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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02454972
Recruitment Status : Completed
First Posted : May 27, 2015
Results First Posted : November 22, 2021
Last Update Posted : March 2, 2023
Sponsor:
Information provided by (Responsible Party):
PharmaMar

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Advanced Solid Tumors
Intervention Drug: lurbinectedin (PM01183)
Enrollment 345
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.
Pre-assignment Details  
Arm/Group Title Biliary Tract Carcinoma Cohort Carcinoma of Unknown Primary Site Cohort Endometrial Carcinoma Cohort Ewing's Family of Tumors Cohort Germ Cell Tumors Cohort Head and Neck Carcinoma Cohort BRCA1/2-associated Metastatic Breast Carcinoma Cohort Neuroendocrine Tumors Cohort Small Cell Lung Cancer Cohort
Hide Arm/Group Description

Patients with Pathologically proven diagnosis of biliary tract carcinoma

Lurbinectedin was administered over a minimum total volume of 100 mL of solution for infusion (either on 5% glucose or 0.9% sodium chloride), through a central catheter, or over a minimum total volume of 250 mL if administered through a peripheral line, always over one hour at a fixed infusion rate. Starting dose was 3.2 mg/m^2. Dose was capped at body surface area (BSA) of 2.0 m^2 (i.e., dose did not exceed 6.4 mg).

Patients received lurbinectedin intravenously (i.v.) as a one-hour infusion on Day 1 q3wk (three weeks = one treatment cycle).

Patients with pathologically proven diagnosis of carcinoma of unknown primary site

Lurbinectedin was administered over a minimum total volume of 100 mL of solution for infusion (either on 5% glucose or 0.9% sodium chloride), through a central catheter, or over a minimum total volume of 250 mL if administered through a peripheral line, always over one hour at a fixed infusion rate. Starting dose was 3.2 mg/m^2. Dose was capped at body surface area (BSA) of 2.0 m^2 (i.e., dose did not exceed 6.4 mg).

Patients received lurbinectedin intravenously (i.v.) as a one-hour infusion on Day 1 q3wk (three weeks = one treatment cycle).

Patients with pathologically proven diagnosis of endometrial carcinoma

Lurbinectedin was administered over a minimum total volume of 100 mL of solution for infusion (either on 5% glucose or 0.9% sodium chloride), through a central catheter, or over a minimum total volume of 250 mL if administered through a peripheral line, always over one hour at a fixed infusion rate. Starting dose was 3.2 mg/m^2. Dose was capped at body surface area (BSA) of 2.0 m^2 (i.e., dose did not exceed 6.4 mg).

Patients received lurbinectedin intravenously (i.v.) as a one-hour infusion on Day 1 q3wk (three weeks = one treatment cycle).

Patients with pathologically proven diagnosis of Ewing's Family of Tumors

Lurbinectedin was administered over a minimum total volume of 100 mL of solution for infusion (either on 5% glucose or 0.9% sodium chloride), through a central catheter, or over a minimum total volume of 250 mL if administered through a peripheral line, always over one hour at a fixed infusion rate. Starting dose was 3.2 mg/m^2. Dose was capped at body surface area (BSA) of 2.0 m^2 (i.e., dose did not exceed 6.4 mg).

Patients received lurbinectedin intravenously (i.v.) as a one-hour infusion on Day 1 q3wk (three weeks = one treatment cycle).

Patients with pathologically proven diagnosis of Germ Cell Tumors, excluding immature teratoma, or teratoma with malignant transformation.

Lurbinectedin was administered over a minimum total volume of 100 mL of solution for infusion (either on 5% glucose or 0.9% sodium chloride), through a central catheter, or over a minimum total volume of 250 mL if administered through a peripheral line, always over one hour at a fixed infusion rate. Starting dose was 3.2 mg/m^2. Dose was capped at body surface area (BSA) of 2.0 m^2 (i.e., dose did not exceed 6.4 mg).

Patients received lurbinectedin intravenously (i.v.) as a one-hour infusion on Day 1 q3wk (three weeks = one treatment cycle).

Patients with Pathologically proven diagnosis of Head and Neck Carcinoma. Salivary glands tumors were excluded.

Lurbinectedin was administered over a minimum total volume of 100 mL of solution for infusion (either on 5% glucose or 0.9% sodium chloride), through a central catheter, or over a minimum total volume of 250 mL if administered through a peripheral line, always over one hour at a fixed infusion rate. Starting dose was 3.2 mg/m^2. Dose was capped at body surface area (BSA) of 2.0 m^2 (i.e., dose did not exceed 6.4 mg).

