEComa	4 Participants n=34 Participants
Primary Tumor Location of PEComa Uterus	8 Participants n=34 Participants
Primary Tumor Location of PEComa Pelvis, extrauterine	6 Participants n=34 Participants
Primary Tumor Location of PEComa Retroperitoneum	6 Participants n=34 Participants
Primary Tumor Location of PEComa Lung	4 Participants n=34 Participants
Primary Tumor Location of PEComa Kidney	4 Participants n=34 Participants
Primary Tumor Location of PEComa Aorta	1 Participants n=34 Participants
Primary Tumor Location of PEComa Brain	1 Participants n=34 Participants
Primary Tumor Location of PEComa Liver	1 Participants n=34 Participants
Primary Tumor Location of PEComa Muscle	1 Participants n=34 Participants
Primary Tumor Location of PEComa Ovary	1 Participants n=34 Participants
Primary Tumor Location of PEComa Small Bowel	1 Participants n=34 Participants
Number of Metastatic Sites 1 metastatic site (best)	11 Participants n=29 Participants • Patients with metastatic lesions n = 29.
Number of Metastatic Sites 2 metastatic sites	9 Participants n=29 Participants • Patients with metastatic lesions n = 29.
Number of Metastatic Sites 3 metastatic sites	7 Participants n=29 Participants • Patients with metastatic lesions n = 29.
Number of Metastatic Sites >3 metastatic sites (worst)	2 Participants n=29 Participants • Patients with metastatic lesions n = 29.

PRIMARY outcome

Timeframe: through study completion (up to 72 months)

Population: Efficacy Evaluable Patient Population

Best ORR was assessed by Independent Radiology Review using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 Criteria. Best overall response was the best response recorded from the start of the study treatment until the end of treatment taking into account the requirement for confirmation.

Per RECIST v1.1, best overall response assignment depended on the findings of both target and non-target disease and also took into consideration the appearance of new lesions. Overall response rate was defined as the percentage of patients who achieve a confirmed PR or CR per RECIST 1.1. At each timepoint, objective tumor response for target lesions were assessed as such: Complete Response (CR), disappearance of all target lesions, and pathological lymph nodes must have a reduction <10 mm; Partial Response (PR), ≥30% decrease in the sum of the longest diameter of target lesions.

Outcome measures

Measure	Nab-Sirolimus n=31 Participants Patients with malignant PEComa were treated with 100 mg/m2 nab-sirolimus (30 mins IV infusion) on Days 1 and 8 in a 21-day cycle.
Objective Overall Response Rate (ORR) Best Overall Confirmed Complete Response	2 Participants
Objective Overall Response Rate (ORR) Best Overall Confirmed Partial Response	10 Participants
Objective Overall Response Rate (ORR) Best Overall Stable Disease	16 Participants
Objective Overall Response Rate (ORR) Best Overall Progressive Disease	3 Participants

SECONDARY outcome

Timeframe: From Initial response until tumor progression, through study completion (up to 72 months)

Population: Patients with a confirmed partial or complete response

The time from the start of CR or PR to the first date of documented PD or death.

Outcome measures

Measure	Nab-Sirolimus n=12 Participants Patients with malignant PEComa were treated with 100 mg/m2 nab-sirolimus (30 mins IV infusion) on Days 1 and 8 in a 21-day cycle.
Duration of Response	39.7 months Interval 6.5 to Upper limit was not reached due to insufficient number of participants with events

SECONDARY outcome

Timeframe: 6 months

Population: Efficacy Evaluable Patient Population

The time from the first dose date to the first observation of a disease progression or death due to any cause by 6 mo. Disease progression was assessed radiologically per RECIST v1.1, and was defined as ≥20% increase in the sum of diameters of target lesions, and absolute increase of ≥5 mm. PFS rate at 6 months was summarized using Kaplan-Meier methods (percentage rounded up to 1 digit decimal).

Outcome measures

Measure	Nab-Sirolimus n=31 Participants Patients with malignant PEComa were treated with 100 mg/m2 nab-sirolimus (30 mins IV infusion) on Days 1 and 8 in a 21-day cycle.
Progression-free Survival Rate at 6 Months	69.5 percentage of participants Interval 47.6 to 83.7

SECONDARY outcome

Timeframe: from start of treatment to first documented disease progression, through study completion (up to 72 months)

Population: Efficacy Evaluable Patient Population

The time from the first dose date to the first observation of a disease progression or death due to any cause.

