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Clinical Trial of Lurbinectedin (PM01183) in Selected Advanced Solid Tumors

A Multicenter Phase II Clinical Trial of Lurbinectedin (PM01183) in Selected Advanced Solid Tumors

Status
Completed
Phases
Phase 2
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT02454972
Enrollment
345
Registered
2015-05-27
Start date
2015-08-25
Completion date
2020-09-18
Last updated
2023-03-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Advanced Solid Tumors

Brief summary

Multicenter, open-label, exploratory, phase II clinical trial to evaluate the efficacy and safety of PM01183 in previously treated patients with advanced solid tumors

Detailed description

Patients with relapsed small cell lung cancer (SCLC), head and neck carcinoma (H&N), neuroendocrine tumors (NETs), biliary tract carcinoma, endometrial carcinoma, BRCA 1/2-associated metastatic breast carcinoma, carcinoma of unknown primary site, germ cell tumors (GCTs), and Ewing's family of tumors (EFTs) will be enrolled in nine different cohorts. Up to 25 evaluable patients are planned to be enrolled in each cohort (50 in the endometrial carcinoma and 100 in the SCLC cohort).

Interventions

DRUGlurbinectedin (PM01183)

Sponsors

PharmaMar
Lead SponsorINDUSTRY

Study design

Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE

Eligibility

Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No

Inclusion criteria

* Age ≥ 18 years. * Voluntary signed informed consent (IC) * Pathologically proven diagnosis of any of the following malignancies: * Small cell lung cancer (SCLC). * Head and neck carcinoma (H&N). Salivary glands tumors are excluded. * Neuroendocrine tumors (NETs), grade 2 and grade 3 according to World Health Organization classification. * Biliary tract carcinoma. * Endometrial carcinoma. * BRCA 1/2- associated metastatic breast carcinoma * Carcinoma of unknown primary site. * Germ cell tumor (GCTs), excluding immature teratoma, or teratoma with malignant transformation. * Ewing's family of tumors (EFTs) * Prior treatment. Patients must have received: * SCLC, endometrial carcinoma: one prior chemotherapy-containing line. * H&N, NETs, biliary tract, CUP: one or two prior chemotherapy-containing lines * GCTs: no limit of prior therapy * EFTs: no more than two prior chemotherapy-containing lines in the metastatic/recurrent setting. * BRCA 1/2-associated metastatic breast carcinoma: at least one but no more than three prior chemotherapy-containing lines. * Performance status ≤ 2 \[Eastern Cooperative Oncology Group (ECOG)\] * Adequate major organ function * At least three weeks since the last chemotherapy * Women of childbearing potential must have pregnancy excluded by appropriate testing before study entry

Exclusion criteria

* Prior treatment with PM01183 or trabectedin * Prior or concurrent malignant disease unless in complete remission for more than five years * Known central nervous system (CNS) involvement * Relevant diseases or clinical situations which may increase the patient's risk * Pregnant or breastfeeding women and fertile patients (men and women) who are not using an effective method of contraception

Design outcomes

Primary

MeasureTime frameDescription
Overall Response Rate (ORR)From the start of treatment to the date of progression or the start of a subsequent therapy or end of patient's follow-up, until Cycle 6 (21-day cycle)Overall Response Rate was defined as the percentage of patients with a confirmed response, either CR or PR, according to the RECIST v.1.1. Complete Response (CR): Disappearance of all target lesions; Partial Response (PR): ≥30% decrease in the sum of the longest diameters of target lesions compared with baseline; Progressive disease: ≥20% increase in the sum of the longest diameter of target lesions compared with the smallest-sum longest; diameter recorded or the appearance of one or more new lesions; Stable Disease: Neither PR or PD
Response by Investigator AssessmentFrom the start of treatment to the date of progression or the start of a subsequent therapy or end of patient's follow-up, until Cycle 6 (21-day cycle)When response is the primary endpoint, and thus all patients must have measurable disease to enter the trial, all patients included in the study must be accounted for in the report of the results, even if there are major protocol treatment deviations or if they are not evaluable. Each patient will be assigned one of the following: Complete Response: Disappearance of all target lesions; Partial Response: ≥30% decrease in the sum of the longest diameters of target lesions; Progressive disease: ≥20% increase in the sum of the longest diameter of target lesions; diameter recorded or the appearance of one or more new lesions; Stable Disease: Neither PR or PD; Inevaluable for response: specify reasons (for example: early death, malignant disease, toxicity; tumour assessments not repeated/incomplete; other). Normally, all eligible patients should be included in the denominator for the calculation of the response rate for phase II trials.