Patients received lurbinectedin intravenously (i.v.) as a one-hour infusion on Day 1 q3wk (three weeks = one treatment cycle).

Patients with pathologically proven diagnosis of BRCA1/2-associated metastatic breast carcinoma

Lurbinectedin was administered over a minimum total volume of 100 mL of solution for infusion (either on 5% glucose or 0.9% sodium chloride), through a central catheter, or over a minimum total volume of 250 mL if administered through a peripheral line, always over one hour at a fixed infusion rate. Starting dose was 3.2 mg/m^2. Dose was capped at body surface area (BSA) of 2.0 m^2 (i.e., dose did not exceed 6.4 mg).

Patients received lurbinectedin intravenously (i.v.) as a one-hour infusion on Day 1 q3wk (three weeks = one treatment cycle).

Patients with Pathologically proven diagnosis of Neuroendocrine Tumors, grade 2 and 3 according to World Health Organization (WHO) classification.

Lurbinectedin was administered over a minimum total volume of 100 mL of solution for infusion (either on 5% glucose or 0.9% sodium chloride), through a central catheter, or over a minimum total volume of 250 mL if administered through a peripheral line, always over one hour at a fixed infusion rate. Starting dose was 3.2 mg/m^2. Dose was capped at body surface area (BSA) of 2.0 m^2 (i.e., dose did not exceed 6.4 mg).

Patients received lurbinectedin intravenously (i.v.) as a one-hour infusion on Day 1 q3wk (three weeks = one treatment cycle).

Patients with pathologically proven diagnosis of small cell lung cancer

Lurbinectedin was administered over a minimum total volume of 100 mL of solution for infusion (either on 5% glucose or 0.9% sodium chloride), through a central catheter, or over a minimum total volume of 250 mL if administered through a peripheral line, always over one hour at a fixed infusion rate. Starting dose was 3.2 mg/m^2. Dose was capped at body surface area (BSA) of 2.0 m^2 (i.e., dose did not exceed 6.4 mg).