Outcome measures

Measure	Nab-Sirolimus n=31 Participants Patients with malignant PEComa were treated with 100 mg/m2 nab-sirolimus (30 mins IV infusion) on Days 1 and 8 in a 21-day cycle.
Progression-free Survival (Median)	10.6 months Interval 5.5 to 53.1

SECONDARY outcome

Timeframe: From start of treatment to date of death (of any cause), through study completion (up to 72 months)

Population: Treated Patient Population

Defined as teh time from first dose date to the date of death due to any cause

Outcome measures

Measure	Nab-Sirolimus n=34 Participants Patients with malignant PEComa were treated with 100 mg/m2 nab-sirolimus (30 mins IV infusion) on Days 1 and 8 in a 21-day cycle.
Overall Survival	53.1 months Interval 22.2 to Upper limit was not reached due to insufficient number of participants with events

POST_HOC outcome

Timeframe: through study completion (up to 72 months)

The percentage of patients who achieve confirmed CR or PR or SD ≥12 weeks following study treatment initiation.

Outcome measures

Measure	Nab-Sirolimus n=31 Participants Patients with malignant PEComa were treated with 100 mg/m2 nab-sirolimus (30 mins IV infusion) on Days 1 and 8 in a 21-day cycle.
Disease Control Rate	10 Participants

Adverse Events

Experimental: Nab-Sirolimus

Serious events: 16 serious events

Other events: 34 other events

Deaths: 2 deaths

Serious adverse events

Measure	Experimental: Nab-Sirolimus n=34 participants at risk Patients with malignant PEComa were treated with 100 mg/m2 nab-sirolimus (30 mins IV infusion) on Days 1 and 8 in a 21-day cycle.
Blood and lymphatic system disorders Pancytopenia	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Cardiac disorders Acute coronary syndrome	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Cardiac disorders Bradycardia	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Gastrointestinal disorders Abdominal pain	5.9% 2/34 • Number of events 2 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Gastrointestinal disorders Diarrhoea	5.9% 2/34 • Number of events 2 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Gastrointestinal disorders Enteritis	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Gastrointestinal disorders Intestinal Obstruction	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Gastrointestinal disorders Upper gastrointestinal haemorrhage	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
General disorders Non-cardiac chest pain	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
General disorders Oedema	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Blood and lymphatic system disorders Pyrexia	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Infections and infestations Clostridium difficile infection	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Infections and infestations Enteritis infectious	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Infections and infestations Gangrene	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Infections and infestations Gastroenteritis viral	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Infections and infestations Pneumonia	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Infections and infestations Upper respiratory tract infection	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Injury, poisoning and procedural complications Foot fracture	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Injury, poisoning and procedural complications Fall	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Injury, poisoning and procedural complications Post procedural haemorrhage	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Metabolism and nutrition disorders Dehydration	5.9% 2/34 • Number of events 2 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Metabolism and nutrition disorders Hypoglycaemia	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Musculoskeletal and connective tissue disorders Arthralgia	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Musculoskeletal and connective tissue disorders Musculoskeletal chest pain	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Renal and urinary disorders Acute kidney injury	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Respiratory, thoracic and mediastinal disorders Haemothorax	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Vascular disorders Hypotension	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Nervous system disorders Syncope	5.9% 2/34 • Number of events 2 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Gastrointestinal disorders Abdominal hernia	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Gastrointestinal disorders Small intestinal obstruction	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Gastrointestinal disorders Vomiting	2.9% 1/34 • Number of events 1 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.