Secondary

MeasureTime frameDescription
Disease Control RateFrom the start of treatment to the date of progression or the start of a subsequent therapy or end of patient's follow-up, until Cycle 6Disease Control Rate was defined as Overall Response Rate or Stable Disease
Progression Free Survival (PFS)From the date of first infusion to the date of progression disease, death (of any cause), or last tumor evaluation, up to an average of 5 yearsProgression-free Survival (PFS), defined as the period of time from the date of first infusion to the date of PD, death (of any cause), or last tumor evaluation. Progressive disease: ≥20% increase in the sum of the longest diameter of target lesions compared with the smallest-sum longest
Progression-free Survival at 4 MonthsAt 4 monthsProgression-free Survival at 4, defined as the probability of being free from progression and death after the first infusion at 4 months. Progressive disease: ≥20% increase in the sum of the longest diameter of target lesions compared with the smallest-sum longest
Duration of ResponseFrom the start of treatment to the date of progression or the start of a subsequent therapy or end of patient's follow-up, until Cycle 6 (21-day cycle)Duration of Response by Investigator's Assessment, defined as the time between the date when the response criteria (PR or CR, whichever one is first reached) are fulfilled to the first date when disease progression (PD), recurrence or death is documented.
Overall Survival (OS)From the date of first infusion to the date of death or last contact, up to an average of 5 yearsOverall survival defined as the period of time from the date of first infusion to the date of death or last contact in case of patients lost to follow-up or alive at the clinical cutoff established for the cohort.
Overall Survival at 6 MonthsAt 6 monthsOverall Survival at 6 months defined as the probability of being alive after the first infusion at 6 months
Overall Survival at 12 MonthsAt 12 monthsOverall Survival at 12 months defined as the probability of being alive after the first infusion at 12 months
Progression-free Survival at 6 MonthsAt 6 monthsProgression-free Survival at 6, defined as the probability of being free from progression and death after the first infusion at 6 months. Progressive disease: ≥20% increase in the sum of the longest diameter of target lesions compared with the smallest-sum longest
Clinical BenefitFrom the start of treatment to the date of progression or the start of a subsequent therapy or end of patient's follow-up, until Cycle 6 (21-day cycle)Clinical Benefit Rate was defined as Overall Response Rate or Stable Disease lasting over four months (SD ≥ 4 months)

Countries

Belgium, France, Germany, Italy, Spain, Sweden, Switzerland, United Kingdom, United States

Participant flow

Recruitment details

The first patient registration was on 25 August 2015 and the first study treatment administration was on 25 August 2015. The last patient registration was on 30 November 2018 and the last study treatment administration was on 29 November 2019. The date of last follow-up (cutoff-date) was 18 September 2020.

Participants by arm

ArmCount
Biliary Tract Carcinoma Cohort
Patients with Pathologically proven diagnosis of biliary tract carcinoma
19
Carcinoma of Unknown Primary Site Cohort
Patients with pathologically proven diagnosis of carcinoma of unknown primary site
19
Endometrial Carcinoma Cohort
Patients with pathologically proven diagnosis of endometrial carcinoma
73
Ewing's Family of Tumors Cohort
Patients with pathologically proven diagnosis of Ewing's Family of Tumors
28
Germ Cell Tumors Cohort
Patients with pathologically proven diagnosis of Germ Cell Tumors, excluding immature teratoma, or teratoma with malignant transformation.
23
Head and Neck Carcinoma Cohort
Patients with Pathologically proven diagnosis of Head and Neck Carcinoma. Salivary glands tumors were excluded.
15
BRCA1/2-associated Metastatic Breast Carcinoma Cohort
Patients with pathologically proven diagnosis of BRCA1/2-associated metastatic breast carcinoma
21
Neuroendocrine Tumors Cohort
Patients with Pathologically proven diagnosis of Neuroendocrine Tumors, grade 2 and 3 according to World Health Organization (WHO) classification.
32
Small Cell Lung Cancer Cohort
Patients with pathologically proven diagnosis of small cell lung cancer
105
Total335

Withdrawals & dropouts

PeriodReasonFG000FG001FG002FG003FG004FG005FG006FG007FG008
Overall StudyDeath0051000095
Overall StudyLost to Follow-up000000002
Overall StudyMultiple delay/holds on treatment000100000
Overall StudyNever treated003110005
Overall StudyNon treatment-related adverse event201001010
Overall StudyPatient moves to compassionate use000000200
Overall StudyPhysician Decision012120010
Overall StudyProgressive disease17165923161219270
Overall StudyStudy termination000000006
Overall StudyTreatment-related adverse events011020020
Overall StudyWithdrawal by Subject015232012