Patients received lurbinectedin intravenously (i.v.) as a one-hour infusion on Day 1 q3wk (three weeks = one treatment cycle).
Period Title: Overall Study
Started 19 19 76 29 24 15 21 32 110
Treated 19 19 73 28 23 15 21 32 105
Completed 0 0 0 0 0 0 0 0 0
Not Completed 19 19 76 29 24 15 21 32 110
Reason Not Completed
Progressive disease             17             16             59             23             16             12             19             27             0
Death             0             0             5             1             0             0             0             0             95
Physician Decision             0             1             2             1             2             0             0             1             0
Non treatment-related adverse event             2             0             1             0             0             1             0             1             0
Treatment-related adverse events             0             1             1             0             2             0             0             2             0
Patient moves to compassionate use             0             0             0             0             0             0             2             0             0
Withdrawal by Subject             0             1             5             2             3             2             0             1             2
Multiple delay/holds on treatment             0             0             0             1             0             0             0             0             0
Never treated             0             0             3             1             1             0             0             0             5
Lost to Follow-up             0             0             0             0             0             0             0             0             2
Study termination             0             0             0             0             0             0             0             0             6
Arm/Group Title Biliary Tract Carcinoma Cohort Carcinoma of Unknown Primary Site Cohort Endometrial Carcinoma Cohort Ewing's Family of Tumors Cohort Germ Cell Tumors Cohort Head and Neck Carcinoma Cohort BRCA1/2-associated Metastatic Breast Carcinoma Cohort Neuroendocrine Tumors Cohort Small Cell Lung Cancer Cohort Total
Hide Arm/Group Description Patients with Pathologically proven diagnosis of biliary tract carcinoma Patients with pathologically proven diagnosis of carcinoma of unknown primary site Patients with pathologically proven diagnosis of endometrial carcinoma Patients with pathologically proven diagnosis of Ewing's Family of Tumors Patients with pathologically proven diagnosis of Germ Cell Tumors, excluding immature teratoma, or teratoma with malignant transformation. Patients with Pathologically proven diagnosis of Head and Neck Carcinoma. Salivary glands tumors were excluded. Patients with pathologically proven diagnosis of BRCA1/2-associated metastatic breast carcinoma Patients with Pathologically proven diagnosis of Neuroendocrine Tumors, grade 2 and 3 according to World Health Organization (WHO) classification. Patients with pathologically proven diagnosis of small cell lung cancer Total of all reporting groups
Overall Number of Baseline Participants 19 19 73 28 23 15 21 32 105 335
Hide Baseline Analysis Population Description
Treated patients only
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
13
  68.4%
10
  52.6%
37
  50.7%
27
  96.4%
20
  87.0%
9
  60.0%
19
  90.5%
19
  59.4%
68
  64.8%
222
  66.3%
>=65 years
6
  31.6%
9
  47.4%
36
  49.3%
1
   3.6%
3
  13.0%
6
  40.0%
2
   9.5%
13
  40.6%
37
  35.2%
113
  33.7%
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
61
(34 to 74)
61
(27 to 78)
64
(32 to 80)
33
(18 to 74)
36
(21 to 73)
62
(39 to 81)
45
(29 to 73)
63
(23 to 77)
60
(40 to 83)
60
(18 to 83)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
Female
10
  52.6%
11
  57.9%
73
 100.0%
12
  42.9%
7
  30.4%
1
   6.7%
21
 100.0%
12
  37.5%
42
  40.0%
189
  56.4%
Male
9
  47.4%
8
  42.1%
0
   0.0%
16
  57.1%
16
  69.6%
14
  93.3%
0
   0.0%
20
  62.5%
63
  60.0%
146
  43.6%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
White
13
  68.4%
15
  78.9%
45
  61.6%
21
  75.0%
15
  65.2%
12
  80.0%
18
  85.7%
24
  75.0%
79
  75.2%
242
  72.2%
Black of African American
0
   0.0%
0
   0.0%
5
   6.8%
1
   3.6%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1
   1.0%
7
   2.1%
Asian
0
   0.0%
0
   0.0%
1
   1.4%
2
   7.1%
1
   4.3%
1
   6.7%
0
   0.0%
0
   0.0%
1
   1.0%
6
   1.8%
American Indian or Alaska native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1
   3.1%
0
   0.0%
1
   0.3%
Not race available
6
  31.6%
4
  21.1%
22
  30.1%
4
  14.3%
7
  30.4%
2
  13.3%
3
  14.3%
7
  21.9%
24
  22.9%
79
  23.6%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
 Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
Sweden
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1
   3.1%
0
   0.0%
1
   0.3%
Belgium
2
  10.5%
2
  10.5%
3
   4.1%
1
   3.6%
0
   0.0%
0
   0.0%
0
   0.0%
2
   6.3%
3
   2.9%
13
   3.9%
United States
3
  15.8%
6
  31.6%
17
  23.3%
16
  57.1%
12
  52.2%
4
  26.7%
2
   9.5%
6
  18.8%
11
  10.5%
77
  23.0%
Italy
0
   0.0%
0
   0.0%
0
   0.0%
3
  10.7%
1
   4.3%
0
   0.0%
0
   0.0%
0
   0.0%
2
   1.9%
6
   1.8%
United Kingdom
0
   0.0%
1
   5.3%
9
  12.3%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
3
   2.9%
13
   3.9%
France
4
  21.1%
2
  10.5%
17
  23.3%
3
  10.7%
6
  26.1%
2
  13.3%
3
  14.3%
5
  15.6%
20
  19.0%
62
  18.5%
Switzerland
0
   0.0%
0
   0.0%
2
   2.7%
0
   0.0%
0
   0.0%
0
   0.0%
1
   4.8%
0
   0.0%
7
   6.7%
10
   3.0%
Germany
0
   0.0%
0
   0.0%
2
   2.7%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
2
   0.6%
Spain
10
  52.6%
8
  42.1%
23
  31.5%
5
  17.9%
4
  17.4%
9
  60.0%
15
  71.4%
18
  56.3%
59
  56.2%
151
  45.1%
Eastern Cooperative Oncology Group performance status   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
0
5
  26.3%
8
  42.1%
32
  43.8%
11
  39.3%
2
   8.7%
5
  33.3%
18
  85.7%
8
  25.0%
38
  36.2%
127
  37.9%
1
14
  73.7%
10
  52.6%
35
  47.9%
16
  57.1%
19
  82.6%
10
  66.7%
2
   9.5%
23
  71.9%
59
  56.2%
188
  56.1%
2
0
   0.0%
1
   5.3%
6
   8.2%
1
   3.6%
2
   8.7%
0
   0.0%
1
   4.8%
1
   3.1%
8
   7.6%
20
   6.0%
[1]
Measure Description:

