Trial Outcomes & Findings for Study on Safety and Efficacy of NMS-01940153E in Adult Patients With Unresectable Hepatocellular Carcinoma (HCC) Previously Treated With Systemic Therapy (NCT NCT05630937)

Last Updated: 2025-06-19

Results Overview

All 12 Phase I treated patients were evaluable for DLT (Dose-Limiting Toxicity Evaluable Set) and included 6 patients treated at each of the two dose levels explored (i.e., 100 mg/m2/week and 135 mg/m2/week) who received at least 66% of the study drug in the first 28-day cycle of treatment and underwent a DLT assessment within the DLT window. Participants who experienced DLTs are presented.

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

31 participants

Primary outcome timeframe

Phase I: From screening to end of first 28-day cycle (17 months)

Results posted on

2025-06-19

Participant Flow

Participant milestones

Participant milestones
Measure	Phase 1 Participants: 100 mg/m2/Week All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E.	Phase I Participants: 135 mg/m2/Week All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E.	Phase II Participants: 100 mg/m2/Week All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E.	Enrolled But Not Treated Participants who were enrolled but not randomized and did not receive treatment.
Overall Study STARTED	6	6	18	1
Overall Study COMPLETED	5	5	9	0
Overall Study NOT COMPLETED	1	1	9	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Phase 1 Participants: 100 mg/m2/Week All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E.	Phase I Participants: 135 mg/m2/Week All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E.	Phase II Participants: 100 mg/m2/Week All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E.	Enrolled But Not Treated Participants who were enrolled but not randomized and did not receive treatment.
Overall Study Study terminated by sponsor	1	1	8	0
Overall Study Lost to Follow-up	0	0	1	0
Overall Study Enrolled but not treated	0	0	0	1

Baseline Characteristics

Study on Safety and Efficacy of NMS-01940153E in Adult Patients With Unresectable Hepatocellular Carcinoma (HCC) Previously Treated With Systemic Therapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Phase I Participants: 100 mg/m2/Week n=6 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	Phase I Treated Participants: 135 mg/m2/Week n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II Participants: 100 mg/m2/Week n=18 Participants All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E	Total n=30 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants	0 Participants n=7 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=99 Participants	3 Participants n=107 Participants	10 Participants n=206 Participants	16 Participants n=7 Participants
Age, Categorical >=65 years	3 Participants n=99 Participants	3 Participants n=107 Participants	8 Participants n=206 Participants	14 Participants n=7 Participants
Sex: Female, Male Female	1 Participants n=99 Participants	0 Participants n=107 Participants	1 Participants n=206 Participants	2 Participants n=7 Participants
Sex: Female, Male Male	5 Participants n=99 Participants	6 Participants n=107 Participants	17 Participants n=206 Participants	28 Participants n=7 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants	0 Participants n=7 Participants
Race (NIH/OMB) Asian	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants	0 Participants n=7 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants	0 Participants n=7 Participants
Race (NIH/OMB) Black or African American	1 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants	1 Participants n=7 Participants
Race (NIH/OMB) White	5 Participants n=99 Participants	6 Participants n=107 Participants	18 Participants n=206 Participants	29 Participants n=7 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants	0 Participants n=7 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants	0 Participants n=7 Participants
Eastern cooperative oncology group performance status ECOG Performance Status 0	3 participants n=99 Participants	3 participants n=107 Participants	10 participants n=206 Participants	16 participants n=7 Participants
Eastern cooperative oncology group performance status ECOG Performance Status 1	3 participants n=99 Participants	3 participants n=107 Participants	8 participants n=206 Participants	14 participants n=7 Participants

PRIMARY outcome

Timeframe: Phase I: From screening to end of first 28-day cycle (17 months)

Population: Phase I treated participants

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Phase I Drug Related Dose Limiting Toxicities (DLTs)	0 Participants	2 Participants	—

PRIMARY outcome

Timeframe: Phase II: From Phase II start to Study Completion (23 months)

Population: Evaluable population

The objective response rate (ORR) was calculated as the proportion of evaluable patients who achieved, as best overall response (BOR), confirmed complete response (CR) or partial response (PR) measured by investigator-assessed RECIST 1.1 (Phase II).