Baseline characteristics

CharacteristicTotalBiliary Tract Carcinoma CohortCarcinoma of Unknown Primary Site CohortEndometrial Carcinoma CohortEwing's Family of Tumors CohortGerm Cell Tumors CohortHead and Neck Carcinoma CohortBRCA1/2-associated Metastatic Breast Carcinoma CohortNeuroendocrine Tumors CohortSmall Cell Lung Cancer Cohort
Advanced chemotherapy lines
0 lines
145 Participants19 Participants0 Participants15 Participants5 Participants0 Participants0 Participants1 Participants0 Participants105 Participants
Advanced chemotherapy lines
1 line
94 Participants0 Participants12 Participants47 Participants7 Participants1 Participants5 Participants5 Participants17 Participants0 Participants
Advanced chemotherapy lines
2 lines
58 Participants0 Participants7 Participants8 Participants13 Participants4 Participants9 Participants7 Participants10 Participants0 Participants
Advanced chemotherapy lines
3 lines
24 Participants0 Participants0 Participants2 Participants3 Participants8 Participants1 Participants6 Participants4 Participants0 Participants
Advanced chemotherapy lines
4 or more lines
14 Participants0 Participants0 Participants1 Participants0 Participants10 Participants0 Participants2 Participants1 Participants0 Participants
Age, Categorical
<=18 years
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Age, Categorical
>=65 years
113 Participants6 Participants9 Participants36 Participants1 Participants3 Participants6 Participants2 Participants13 Participants37 Participants
Age, Categorical
Between 18 and 65 years
222 Participants13 Participants10 Participants37 Participants27 Participants20 Participants9 Participants19 Participants19 Participants68 Participants
Age, Continuous60 years61 years61 years64 years33 years36 years62 years45 years63 years60 years
Albumin
<3.5 g/dL
50 Participants5 Participants4 Participants9 Participants2 Participants4 Participants3 Participants1 Participants9 Participants13 Participants
Albumin
≥3.5 g/dL
285 Participants14 Participants15 Participants64 Participants26 Participants19 Participants12 Participants20 Participants23 Participants92 Participants
Albumin4.0 g/dL3.7 g/dL3.7 g/dL4.1 g/dL4.2 g/dL4.1 g/dL3.8 g/dL4.1 g/dL4.0 g/dL4.1 g/dL
Best response to last therapy
Complete response
20 Participants0 Participants3 Participants7 Participants0 Participants0 Participants0 Participants1 Participants0 Participants9 Participants
Best response to last therapy
Partial reponse
112 Participants4 Participants4 Participants20 Participants0 Participants2 Participants5 Participants2 Participants5 Participants70 Participants
Best response to last therapy
Progression disease
71 Participants8 Participants4 Participants15 Participants0 Participants15 Participants7 Participants7 Participants11 Participants4 Participants
Best response to last therapy
Stable disease
71 Participants7 Participants5 Participants13 Participants0 Participants5 Participants2 Participants6 Participants14 Participants19 Participants
Best response to last therapy
Unknown
61 Participants0 Participants3 Participants18 Participants28 Participants1 Participants1 Participants5 Participants2 Participants3 Participants
Body surface area1.8 m^21.9 m^21.8 m^21.8 m^21.9 m^22.0 m^21.8 m^21.7 m^21.7 m^21.8 m^2
Eastern Cooperative Oncology Group performance status
0
127 Participants5 Participants8 Participants32 Participants11 Participants2 Participants5 Participants18 Participants8 Participants38 Participants
Eastern Cooperative Oncology Group performance status
1
188 Participants14 Participants10 Participants35 Participants16 Participants19 Participants10 Participants2 Participants23 Participants59 Participants
Eastern Cooperative Oncology Group performance status
2
20 Participants0 Participants1 Participants6 Participants1 Participants2 Participants0 Participants1 Participants1 Participants8 Participants
Height165 cm168 cm164.5 cm161 cm173 cm177 cm170 cm163.0 cm169 cm167 cm
Number of sites at baseline3 sites at baseline3 sites at baseline2 sites at baseline2 sites at baseline2 sites at baseline3 sites at baseline2 sites at baseline2 sites at baseline3 sites at baseline3 sites at baseline
Prior radiotherapy190 Participants2 Participants8 Participants39 Participants20 Participants7 Participants11 Participants20 Participants7 Participants76 Participants
Prior surgery148 Participants2 Participants2 Participants62 Participants20 Participants21 Participants9 Participants19 Participants11 Participants2 Participants
Race/Ethnicity, Customized
American Indian or Alaska native
1 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants1 Participants0 Participants
Race/Ethnicity, Customized
Asian