Eastern Cooperative Oncology Group performance status 0 Fully active, able to carry on all pre-disease performance without restriction

  1. Restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature
  2. Ambulatory and capable of all selfcare but unable to carry out any work activities; up and about more than 50% of waking hours
  3. Capable of only limited selfcare; confined to bed or chair more than 50% of waking hours
  4. Completely disabled; cannot carry on any selfcare; totally confined to bed or chair
  5. Dead
Weight  
Median (Full Range)
Unit of measure:  Kg
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
72.2
(45.0 to 115.0)
65.0
(50.1 to 108.2)
70.0
(42.5 to 140.8)
77.0
(51.0 to 127.7)
78.5
(59.0 to 116.3)
73.0
(49.0 to 107.0)
65.5
(52.0 to 115.1)
64.0
(47.0 to 121.0)
71.0
(46.0 to 138.3)
71.0
(42.5 to 140.8)
Height  
Median (Full Range)
Unit of measure:  Cm
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
168
(148 to 187)
164.5
(141 to 187)
161
(143 to 180)
173
(155 to 193)
177
(157 to 190)
170
(155 to 186)
163.0
(147 to 177)
169
(150 to 190)
167
(150 to 183)
165
(141 to 193)
Body surface area  
Median (Full Range)
Unit of measure:  M^2
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
1.9
(1.4 to 2.4)
1.8
(1.5 to 2.4)
1.8
(1.3 to 2.6)
1.9
(1.6 to 2.4)
2.0
(1.7 to 2.4)
1.8
(1.5 to 2.2)
1.7
(1.5 to 2.1)
1.7
(1.4 to 2.5)
1.8
(1.4 to 2.6)
1.8
(1.3 to 2.6)
Albumin  
Median (Full Range)
Unit of measure:  g/dL
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
3.7
(2.9 to 4.7)
3.7
(3.2 to 4.4)
4.1
(2.7 to 4.7)
4.2
(3.3 to 4.9)
4.1
(3.2 to 4.8)
3.8
(3.0 to 4.4)
4.1
(3.0 to 5.0)
4.0
(3.1 to 4.6)
4.1
(3.1 to 5.1)
4.0
(2.7 to 5.1)
Albumin  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
<3.5 g/dL
5
  26.3%
4
  21.1%
9
  12.3%
2
   7.1%
4
  17.4%
3
  20.0%
1
   4.8%
9
  28.1%
13
  12.4%
50
  14.9%
≥3.5 g/dL
14
  73.7%
15
  78.9%
64
  87.7%
26
  92.9%
19
  82.6%
12
  80.0%
20
  95.2%
23
  71.9%
92
  87.6%
285
  85.1%
Stage at diagnosis  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
Early
0
   0.0%
0
   0.0%
23
  31.5%
14
  50.0%
8
  34.8%
4
  26.7%
10
  47.6%
5
  15.6%
3
   2.9%
67
  20.0%
Locally advanced
4
  21.1%
0
   0.0%
27
  37.0%
5
  17.9%
1
   4.3%
7
  46.7%
6
  28.6%
8
  25.0%
29
  27.6%
87
  26.0%
Metastatic
15
  78.9%
19
 100.0%
23
  31.5%
9
  32.1%
14
  60.9%
4
  26.7%
5
  23.8%
19
  59.4%
73
  69.5%
181
  54.0%
Number of sites at baseline  
Median (Full Range)
Unit of measure:  Sites at baseline
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
3
(2 to 6)
2
(1 to 4)
2
(1 to 7)
2
(1 to 6)
3
(1 to 4)
2
(1 to 6)
2
(1 to 4)
3
(1 to 7)
3
(1 to 6)
3
(1 to 7)
Sites at baseline  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
<3 sites
6
  31.6%
12
  63.2%
40
  54.8%
18
  64.3%
7
  30.4%
10
  66.7%
12
  57.1%
13
  40.6%
26
  24.8%
144
  43.0%
≥3 sites
13
  68.4%
7
  36.8%
33
  45.2%
10
  35.7%
16
  69.6%
5
  33.3%
9
  42.9%
19
  59.4%
79
  75.2%
191
  57.0%
Time from diagnosis to registration  
Median (Full Range)
Unit of measure:  Months
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
8.4
(3.8 to 23.0)
10.8
(4.6 to 62.9)
18.4
(4.3 to 190.9)
28.6
(6.9 to 140.5)
48.2
(7.3 to 308.8)
19.5
(2.9 to 426.1)
50.1
(16.2 to 236.0)
13.3
(3.0 to 93.2)
8.2
(2.1 to 20.0)
13.7
(2.1 to 426.1)
Time from advanced disease to registration  
Median (Full Range)
Unit of measure:  Month
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
10.6
(6.3 to 23.0)
NA [1] 
(NA to NA)
17.5
(0.9 to 97.0)
20.4
(0.4 to 54.2)
33.5
(10.7 to 86.8)
18.4
(9.7 to 72.7)
31.9
(14.1 to 115.9)
17.2
(3.8 to 93.2)
9.1
(0.3 to 20.0)
15.0
(0.3 to 115.9)
[1]
All patients were metastatic
Prior surgery  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
2
  10.5%
2
  10.5%
62
  84.9%
20
  71.4%
21
  91.3%
9
  60.0%
19
  90.5%
11
  34.4%
2
   1.9%
148
  44.2%
Prior radiotherapy  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
2
  10.5%
8
  42.1%
39
  53.4%
20
  71.4%
7
  30.4%
11
  73.3%
20
  95.2%
7
  21.9%
76
  72.4%
190
  56.7%
Systemic lines  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
1 line
13
  68.4%
12
  63.2%
54
  74.0%
5
  17.9%
0
   0.0%
5
  33.3%
0
   0.0%
14
  43.8%
98
  93.3%
201
  60.0%
2 lines
6
  31.6%
7
  36.8%
15
  20.5%
15
  53.6%
4
  17.4%
8
  53.3%
7
  33.3%
13
  40.6%
7
   6.7%
82
  24.5%
3 lines