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=14 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Phase II Objective Response Rate Stable Disease	3 Participants	—	—
Phase II Objective Response Rate Progressive Disease	11 Participants	—	—
Phase II Objective Response Rate Complete Response	0 Participants	—	—
Phase II Objective Response Rate Partial Response	0 Participants	—	—

SECONDARY outcome

Timeframe: Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months)

Population: All treated participants

The maximum Common Terminology Criteria (CTC) grade (graded using National Cancer Institute \[NCI\] Common Terminology Criteria for Adverse Events \[CTCAE\] Version 5.0) experienced by each participant is presented. If an AE was reported for a participant more than once during treatment, the worst CTC Grade is presented here. Phase 2 started before Phase 1 completed. Grade 1 Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental activities of daily living. Grade 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activities of daily living. Grade 4 Life-threatening consequences; urgent intervention indicated.

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) n=18 Participants All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Treatment-emergent Adverse Events by Maximum CTC Grade Grade 1	0 Participants	0 Participants	3 Participants
Treatment-emergent Adverse Events by Maximum CTC Grade Grade 2	1 Participants	1 Participants	8 Participants
Treatment-emergent Adverse Events by Maximum CTC Grade Grade 3	3 Participants	3 Participants	7 Participants
Treatment-emergent Adverse Events by Maximum CTC Grade Grade 4	1 Participants	2 Participants	0 Participants

SECONDARY outcome

Timeframe: Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months)

Population: All treated participants

The number of treatment-emergent adverse events related to NMS-01940153E by maximum CTC grade experienced (graded using NCI CTCAE Version 5.0). Whether the the AE was related or not was assessed by the investigator. If an AE was reported for a participant more than once during treatment, the worst CTC Grade is presented. Grade 1 Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental activities of daily living. Grade 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activities of daily living. Grade 4 Life-threatening. Phase 2 started before Phase 1 completed.

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) n=18 Participants All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Treatment-emergent Adverse Events Related to NMS-01940153E Grade 1	0 events	0 events	7 events
Treatment-emergent Adverse Events Related to NMS-01940153E Grade 2	1 events	1 events	3 events
Treatment-emergent Adverse Events Related to NMS-01940153E Grade 3	2 events	2 events	2 events
Treatment-emergent Adverse Events Related to NMS-01940153E Grade 4	1 events	2 events	0 events

SECONDARY outcome

Timeframe: From screening to 28 days follow-up, an average 6 months

Population: All treated participants

Number of treatment emergent abnormalities (at any grade) at all dose levels are presented. CTC = Common Terminology Criteria WBC = white blood cells

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=24 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) n=30 Participants All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Hematology: Overall Treatment-emergent Abnormalities by Dose Level and Maximum CTC Grade Hemoglobin Decreased	13 events	5 events	18 events
Hematology: Overall Treatment-emergent Abnormalities by Dose Level and Maximum CTC Grade Neutrophil Count Decreased	6 events	4 events	10 events
Hematology: Overall Treatment-emergent Abnormalities by Dose Level and Maximum CTC Grade WBC Decreased	8 events	6 events	14 events
Hematology: Overall Treatment-emergent Abnormalities by Dose Level and Maximum CTC Grade Platelet Count Decreased	6 events	4 events	10 events

SECONDARY outcome

Timeframe: From screening to 28 days follow-up, an average 6 months

Population: Only participants who experienced G3 or G4 were analyzed.