6 Participants0 Participants0 Participants1 Participants2 Participants1 Participants1 Participants0 Participants0 Participants1 Participants
Race/Ethnicity, Customized
Black of African American
7 Participants0 Participants0 Participants5 Participants1 Participants0 Participants0 Participants0 Participants0 Participants1 Participants
Race/Ethnicity, Customized
Not race available
79 Participants6 Participants4 Participants22 Participants4 Participants7 Participants2 Participants3 Participants7 Participants24 Participants
Race/Ethnicity, Customized
White
242 Participants13 Participants15 Participants45 Participants21 Participants15 Participants12 Participants18 Participants24 Participants79 Participants
Region of Enrollment
Belgium
13 Participants2 Participants2 Participants3 Participants1 Participants0 Participants0 Participants0 Participants2 Participants3 Participants
Region of Enrollment
France
62 Participants4 Participants2 Participants17 Participants3 Participants6 Participants2 Participants3 Participants5 Participants20 Participants
Region of Enrollment
Germany
2 Participants0 Participants0 Participants2 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Region of Enrollment
Italy
6 Participants0 Participants0 Participants0 Participants3 Participants1 Participants0 Participants0 Participants0 Participants2 Participants
Region of Enrollment
Spain
151 Participants10 Participants8 Participants23 Participants5 Participants4 Participants9 Participants15 Participants18 Participants59 Participants
Region of Enrollment
Sweden
1 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants1 Participants0 Participants
Region of Enrollment
Switzerland
10 Participants0 Participants0 Participants2 Participants0 Participants0 Participants0 Participants1 Participants0 Participants7 Participants
Region of Enrollment
United Kingdom
13 Participants0 Participants1 Participants9 Participants0 Participants0 Participants0 Participants0 Participants0 Participants3 Participants
Region of Enrollment
United States
77 Participants3 Participants6 Participants17 Participants16 Participants12 Participants4 Participants2 Participants6 Participants11 Participants
Sex: Female, Male
Female
189 Participants10 Participants11 Participants73 Participants12 Participants7 Participants1 Participants21 Participants12 Participants42 Participants
Sex: Female, Male
Male
146 Participants9 Participants8 Participants0 Participants16 Participants16 Participants14 Participants0 Participants20 Participants63 Participants
Sites at baseline
<3 sites
144 Participants6 Participants12 Participants40 Participants18 Participants7 Participants10 Participants12 Participants13 Participants26 Participants
Sites at baseline
≥3 sites
191 Participants13 Participants7 Participants33 Participants10 Participants16 Participants5 Participants9 Participants19 Participants79 Participants
Stage at diagnosis
Early
67 Participants0 Participants0 Participants23 Participants14 Participants8 Participants4 Participants10 Participants5 Participants3 Participants
Stage at diagnosis
Locally advanced
87 Participants4 Participants0 Participants27 Participants5 Participants1 Participants7 Participants6 Participants8 Participants29 Participants
Stage at diagnosis
Metastatic
181 Participants15 Participants19 Participants23 Participants9 Participants14 Participants4 Participants5 Participants19 Participants73 Participants
Systemic lines
1 line
201 Participants13 Participants12 Participants54 Participants5 Participants0 Participants5 Participants0 Participants14 Participants98 Participants
Systemic lines
2 lines
82 Participants6 Participants7 Participants15 Participants15 Participants4 Participants8 Participants7 Participants13 Participants7 Participants
Systemic lines
3 lines
29 Participants0 Participants0 Participants3 Participants5 Participants8 Participants2 Participants7 Participants4 Participants0 Participants
Systemic lines
4 or more lines
23 Participants0 Participants0 Participants1 Participants3 Participants11 Participants0 Participants7 Participants1 Participants0 Participants
Time from advanced disease to registration15.0 month10.6 monthNA month17.5 month20.4 month33.5 month18.4 month31.9 month17.2 month9.1 month
Time from diagnosis to registration13.7 months8.4 months10.8 months18.4 months28.6 months48.2 months19.5 months50.1 months13.3 months8.2 months
Time from last progression disease before study entry2.1 weeks3.0 weeks2.6 weeks2.9 weeks2.1 weeks1.4 weeks2.4 weeks1.6 weeks2.6 weeks1.6 weeks
Time to progression to last prior therapy6.4 months5.2 months3.9 months8.0 months8.7 months2.7 months4.6 months5.0 months3.6 months6.5 months
Weight71.0 kg72.2 kg65.0 kg70.0 kg77.0 kg78.5 kg73.0 kg65.5 kg64.0 kg71.0 kg