0
   0.0%
0
   0.0%
3
   4.1%
5
  17.9%
8
  34.8%
2
  13.3%
7
  33.3%
4
  12.5%
0
   0.0%
29
   8.7%
4 or more lines
0
   0.0%
0
   0.0%
1
   1.4%
3
  10.7%
11
  47.8%
0
   0.0%
7
  33.3%
1
   3.1%
0
   0.0%
23
   6.9%
Advanced chemotherapy lines  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
0 lines
19
 100.0%
0
   0.0%
15
  20.5%
5
  17.9%
0
   0.0%
0
   0.0%
1
   4.8%
0
   0.0%
105
 100.0%
145
  43.3%
1 line
0
   0.0%
12
  63.2%
47
  64.4%
7
  25.0%
1
   4.3%
5
  33.3%
5
  23.8%
17
  53.1%
0
   0.0%
94
  28.1%
2 lines
0
   0.0%
7
  36.8%
8
  11.0%
13
  46.4%
4
  17.4%
9
  60.0%
7
  33.3%
10
  31.3%
0
   0.0%
58
  17.3%
3 lines
0
   0.0%
0
   0.0%
2
   2.7%
3
  10.7%
8
  34.8%
1
   6.7%
6
  28.6%
4
  12.5%
0
   0.0%
24
   7.2%
4 or more lines
0
   0.0%
0
   0.0%
1
   1.4%
0
   0.0%
10
  43.5%
0
   0.0%
2
   9.5%
1
   3.1%
0
   0.0%
14
   4.2%
Best response to last therapy   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
Complete response
0
   0.0%
3
  15.8%
7
   9.6%
0
   0.0%
0
   0.0%
0
   0.0%
1
   4.8%
0
   0.0%
9
   8.6%
20
   6.0%
Partial reponse
4
  21.1%
4
  21.1%
20
  27.4%
0
   0.0%
2
   8.7%
5
  33.3%
2
   9.5%
5
  15.6%
70
  66.7%
112
  33.4%
Stable disease
7
  36.8%
5
  26.3%
13
  17.8%
0
   0.0%
5
  21.7%
2
  13.3%
6
  28.6%
14
  43.8%
19
  18.1%
71
  21.2%
Progression disease
8
  42.1%
4
  21.1%
15
  20.5%
0
   0.0%
15
  65.2%
7
  46.7%
7
  33.3%
11
  34.4%
4
   3.8%
71
  21.2%
Unknown
0
   0.0%
3
  15.8%
18
  24.7%
28
 100.0%
1
   4.3%
1
   6.7%
5
  23.8%
2
   6.3%
3
   2.9%
61
  18.2%
[1]
Measure Description: According to the RECIST v.1.1, Complete Response: 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 (PD): ≥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 to progression to last prior therapy  
Median (Full Range)
Unit of measure:  Months
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
5.2
(1.0 to 14.0)
3.9
(0.9 to 22.9)
8.0
(1.4 to 23.5)
8.7
(0.4 to 25.8)
2.7
(0.8 to 29.6)
4.6
(1.4 to 26.1)
5.0
(1.3 to 32.1)
3.6
(1.1 to 24.0)
6.5
(1.4 to 17.8)
6.4
(0.4 to 32.1)
Time from last progression disease before study entry  
Median (Full Range)
Unit of measure:  Weeks
Number Analyzed 19 participants 19 participants 73 participants 28 participants 23 participants 15 participants 21 participants 32 participants 105 participants 335 participants
3.0
(0.3 to 6.7)
2.6
(0.1 to 33.7)
2.9
(0.0 to 24.6)
2.1
(0.0 to 7.1)
1.4
(0.3 to 6.4)
2.4
(0.0 to 24.7)
1.6
(0.1 to 7.6)
2.6
(0.6 to 21.9)
1.6
(0.0 to 10.0)
2.1
(0.0 to 33.7)
1.Primary Outcome
Title Overall Response Rate (ORR)
Hide Description 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)
Hide Outcome Measure Data
Hide Analysis Population Description
Treated patients and with disease measurement
Arm/Group Title Biliary Tract Carcinoma Cohort Carcinoma of Unknown Primary Site Cohort Endometrial Carcinoma Cohort Ewing's Family of Tumors Cohort Germ Cell Tumors Cohort Head and Neck Carcinoma Cohort BRCA1/2-associated Metastatic Breast Carcinoma Cohort Neuroendocrine Tumors Cohort Small Cell Lung Cancer Cohort
Hide Arm/Group Description:
Patients with Pathologically proven diagnosis of biliary tract carcinoma
Patients with pathologically proven diagnosis of carcinoma of unknown primary site
Patients with pathologically proven diagnosis of endometrial carcinoma
Patients with pathologically proven diagnosis of Ewing's Family of Tumors
Patients with pathologically proven diagnosis of Germ Cell Tumors, excluding immature teratoma, or teratoma with malignant transformation.
Patients with Pathologically proven diagnosis of Head and Neck Carcinoma. Salivary glands tumors were excluded.
Patients with pathologically proven diagnosis of BRCA1/2-associated metastatic breast carcinoma
Patients with Pathologically proven diagnosis of Neuroendocrine Tumors, grade 2 and 3 according to World Health Organization (WHO) classification.
Patients with pathologically proven diagnosis of small cell lung cancer
Overall Number of Participants Analyzed 18 19 71 28 23 13 21 31 105
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
5.6
(0.1 to 27.3)
0.0
(0.0 to 17.6)
11.3
(5.0 to 21.0)
14.3
(4.0 to 32.7)
4.3
(0.1 to 21.9)
0.0
(0.0 to 24.7)
28.6
(11.3 to 55.2)
6.5
(0.8 to 21.4)
36.2
(27.0 to 46.1)
2.Primary Outcome
Title Response by Investigator Assessment
Hide Description