The mean days to first occurrence of neutrophil count decrease are presented for all dose levels. Grade 0: ≥2,000/mm3 Grade 1: ≥1,500-\< 2,000/mm3 Grade 2: ≥1,000- \< 1,500/mm3 Grade 3: ≥500- \< 1,000/mm3 Grade 4: \<500/mm3 * G3 = Time (days) to Treatment Start to First Occurrence of Neutrophil Count Decrease \>=Grade 3 * G3 to Recovery to G1 = Time (days) to First Occurrence of Neutrophil Count Decrease \>= Grade 3 to Recovery to Grade 1

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=2 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=4 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) n=6 Participants All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Neutrophils Count Decrease: Time to First Occurrence of >=Grade 3 and Time to Recovery to Grade 1 >=G3	63.00 days Standard Deviation 19.80	28.00 days Standard Deviation 15.36	39.67 days Standard Deviation 23.38
Neutrophils Count Decrease: Time to First Occurrence of >=Grade 3 and Time to Recovery to Grade 1 >= G3 to Recovery to G1	7.00 days Standard Deviation 1.41	7.33 days Standard Deviation 3.51	7.20 days Standard Deviation 2.59

SECONDARY outcome

Timeframe: From screening to 28 days follow-up, an average 6 months

Population: All treated participants

Treatment-emergent abnormalities in blood chemistry at any grade are presented by dose level. ALP = Alkaline phosphatase ALT = Alanine aminotransferase AST = Aspartate aminotransferase GGT = Gamma-glutamyl transferase LDH = Lactate dehydrogenase

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=24 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) n=30 Participants All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Blood Chemistry: Treatment-emergent Abnormalities by Dose Level Hypoalbuminemia	8 events	4 events	12 events
Blood Chemistry: Treatment-emergent Abnormalities by Dose Level Hypoglycemia	0 events	1 events	1 events
Blood Chemistry: Treatment-emergent Abnormalities by Dose Level AST Increased	12 events	3 events	15 events
Blood Chemistry: Treatment-emergent Abnormalities by Dose Level ALT Increased	8 events	2 events	10 events
Blood Chemistry: Treatment-emergent Abnormalities by Dose Level GGT Increased	9 events	1 events	10 events
Blood Chemistry: Treatment-emergent Abnormalities by Dose Level Creatinine Increased	6 events	3 events	9 events
Blood Chemistry: Treatment-emergent Abnormalities by Dose Level Blood Bilirubin Increased	7 events	1 events	8 events
Blood Chemistry: Treatment-emergent Abnormalities by Dose Level ALP Increased	10 events	1 events	11 events
Blood Chemistry: Treatment-emergent Abnormalities by Dose Level Blood LDH Increased	14 events	4 events	18 events
Blood Chemistry: Treatment-emergent Abnormalities by Dose Level Hypocalcemia	1 events	1 events	2 events
Blood Chemistry: Treatment-emergent Abnormalities by Dose Level Hypernatremia	0 events	1 events	1 events
Blood Chemistry: Treatment-emergent Abnormalities by Dose Level Hyperkalemia	7 events	0 events	7 events
Blood Chemistry: Treatment-emergent Abnormalities by Dose Level Hypomagnesemia	5 events	3 events	8 events

SECONDARY outcome

Timeframe: From screening to 28 days follow-up, an average 6 months

Population: All treated participants

Treatment Emergent abnormalities by dose Level are presented for blood chemistry and coagulation parameters. INR = international normalized ratio LNL = lower normal limit NL = normal limit ULN = upper limit of normal

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=24 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) n=30 Participants All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Hyponatremia: Total	4 events	2 events	6 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Hyponatremia: Below LNL	4 events	2 events	6 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Hypokalemia: Total	0 events	1 events	1 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Hypokalemia: Below LNL	0 events	1 events	1 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Phosphatemia: Total	11 events	2 events	13 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Phosphatemia: Above ULN	3 events	0 events	3 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Phosphatemia: Below LNL	7 events	2 events	9 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Phosphatemia: Above/Below NL	1 events	0 events	1 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Hyperglycemia: Total	9 events	2 events	11 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Hyperglycemia: Above ULN	9 events	1 events	10 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Hyperglycemia: Above/Below NL	0 events	1 events	1 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Blood Urea Nitrogen: Total	2 events	0 events	2 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Blood Urea Nitrogen: Above ULN	2 events	0 events	2 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Urea: Total	6 events	0 events	6 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Urea: Above ULN	5 events	0 events	5 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Urea: Below LNL	1 events	0 events	1 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Unconjugated Bilirubin: Total	3 events	0 events	3 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Unconjugated Bilirubin: Above ULN	3 events	0 events	3 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Total Protein: Total	7 events	2 events	9 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Total Protein: Above ULN	2 events	0 events	2 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Total Protein: Below LNL	4 events	2 events	6 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities Total Protein: Above/Below NL	1 events	0 events	1 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities INR: Total	11 events	1 events	12 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities INR: Above ULN	8 events	1 events	9 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities INR: Below LNL	1 events	0 events	1 events
Blood Chemistry and Coagulation: Treatment-emergent Abnormalities INR: Above/Below NL	2 events	0 events	2 events