Adverse events

Event typeEG000
affected / at risk
deaths
Total, all-cause mortality
261 / 335
other
Total, other adverse events
330 / 335
serious
Total, serious adverse events
136 / 335

Outcome results

Primary

Overall Response Rate (ORR)

Overall Response Rate was defined as the percentage of patients with a confirmed response, either CR or PR, according to the RECIST v.1.1. Complete Response (CR): Disappearance of all target lesions; Partial Response (PR): ≥30% decrease in the sum of the longest diameters of target lesions compared with baseline; Progressive disease: ≥20% increase in the sum of the longest diameter of target lesions compared with the smallest-sum longest; diameter recorded or the appearance of one or more new lesions; Stable Disease: Neither PR or PD

Time frame: From the start of treatment to the date of progression or the start of a subsequent therapy or end of patient's follow-up, until Cycle 6 (21-day cycle)

Population: Treated patients and with disease measurement

ArmMeasureValue (NUMBER)
Biliary Tract Carcinoma CohortOverall Response Rate (ORR)5.6 percentage of participants
Carcinoma of Unknown Primary Site CohortOverall Response Rate (ORR)0.0 percentage of participants
Endometrial Carcinoma CohortOverall Response Rate (ORR)11.3 percentage of participants
Ewing's Family of Tumors CohortOverall Response Rate (ORR)14.3 percentage of participants
Germ Cell Tumors CohortOverall Response Rate (ORR)4.3 percentage of participants
Head and Neck Carcinoma CohortOverall Response Rate (ORR)0.0 percentage of participants
BRCA1/2-associated Metastatic Breast Carcinoma CohortOverall Response Rate (ORR)28.6 percentage of participants
Neuroendocrine Tumors CohortOverall Response Rate (ORR)6.5 percentage of participants
Small Cell Lung Cancer CohortOverall Response Rate (ORR)36.2 percentage of participants
Primary

Response by Investigator Assessment

When response is the primary endpoint, and thus all patients must have measurable disease to enter the trial, all patients included in the study must be accounted for in the report of the results, even if there are major protocol treatment deviations or if they are not evaluable. Each patient will be assigned one of the following: Complete Response: Disappearance of all target lesions; Partial Response: ≥30% decrease in the sum of the longest diameters of target lesions; Progressive disease: ≥20% increase in the sum of the longest diameter of target lesions; diameter recorded or the appearance of one or more new lesions; Stable Disease: Neither PR or PD; Inevaluable for response: specify reasons (for example: early death, malignant disease, toxicity; tumour assessments not repeated/incomplete; other). Normally, all eligible patients should be included in the denominator for the calculation of the response rate for phase II trials.

Time frame: From the start of treatment to the date of progression or the start of a subsequent therapy or end of patient's follow-up, until Cycle 6 (21-day cycle)