Other adverse events

Measure	Experimental: Nab-Sirolimus n=34 participants at risk Patients with malignant PEComa were treated with 100 mg/m2 nab-sirolimus (30 mins IV infusion) on Days 1 and 8 in a 21-day cycle.
Gastrointestinal disorders Stomatitis	82.4% 28/34 • Number of events 69 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Gastrointestinal disorders Nausea	50.0% 17/34 • Number of events 40 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Gastrointestinal disorders Diarrhoea	47.1% 16/34 • Number of events 39 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Gastrointestinal disorders Abdominal Pain	44.1% 15/34 • Number of events 33 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Gastrointestinal disorders Vomiting	38.2% 13/34 • Number of events 31 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
General disorders Fatigue	73.5% 25/34 • Number of events 47 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
General disorders Edema	55.9% 19/34 • Number of events 32 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Metabolism and nutrition disorders Hyperglycemia	47.1% 16/34 • Number of events 45 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Metabolism and nutrition disorders Decreased appetite	44.1% 15/34 • Number of events 21 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Metabolism and nutrition disorders Hypokalemia	44.1% 15/34 • Number of events 33 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Metabolism and nutrition disorders Hypertriglyceridemia	38.2% 13/34 • Number of events 25 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Metabolism and nutrition disorders Hypercholesterolemia	35.3% 12/34 • Number of events 21 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Blood and lymphatic system disorders Anemia	61.8% 21/34 • Number of events 110 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Blood and lymphatic system disorders Thrombocytopenia	35.3% 12/34 • Number of events 37 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Blood and lymphatic system disorders Hemorrhage	32.4% 11/34 • Number of events 28 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Infections and infestations Infections	58.8% 20/34 • Number of events 86 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Nervous system disorders Taste Disorder	35.3% 12/34 • Number of events 25 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Nervous system disorders Headache	35.3% 12/34 • Number of events 24 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	52.9% 18/34 • Number of events 37 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Respiratory, thoracic and mediastinal disorders Cough	44.1% 15/34 • Number of events 21 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Investigations Weight Decreased	50.0% 17/34 • Number of events 45 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Skin and subcutaneous tissue disorders Rash	67.6% 23/34 • Number of events 55 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Metabolism and nutrition disorders Hypomagnesemia	29.4% 10/34 • Number of events 18 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Investigations ALT	26.5% 9/34 • Number of events 20 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Investigations AST	26.5% 9/34 • Number of events 18 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Blood and lymphatic system disorders Leukopenia	26.5% 9/34 • Number of events 43 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Nervous system disorders Peripheral neuropathy	23.5% 8/34 • Number of events 8 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Nervous system disorders Dizziness	20.6% 7/34 • Number of events 11 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Respiratory, thoracic and mediastinal disorders Non-infectious pneumonitis	20.6% 7/34 • Number of events 22 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
General disorders Pain	20.6% 7/34 • Number of events 10 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Blood and lymphatic system disorders Lymphopenia	17.6% 6/34 • Number of events 21 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Metabolism and nutrition disorders Dehydration	14.7% 5/34 • Number of events 8 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Respiratory, thoracic and mediastinal disorders Dyspnea	14.7% 5/34 • Number of events 10 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Blood and lymphatic system disorders Neutropenia	14.7% 5/34 • Number of events 14 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
General disorders Fracture	11.8% 4/34 • Number of events 4 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Cardiac disorders Tachycardia	11.8% 4/34 • Number of events 5 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
General disorders Dental pain	8.8% 3/34 • Number of events 5 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Skin and subcutaneous tissue disorders Dermatitis	5.9% 2/34 • Number of events 4 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Ear and labyrinth disorders Ear pain	5.9% 2/34 • Number of events 4 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Musculoskeletal and connective tissue disorders Constipation	23.5% 8/34 • Number of events 11 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Gastrointestinal disorders Hemorrhoids	11.8% 4/34 • Number of events 4 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Investigations Lipase increased	29.4% 10/34 • Number of events 20 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Investigations Amylase increased	26.5% 9/34 • Number of events 40 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Investigations Creatinine increased	26.5% 9/34 • Number of events 20 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Skin and subcutaneous tissue disorders Alopecia	26.5% 9/34 • Number of events 11 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.
Nervous system disorders Dysgeusia	32.4% 11/34 • Number of events 21 • Safety and tolerability were monitored through continuous reporting of treatment-emergent and treatment-related adverse events (AEs) and serious AEs throughout the study, from the time the patient signed informed consent until 28 days after the last dose of study drug, up to 72 months.

Additional Information

Medical Information

Aadi Bioscience

Phone: 1-888-246-2234

Email: MedInfo@aadibio.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place