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)
Hide Outcome Measure Data
Hide Analysis Population Description
Treated patients with disease measurement
Arm/Group Title Biliary Tract Carcinoma Cohort Carcinoma of Unknown Primary Site Cohort Endometrial Carcinoma Cohort Ewing's Family of Tumors Cohort Germ Cell Tumors Cohort Head and Neck Carcinoma Cohort BRCA1/2-associated Metastatic Breast Carcinoma Cohort Neuroendocrine Tumors Cohort Small Cell Lung Cancer Cohort
Hide Arm/Group Description:
Patients with Pathologically proven diagnosis of biliary tract carcinoma
Patients with pathologically proven diagnosis of carcinoma of unknown primary site
Patients with pathologically proven diagnosis of endometrial carcinoma
Patients with pathologically proven diagnosis of Ewing's Family of Tumors
Patients with pathologically proven diagnosis of Germ Cell Tumors, excluding immature teratoma, or teratoma with malignant transformation.
Patients with Pathologically proven diagnosis of Head and Neck Carcinoma. Salivary glands tumors were excluded.
Patients with pathologically proven diagnosis of BRCA1/2-associated metastatic breast carcinoma
Patients with Pathologically proven diagnosis of Neuroendocrine Tumors, grade 2 and 3 according to World Health Organization (WHO) classification.
Patients with pathologically proven diagnosis of small cell lung cancer
Overall Number of Participants Analyzed 18 19 71 28 23 13 21 31 105
Measure Type: Count of Participants
Unit of Measure: Participants
Complete Response
0
   0.0%
0
   0.0%
2
   2.8%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Partial Response
1
   5.6%
0
   0.0%
6
   8.5%
4
  14.3%
1
   4.3%
0
   0.0%
6
  28.6%
2
   6.5%
38
  36.2%
Stable Disease
5
  27.8%
11
  57.9%
29
  40.8%
12
  42.9%
8
  34.8%
3
  23.1%
10
  47.6%
9
  29.0%
34
  32.4%
Progressive disease
11
  61.1%
7
  36.8%
30
  42.3%
9
  32.1%
12
  52.2%
8
  61.5%
5
  23.8%
18
  58.1%
28
  26.7%
Inevaluable for response
1
   5.6%
1
   5.3%
4
   5.6%
3
  10.7%
2
   8.7%
2
  15.4%
0
   0.0%
2
   6.5%
5
   4.8%
3.Secondary Outcome
Title Duration of Response
Hide Description 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)
Hide Outcome Measure Data
Hide Analysis Population Description
Patients with response to treatment
Arm/Group Title Biliary Tract Carcinoma Cohort Carcinoma of Unknown Primary Site Cohort Endometrial Carcinoma Cohort Ewing's Family of Tumors Cohort Germ Cell Tumors Cohort Head and Neck Carcinoma Cohort BRCA1/2-associated Metastatic Breast Carcinoma Cohort Neuroendocrine Tumors Cohort Small Cell Lung Cancer Cohort
Hide Arm/Group Description:
Patients with Pathologically proven diagnosis of biliary tract carcinoma
Patients with pathologically proven diagnosis of carcinoma of unknown primary site
Patients with pathologically proven diagnosis of endometrial carcinoma
Patients with pathologically proven diagnosis of Ewing's Family of Tumors
Patients with pathologically proven diagnosis of Germ Cell Tumors, excluding immature teratoma, or teratoma with malignant transformation.
Patients with Pathologically proven diagnosis of Head and Neck Carcinoma. Salivary glands tumors were excluded.
Patients with pathologically proven diagnosis of BRCA1/2-associated metastatic breast carcinoma
Patients with Pathologically proven diagnosis of Neuroendocrine Tumors, grade 2 and 3 according to World Health Organization (WHO) classification.
Patients with pathologically proven diagnosis of small cell lung cancer
Overall Number of Participants Analyzed 1 0 8 4 1 0 6 2 38
Median (95% Confidence Interval)
Unit of Measure: months
3.4 [1] 
(NA to NA)
9.2
(3.4 to 18.0)
4.2
(2.9 to 5.5)
10.6 [1] 