Population: Treated patients with disease measurement

ArmMeasureCategoryValue (COUNT_OF_PARTICIPANTS)
Biliary Tract Carcinoma CohortResponse by Investigator AssessmentProgressive disease11 Participants
Biliary Tract Carcinoma CohortResponse by Investigator AssessmentComplete Response0 Participants
Biliary Tract Carcinoma CohortResponse by Investigator AssessmentStable Disease5 Participants
Biliary Tract Carcinoma CohortResponse by Investigator AssessmentPartial Response1 Participants
Biliary Tract Carcinoma CohortResponse by Investigator AssessmentInevaluable for response1 Participants
Carcinoma of Unknown Primary Site CohortResponse by Investigator AssessmentStable Disease11 Participants
Carcinoma of Unknown Primary Site CohortResponse by Investigator AssessmentComplete Response0 Participants
Carcinoma of Unknown Primary Site CohortResponse by Investigator AssessmentProgressive disease7 Participants
Carcinoma of Unknown Primary Site CohortResponse by Investigator AssessmentPartial Response0 Participants
Carcinoma of Unknown Primary Site CohortResponse by Investigator AssessmentInevaluable for response1 Participants
Endometrial Carcinoma CohortResponse by Investigator AssessmentInevaluable for response4 Participants
Endometrial Carcinoma CohortResponse by Investigator AssessmentStable Disease29 Participants
Endometrial Carcinoma CohortResponse by Investigator AssessmentPartial Response6 Participants
Endometrial Carcinoma CohortResponse by Investigator AssessmentComplete Response2 Participants
Endometrial Carcinoma CohortResponse by Investigator AssessmentProgressive disease30 Participants
Ewing's Family of Tumors CohortResponse by Investigator AssessmentInevaluable for response3 Participants
Ewing's Family of Tumors CohortResponse by Investigator AssessmentComplete Response0 Participants
Ewing's Family of Tumors CohortResponse by Investigator AssessmentPartial Response4 Participants
Ewing's Family of Tumors CohortResponse by Investigator AssessmentStable Disease12 Participants
Ewing's Family of Tumors CohortResponse by Investigator AssessmentProgressive disease9 Participants
Germ Cell Tumors CohortResponse by Investigator AssessmentInevaluable for response2 Participants
Germ Cell Tumors CohortResponse by Investigator AssessmentComplete Response0 Participants
Germ Cell Tumors CohortResponse by Investigator AssessmentProgressive disease12 Participants
Germ Cell Tumors CohortResponse by Investigator AssessmentStable Disease8 Participants
Germ Cell Tumors CohortResponse by Investigator AssessmentPartial Response1 Participants
Head and Neck Carcinoma CohortResponse by Investigator AssessmentPartial Response0 Participants
Head and Neck Carcinoma CohortResponse by Investigator AssessmentStable Disease3 Participants
Head and Neck Carcinoma CohortResponse by Investigator AssessmentComplete Response0 Participants
Head and Neck Carcinoma CohortResponse by Investigator AssessmentInevaluable for response2 Participants
Head and Neck Carcinoma CohortResponse by Investigator AssessmentProgressive disease8 Participants
BRCA1/2-associated Metastatic Breast Carcinoma CohortResponse by Investigator AssessmentComplete Response0 Participants
BRCA1/2-associated Metastatic Breast Carcinoma CohortResponse by Investigator AssessmentProgressive disease5 Participants
BRCA1/2-associated Metastatic Breast Carcinoma CohortResponse by Investigator AssessmentPartial Response6 Participants
BRCA1/2-associated Metastatic Breast Carcinoma CohortResponse by Investigator AssessmentInevaluable for response0 Participants
BRCA1/2-associated Metastatic Breast Carcinoma CohortResponse by Investigator AssessmentStable Disease10 Participants
Neuroendocrine Tumors CohortResponse by Investigator AssessmentProgressive disease18 Participants
Neuroendocrine Tumors CohortResponse by Investigator AssessmentComplete Response0 Participants
Neuroendocrine Tumors CohortResponse by Investigator AssessmentPartial Response2 Participants
Neuroendocrine Tumors CohortResponse by Investigator AssessmentStable Disease9 Participants
Neuroendocrine Tumors CohortResponse by Investigator AssessmentInevaluable for response2 Participants
Small Cell Lung Cancer CohortResponse by Investigator AssessmentComplete Response0 Participants
Small Cell Lung Cancer CohortResponse by Investigator AssessmentStable Disease34 Participants
Small Cell Lung Cancer CohortResponse by Investigator AssessmentPartial Response38 Participants
Small Cell Lung Cancer CohortResponse by Investigator AssessmentInevaluable for response5 Participants
Small Cell Lung Cancer CohortResponse by Investigator AssessmentProgressive disease28 Participants
Secondary

Clinical Benefit

Clinical Benefit Rate was defined as Overall Response Rate or Stable Disease lasting over four months (SD ≥ 4 months)

Time frame: From the start of treatment to the date of progression or the start of a subsequent therapy or end of patient's follow-up, until Cycle 6 (21-day cycle)

Population: Treated patients with disease measurement

ArmMeasureValue (NUMBER)
Biliary Tract Carcinoma CohortClinical Benefit11.1 percentage of participants
Carcinoma of Unknown Primary Site CohortClinical Benefit36.8 percentage of participants
Endometrial Carcinoma CohortClinical Benefit35.2 percentage of participants
Ewing's Family of Tumors CohortClinical Benefit39.3 percentage of participants
Germ Cell Tumors CohortClinical Benefit26.1 percentage of participants
Head and Neck Carcinoma CohortClinical Benefit15.4 percentage of participants
BRCA1/2-associated Metastatic Breast Carcinoma CohortClinical Benefit57.1 percentage of participants
Neuroendocrine Tumors CohortClinical Benefit29.0 percentage of participants
Small Cell Lung Cancer CohortClinical Benefit45.7 percentage of participants
Secondary

Disease Control Rate

Disease Control Rate was defined as Overall Response Rate or Stable Disease

Time frame: From the start of treatment to the date of progression or the start of a subsequent therapy or end of patient's follow-up, until Cycle 6