SECONDARY outcome

Timeframe: From screening to 28 days follow-up, an average 6 months

Population: All treated participants

The number of participants who experienced electrocardiogram abnormalities are presented.

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) n=18 Participants All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Electrocardiogram Abnormalities	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Day 1 to Day 15

Population: All Phase I participants

Plasma pharmacokinetic (PK) profiles of NMS-01940153E were characterized on Day 1 and 15 of the first Cycle after infusion. Tmax = Time to maximum observed plasma concentration Tlast = Time of last detectable concentration

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Tmax and Tlast of NMS-01940153E Tmax: Day 1	0.933 hours Standard Deviation 0.0327	1.19 hours Standard Deviation 0.513	—
Tmax and Tlast of NMS-01940153E Tmax: Day 15	1.10 hours Standard Deviation 0.216	1.87 hours Standard Deviation 1.90	—
Tmax and Tlast of NMS-01940153E Tlast: Day 1	169 hours Standard Deviation 3.01	154 hours Standard Deviation 39.2	—
Tmax and Tlast of NMS-01940153E Tlast: Day 15	144 hours Standard Deviation 48.5	169 hours Standard Deviation 0.418	—

SECONDARY outcome

Timeframe: Day 1 to Day 15

Population: All Phase I participants

Plasma pharmacokinetic (PK) profiles of NMS-01940153E were characterized on Day 1 and 15 of the first Cycle after infusion. Cmax = Maximum observed plasma concentration Clast = Last detectable concentration

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Cmax and Clast of NMS-01940153E Clast: Day 1	0.0590 μM Standard Deviation 0.0191	0.0840 μM Standard Deviation 0.0335	—
Cmax and Clast of NMS-01940153E Cmax: Day 1	0.754 μM Standard Deviation 0.174	1.91 μM Standard Deviation 0.640	—
Cmax and Clast of NMS-01940153E Cmax: Day 15	1.35 μM Standard Deviation 1.10	1.59 μM Standard Deviation 1.31	—
Cmax and Clast of NMS-01940153E Clast: Day 15	0.113 μM Standard Deviation 0.0454	0.133 μM Standard Deviation 0.0779	—

SECONDARY outcome

Timeframe: From Days 1 to 21 (168 hours after the Day 15 infusion)

Population: All Phase I participants

Plasma pharmacokinetic (PK) profiles of NMS-01940153E were characterized on Day 1 and 15 of the first Cycle after infusion. Infusions occurred on Days 1, 8, and 15. AUClast = Area under the interpolated observed plasma time-concentration curve from infusion start to the last observed plasma concentration AUCweekly = Area under the interpolated observed plasma time-concentration curve from infusion start to 168 hours AUCinf = Area under the interpolated observed plasma time-concentration curve from infusion start extrapolated to infinity based on the last observed plasma concentration