(NA to NA)
8.6 [2] 
(2.9 to NA)
4.7
(4.0 to 5.4)
5.3
(4.1 to 6.2)
[1]
Only 1 patient
[2]
Not reached
4.Secondary Outcome
Title Clinical Benefit
Hide Description 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)
Hide Outcome Measure Data
Hide Analysis Population Description
Treated patients with disease measurement
Arm/Group Title Biliary Tract Carcinoma Cohort Carcinoma of Unknown Primary Site Cohort Endometrial Carcinoma Cohort Ewing's Family of Tumors Cohort Germ Cell Tumors Cohort Head and Neck Carcinoma Cohort BRCA1/2-associated Metastatic Breast Carcinoma Cohort Neuroendocrine Tumors Cohort Small Cell Lung Cancer Cohort
Hide Arm/Group Description:
Patients with Pathologically proven diagnosis of biliary tract carcinoma
Patients with pathologically proven diagnosis of carcinoma of unknown primary site
Patients with pathologically proven diagnosis of endometrial carcinoma
Patients with pathologically proven diagnosis of Ewing's Family of Tumors
Patients with pathologically proven diagnosis of Germ Cell Tumors, excluding immature teratoma, or teratoma with malignant transformation.
Patients with Pathologically proven diagnosis of Head and Neck Carcinoma. Salivary glands tumors were excluded.
Patients with pathologically proven diagnosis of BRCA1/2-associated metastatic breast carcinoma
Patients with Pathologically proven diagnosis of Neuroendocrine Tumors, grade 2 and 3 according to World Health Organization (WHO) classification.
Patients with pathologically proven diagnosis of small cell lung cancer
Overall Number of Participants Analyzed 18 19 71 28 23 13 21 31 105
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
11.1
(1.4 to 34.7)
36.8
(16.3 to 61.6)
35.2
(24.2 to 47.5)
39.3
(21.5 to 59.4)
26.1
(10.2 to 48.4)
15.4
(1.9 to 45.4)
57.1
(34.0 to 78.2)
29.0
(14.2 to 48.0)
45.7
(36.0 to 55.7)
5.Secondary Outcome
Title Disease Control Rate
Hide Description 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
Hide Outcome Measure Data
Hide Analysis Population Description
Treated patients with disease measurement
Arm/Group Title Biliary Tract Carcinoma Cohort Carcinoma of Unknown Primary Site Cohort Endometrial Carcinoma Cohort Ewing's Family of Tumors Cohort Germ Cell Tumors Cohort Head and Neck Carcinoma Cohort BRCA1/2-associated Metastatic Breast Carcinoma Cohort Neuroendocrine Tumors Cohort Small Cell Lung Cancer Cohort
Hide Arm/Group Description:
Patients with Pathologically proven diagnosis of biliary tract carcinoma
Patients with pathologically proven diagnosis of carcinoma of unknown primary site
Patients with pathologically proven diagnosis of endometrial carcinoma
Patients with pathologically proven diagnosis of Ewing's Family of Tumors
Patients with pathologically proven diagnosis of Germ Cell Tumors, excluding immature teratoma, or teratoma with malignant transformation.
Patients with Pathologically proven diagnosis of Head and Neck Carcinoma. Salivary glands tumors were excluded.
Patients with pathologically proven diagnosis of BRCA1/2-associated metastatic breast carcinoma
Patients with Pathologically proven diagnosis of Neuroendocrine Tumors, grade 2 and 3 according to World Health Organization (WHO) classification.
Patients with pathologically proven diagnosis of small cell lung cancer
Overall Number of Participants Analyzed 18 19 71 28 23 13 21 31 105
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of paticipants
33.3
(13.3 to 59.0)
57.9
(33.5 to 79.7)
52.1
(39.9 to 64.1)
57.1
(37.2 to 75.5)
39.1
(19.7 to 61.5)
23.1
(5.0 to 53.8)
76.2
(52.8 to 91.8)
35.5
(19.2 to 54.6)
68.6
(58.8 to 77.3)
6.Secondary Outcome
Title Progression Free Survival (PFS)
Hide Description