Population: Treated patients with disease measurement

ArmMeasureValue (NUMBER)
Biliary Tract Carcinoma CohortDisease Control Rate33.3 percentage of paticipants
Carcinoma of Unknown Primary Site CohortDisease Control Rate57.9 percentage of paticipants
Endometrial Carcinoma CohortDisease Control Rate52.1 percentage of paticipants
Ewing's Family of Tumors CohortDisease Control Rate57.1 percentage of paticipants
Germ Cell Tumors CohortDisease Control Rate39.1 percentage of paticipants
Head and Neck Carcinoma CohortDisease Control Rate23.1 percentage of paticipants
BRCA1/2-associated Metastatic Breast Carcinoma CohortDisease Control Rate76.2 percentage of paticipants
Neuroendocrine Tumors CohortDisease Control Rate35.5 percentage of paticipants
Small Cell Lung Cancer CohortDisease Control Rate68.6 percentage of paticipants
Secondary

Duration of Response

Duration of Response by Investigator's Assessment, defined as the time between the date when the response criteria (PR or CR, whichever one is first reached) are fulfilled to the first date when disease progression (PD), recurrence or death is documented.

Time frame: From the start of treatment to the date of progression or the start of a subsequent therapy or end of patient's follow-up, until Cycle 6 (21-day cycle)

Population: Patients with response to treatment

ArmMeasureValue (MEDIAN)
Biliary Tract Carcinoma CohortDuration of Response3.4 months
Endometrial Carcinoma CohortDuration of Response9.2 months
Ewing's Family of Tumors CohortDuration of Response4.2 months
Germ Cell Tumors CohortDuration of Response10.6 months
BRCA1/2-associated Metastatic Breast Carcinoma CohortDuration of Response8.6 months
Neuroendocrine Tumors CohortDuration of Response4.7 months
Small Cell Lung Cancer CohortDuration of Response5.3 months
Secondary

Overall Survival at 12 Months

Overall Survival at 12 months defined as the probability of being alive after the first infusion at 12 months

Time frame: At 12 months

Population: Treated patients with disease measurement

ArmMeasureValue (NUMBER)
Biliary Tract Carcinoma CohortOverall Survival at 12 Months21.8 percentage of participants
Carcinoma of Unknown Primary Site CohortOverall Survival at 12 Months36.5 percentage of participants
Endometrial Carcinoma CohortOverall Survival at 12 Months45.8 percentage of participants
Ewing's Family of Tumors CohortOverall Survival at 12 Months48.5 percentage of participants
Germ Cell Tumors CohortOverall Survival at 12 Months34.4 percentage of participants
Head and Neck Carcinoma CohortOverall Survival at 12 Months30.8 percentage of participants
BRCA1/2-associated Metastatic Breast Carcinoma CohortOverall Survival at 12 Months58.1 percentage of participants
Neuroendocrine Tumors CohortOverall Survival at 12 Months38.2 percentage of participants
Small Cell Lung Cancer CohortOverall Survival at 12 Months34.8 percentage of participants
Secondary

Overall Survival at 6 Months

Overall Survival at 6 months defined as the probability of being alive after the first infusion at 6 months

Time frame: At 6 months

Population: Treated patients with disease measurement

ArmMeasureValue (NUMBER)
Biliary Tract Carcinoma CohortOverall Survival at 6 Months58.2 percentage of participants
Carcinoma of Unknown Primary Site CohortOverall Survival at 6 Months55.6 percentage of participants
Endometrial Carcinoma CohortOverall Survival at 6 Months62.8 percentage of participants
Ewing's Family of Tumors CohortOverall Survival at 6 Months88.2 percentage of participants
Germ Cell Tumors CohortOverall Survival at 6 Months55.0 percentage of participants
Head and Neck Carcinoma CohortOverall Survival at 6 Months38.5 percentage of participants
BRCA1/2-associated Metastatic Breast Carcinoma CohortOverall Survival at 6 Months79.2 percentage of participants
Neuroendocrine Tumors CohortOverall Survival at 6 Months52.1 percentage of participants
Small Cell Lung Cancer CohortOverall Survival at 6 Months68.6 percentage of participants
Secondary

Overall Survival (OS)

Overall survival defined as the period of time from the date of first infusion to the date of death or last contact in case of patients lost to follow-up or alive at the clinical cutoff established for the cohort.