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
AUClast, AUCweekly, and AUCinf of NMS-01940153E AUClast: Day 1	20.8 h·μM Standard Deviation 4.31	24.8 h·μM Standard Deviation 8.55	—
AUClast, AUCweekly, and AUCinf of NMS-01940153E AUClast: Day 15	30.0 h·μM Standard Deviation 9.16	39.1 h·μM Standard Deviation 14.9	—
AUClast, AUCweekly, and AUCinf of NMS-01940153E AUCweekly: Day 1	20.8 h·μM Standard Deviation 4.35	25.7 h·μM Standard Deviation 7.11	—
AUClast, AUCweekly, and AUCinf of NMS-01940153E AUCweekly: Day 15	32.4 h·μM Standard Deviation 6.49	39.0 h·μM Standard Deviation 14.8	—
AUClast, AUCweekly, and AUCinf of NMS-01940153E AUCinf: Day 1	28.7 h·μM Standard Deviation 6.88	37.1 h·μM Standard Deviation 14.9	—
AUClast, AUCweekly, and AUCinf of NMS-01940153E AUCinf: Day 15	48.1 h·μM Standard Deviation 19.6	62.9 h·μM Standard Deviation 34.1	—

SECONDARY outcome

Timeframe: Day 1 to Day 15

Population: All Phase I participants

Plasma pharmacokinetic (PK) profiles of NMS-01940153E were characterized on Day 1 and 15 of the first Cycle after infusion. t½,z = Half-life of the terminal phase of observed plasma concentration

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
t½,z of NMS-01940153E t½,z Day 1	3.74 days Standard Deviation 0.800	4.17 days Standard Deviation 1.40	—
t½,z of NMS-01940153E t½,z Day 15	4.38 days Standard Deviation 2.64	4.79 days Standard Deviation 1.15	—

SECONDARY outcome

Timeframe: Day 1 to Day 15

Population: All Phase I participants

Plasma pharmacokinetic (PK) profiles of NMS-01940153E were characterized on Day 1 and 15 of the first Cycle after infusion. CL = Clearance based on last observed concentration and extrapolation to infinity CLss = Clearance calculated for a dosing period of 168 h either after a single dose or in steady-state

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
CL and CLss of NMS-01940153E CL: Day 1	10.1 L/h Standard Deviation 3.16	10.2 L/h Standard Deviation 4.01	—
CL and CLss of NMS-01940153E CL: Day 15	6.12 L/h Standard Deviation 2.24	6.19 L/h Standard Deviation 2.08	—
CL and CLss of NMS-01940153E CLss: Day 1	13.8 L/h Standard Deviation 4.14	13.8 L/h Standard Deviation 3.73	—
CL and CLss of NMS-01940153E CLss: Day 15	8.53 L/h Standard Deviation 2.50	9.31 L/h Standard Deviation 2.52	—

SECONDARY outcome

Timeframe: Day 1 to Day 15

Population: All Phase I participants

Plasma pharmacokinetic (PK) PK profiles of NMS-01940153E were characterized on Day 1 and 15 of the first Cycle after infusion. Vss = Volume of distribution at steady state based last observed concentration extrapolated to infinity and Clearance calculated for a dosing period of 168 h either after a single dose or in steady-state Vss,SS = Volume of distribution at steady state based last observed concentration extrapolated to infinity and clearance based on last observed concentration and extrapolation to infinity

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Vss and Vss,SS of NMS-01940153E Vss: Day 1	1230 L Standard Deviation 395	1210 L Standard Deviation 199	—
Vss and Vss,SS of NMS-01940153E Vss: Day 15	787 L Standard Deviation 304	919 L Standard Deviation 245	—
Vss and Vss,SS of NMS-01940153E Vss,SS: Day 1	1700 L Standard Deviation 620	1630 L Standard Deviation 285	—
Vss and Vss,SS of NMS-01940153E Vss,SS: Day 15	1180 L Standard Deviation 649	1380 L Standard Deviation 354	—

SECONDARY outcome

Timeframe: Day 21 (168 hours after the Day 15 infusion)

Population: All Phase I participants

Plasma pharmacokinetic (PK) PK profiles of NMS-01940153E were characterized on Day 1 and 15 of the first Cycle after infusion. Infusions occurred on Days 1, 8, and 15. RA AUCweekly = Accumulation ratio Day 1 to 15 calculated using area under the interpolated observed plasma time-concentration curve from infusion start to 168 hours RA Cmax = Accumulation ratio Day 1 to 15 calculated using maximum observed plasma concentration