Time frame: From the date of first infusion to the date of death or last contact, up to an average of 5 years

Population: Treated patients with disease measurement

ArmMeasureValue (MEDIAN)
Biliary Tract Carcinoma CohortOverall Survival (OS)7.3 months
Carcinoma of Unknown Primary Site CohortOverall Survival (OS)7.7 months
Endometrial Carcinoma CohortOverall Survival (OS)9.3 months
Ewing's Family of Tumors CohortOverall Survival (OS)12.0 months
Germ Cell Tumors CohortOverall Survival (OS)9.2 months
Head and Neck Carcinoma CohortOverall Survival (OS)5.7 months
BRCA1/2-associated Metastatic Breast Carcinoma CohortOverall Survival (OS)16.1 months
Neuroendocrine Tumors CohortOverall Survival (OS)7.4 months
Small Cell Lung Cancer CohortOverall Survival (OS)8.1 months
Secondary

Progression-free Survival at 4 Months

Progression-free Survival at 4, defined as the probability of being free from progression and death after the first infusion at 4 months. Progressive disease: ≥20% increase in the sum of the longest diameter of target lesions compared with the smallest-sum longest

Time frame: At 4 months

Population: Treated patients with disease measurement

ArmMeasureValue (NUMBER)
Biliary Tract Carcinoma CohortProgression-free Survival at 4 Months13.7 percentage of participants
Carcinoma of Unknown Primary Site CohortProgression-free Survival at 4 Months38.9 percentage of participants
Endometrial Carcinoma CohortProgression-free Survival at 4 Months39.7 percentage of participants
Ewing's Family of Tumors CohortProgression-free Survival at 4 Months46.2 percentage of participants
Germ Cell Tumors CohortProgression-free Survival at 4 Months30.7 percentage of participants
Head and Neck Carcinoma CohortProgression-free Survival at 4 Months15.4 percentage of participants
BRCA1/2-associated Metastatic Breast Carcinoma CohortProgression-free Survival at 4 Months57.1 percentage of participants
Neuroendocrine Tumors CohortProgression-free Survival at 4 Months30.0 percentage of participants
Small Cell Lung Cancer CohortProgression-free Survival at 4 Months46.5 percentage of participants
Secondary

Progression-free Survival at 6 Months

Progression-free Survival at 6, defined as the probability of being free from progression and death after the first infusion at 6 months. Progressive disease: ≥20% increase in the sum of the longest diameter of target lesions compared with the smallest-sum longest

Time frame: At 6 months

Population: Treated patients with disease measurement

ArmMeasureValue (NUMBER)
Biliary Tract Carcinoma CohortProgression-free Survival at 6 Months13.7 percentage of participants
Carcinoma of Unknown Primary Site CohortProgression-free Survival at 6 Months22.2 percentage of participants
Endometrial Carcinoma CohortProgression-free Survival at 6 Months29.0 percentage of participants
Ewing's Family of Tumors CohortProgression-free Survival at 6 Months23.1 percentage of participants
Germ Cell Tumors CohortProgression-free Survival at 6 Months30.7 percentage of participants
Head and Neck Carcinoma CohortProgression-free Survival at 6 Months7.7 percentage of participants
BRCA1/2-associated Metastatic Breast Carcinoma CohortProgression-free Survival at 6 Months33.3 percentage of participants
Neuroendocrine Tumors CohortProgression-free Survival at 6 Months16.7 percentage of participants
Small Cell Lung Cancer CohortProgression-free Survival at 6 Months33.4 percentage of participants
Secondary

Progression Free Survival (PFS)

Progression-free Survival (PFS), defined as the period of time from the date of first infusion to the date of PD, death (of any cause), or last tumor evaluation. Progressive disease: ≥20% increase in the sum of the longest diameter of target lesions compared with the smallest-sum longest

Time frame: From the date of first infusion to the date of progression disease, death (of any cause), or last tumor evaluation, up to an average of 5 years

Population: Treated patients with disease measurement

ArmMeasureValue (MEDIAN)
Biliary Tract Carcinoma CohortProgression Free Survival (PFS)1.3 months
Carcinoma of Unknown Primary Site CohortProgression Free Survival (PFS)2.7 months
Endometrial Carcinoma CohortProgression Free Survival (PFS)2.6 months
Ewing's Family of Tumors CohortProgression Free Survival (PFS)2.7 months
Germ Cell Tumors CohortProgression Free Survival (PFS)1.5 months
Head and Neck Carcinoma CohortProgression Free Survival (PFS)1.3 months
BRCA1/2-associated Metastatic Breast Carcinoma CohortProgression Free Survival (PFS)4.1 months
Neuroendocrine Tumors CohortProgression Free Survival (PFS)1.4 months
Small Cell Lung Cancer CohortProgression Free Survival (PFS)3.7 months

Source: ClinicalTrials.gov · Data processed: Feb 27, 2026