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
RA AUCweekly and RA Cmax of NMS-01940153E RA AUCweekly: Day 15	1.66 ratio Standard Deviation 0.188	1.51 ratio Standard Deviation 0.293	—
RA AUCweekly and RA Cmax of NMS-01940153E RA Cmax: Day 15	1.78 ratio Standard Deviation 1.36	0.858 ratio Standard Deviation 0.547	—

SECONDARY outcome

Timeframe: Day 1 to Day 15

Population: All treated participants

Urine pharmacokinetic profiles of NMS-01940153E were characterized on Day 1 and 15 of the first Cycle after infusion. FE = Molar fraction of excreted compound relative to the administered molar dose calculated using urine concentration and volume data

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
FE of NMS-01940153E FE Day 1	0.458 percentage Standard Deviation 0.374	0.357 percentage Standard Deviation 0.260	—
FE of NMS-01940153E FE Day 15	0.563 percentage Standard Deviation 0.424	0.826 percentage Standard Deviation 0.713	—

SECONDARY outcome

Timeframe: Phase I: From the Study Start Date to Phase I Completion (32 months)

Population: Treated Patients with at Least One RECIST 1.1 On-Treatment Assessment

Phase I any dose level, objective response and best overall tumor response are presented, as measured by investigator assessed RECIST 1.1 (Phase I). The objective response rate was calculated as the proportion of evaluable patients who achieved best overall response (BOR), confirmed complete response (CR) or partial response (PR).

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=5 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Phase I Objective Tumor Response (Partial and Complete Response) Objective Response (Partial Response)	1 Participants	1 Participants	—
Phase I Objective Tumor Response (Partial and Complete Response) Objective Response (Complete Response)	0 Participants	0 Participants	—
Phase I Objective Tumor Response (Partial and Complete Response) Stable Disease	1 Participants	2 Participants	—
Phase I Objective Tumor Response (Partial and Complete Response) Progressive Disease	4 Participants	2 Participants	—

SECONDARY outcome

Timeframe: Phase II: From Phase II start to Study Completion (23 months)

Population: Evaluable participants with at least one mRECIST Assessment on Treatment were analyzed

Objective response rate as measured by investigator-assessed mRECIST in Phase II.

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=14 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Phase II Objective Response Rate as Measured by Investigator-assessed mRECIST Stable Disease	3 Participants	—	—
Phase II Objective Response Rate as Measured by Investigator-assessed mRECIST Progressive Disease	11 Participants	—	—
Phase II Objective Response Rate as Measured by Investigator-assessed mRECIST Objective Response (Partial Response)	0 Participants	—	—
Phase II Objective Response Rate as Measured by Investigator-assessed mRECIST Objective Response (Complete Response)	0 Participants	—	—

SECONDARY outcome

Timeframe: Phase I: From the Study Start Date to Phase I Completion (32 months)

Population: Phase I participants who experienced partial response

Duration of response (DoR) as measured by investigator-assessed Response evaluation criteria in solid tumours (RECIST) 1.1. Duration of Response (months): was time in months from response start date to either date of disease progression/death due to progression of disease. It was calculated only for participants with complete response or partial response as best overall response.

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=1 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=1 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Phase I: Duration of Response (DoR) as Measured by Investigator-assessed RECIST 1.1	2.6 months	9.3 months	—

SECONDARY outcome

Timeframe: Phase II: From Phase II start to Study Completion (23 months)

Population: Phase II Evaluable Population. No participants had a RECIST or mRECIST complete or partial response observed.

Duration of response (DoR) as measured by investigator-assessed Response evaluation criteria in solid tumours (RECIST) 1.1 and investigator-assessed modified RECIST (mRECIST). Duration of Response (months): was time in months from response start date to either date of disease progression/death due to progression of disease. It was calculated only for participants with complete response or partial response as best overall response.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months)

Population: All evaluable participants

Progression Free Survival as measured by investigator assessed RECIST 1.1, for all treated participants in Phase I and Phase II. Phase 2 started before Phase 1 completed.

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) n=14 Participants All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Progression Free Survival, as Measured by Investigator-assessed RECIST 1.1, in Phases I and II	1.9 months Interval 1.7 to 6.5	7.8 months Interval 1.8 to 25.1	1.9 months Interval 1.8 to 4.1

SECONDARY outcome

Timeframe: Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months)

Population: Evaluable population

Overall Survival for all treated participants in Phases I and II. The estimates are based on the Kaplan-Meier (KM) method. Phase 2 started before Phase 1 completed.

Outcome measures

Outcome measures
Measure	100 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	135 mg/m2/Week Dose (Phase I) n=6 Participants All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II (Evaluable Population) n=14 Participants All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E in the Evaluable Population. The Evaluable Population was participants of the treated set who had measurable disease at baseline assessed according to RECIST 1.1 and at least one tumor evaluation on treatment, unless they died before the first tumor on-treatment assessment, in which case they were considered treatment failure.
Overall Survival in Phases I and II	9.7 months Interval 5.2 to Insufficient number of participants with events	15.7 months Interval 4.7 to Insufficient number of participants with events	7.3 months Interval 3.9 to Insufficient number of participants with events

Adverse Events

Phase I: 100 mg/m2/Week

Serious events: 1 serious events

Other events: 5 other events

Deaths: 5 deaths

Phase I: 135 mg/m2/Week

Serious events: 2 serious events

Other events: 6 other events

Deaths: 5 deaths

Phase II: 100 mg/m2/Week

Serious events: 5 serious events

Other events: 18 other events

Deaths: 9 deaths

Serious adverse events

Serious adverse events
Measure	Phase I: 100 mg/m2/Week n=6 participants at risk All participants in Phase I who received 100 mg/m2/week intravenous NMS-01940153E	Phase I: 135 mg/m2/Week n=6 participants at risk All participants in Phase I who received 135 mg/m2/week intravenous NMS-01940153E	Phase II: 100 mg/m2/Week n=18 participants at risk All participants in Phase II who received 100 mg/m2/week intravenous NMS-01940153E
Blood and lymphatic system disorders Anaemia	0.00% 0/6 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.	16.7% 1/6 • Number of events 1 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.	0.00% 0/18 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.
Blood and lymphatic system disorders Neutropenia	0.00% 0/6 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.	16.7% 1/6 • Number of events 1 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.	0.00% 0/18 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.
General disorders Disease progression	0.00% 0/6 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.	0.00% 0/6 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.	11.1% 2/18 • Number of events 2 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.
Musculoskeletal and connective tissue disorders Pathological fracture	0.00% 0/6 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.	0.00% 0/6 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.	5.6% 1/18 • Number of events 1 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.
Infections and infestations Sepsis	16.7% 1/6 • Number of events 1 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.	16.7% 1/6 • Number of events 1 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.	5.6% 1/18 • Number of events 1 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.
Infections and infestations Device related bacteraemia	16.7% 1/6 • Number of events 1 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.	0.00% 0/6 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.	0.00% 0/18 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.
Infections and infestations Device related infection	0.00% 0/6 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.	0.00% 0/6 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.	5.6% 1/18 • Number of events 1 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.
Infections and infestations Peritonitis bacterial	0.00% 0/6 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.	0.00% 0/6 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.	5.6% 1/18 • Number of events 1 • Phase I: From the Study Start Date to Phase I Completion (32 months) - Phase II: From Phase II start to Study Completion (23 months). Phase 2 started before Phase 1 completed. Adverse events were coded by MedDRA and severity was graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE, version 5.0). For non-serious AEs, the number of participants who experienced at least 1 occurrence of a treatment-emergent non-serious AEs, by MedDRA System Organ Class and Preferred Term are presented. The one participant who was enrolled but not treated was not assessed for adverse events and therefore this cohort has not been included.

Other adverse events

Additional Information

Alberto Ocana

Nerviano Medical Sciences S.r.l.

Phone: 39 0331 58 1111

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60