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
Hide Outcome Measure Data
Hide Analysis Population Description
Treated patients with disease measurement
Arm/Group Title Biliary Tract Carcinoma Cohort Carcinoma of Unknown Primary Site Cohort Endometrial Carcinoma Cohort Ewing's Family of Tumors Cohort Germ Cell Tumors Cohort Head and Neck Carcinoma Cohort BRCA1/2-associated Metastatic Breast Carcinoma Cohort Neuroendocrine Tumors Cohort Small Cell Lung Cancer Cohort
Hide Arm/Group Description:
Patients with Pathologically proven diagnosis of biliary tract carcinoma
Patients with pathologically proven diagnosis of carcinoma of unknown primary site
Patients with pathologically proven diagnosis of endometrial carcinoma
Patients with pathologically proven diagnosis of Ewing's Family of Tumors
Patients with pathologically proven diagnosis of Germ Cell Tumors, excluding immature teratoma, or teratoma with malignant transformation.
Patients with Pathologically proven diagnosis of Head and Neck Carcinoma. Salivary glands tumors were excluded.
Patients with pathologically proven diagnosis of BRCA1/2-associated metastatic breast carcinoma
Patients with Pathologically proven diagnosis of Neuroendocrine Tumors, grade 2 and 3 according to World Health Organization (WHO) classification.
Patients with pathologically proven diagnosis of small cell lung cancer
Overall Number of Participants Analyzed 18 19 71 28 23 13 21 31 105
Median (95% Confidence Interval)
Unit of Measure: months
1.3
(1.1 to 2.5)
2.7
(1.3 to 4.4)
2.6
(1.4 to 4.0)
2.7
(1.4 to 4.3)
1.5
(0.9 to 8.9)
1.3
(1.2 to 2.1)
4.1
(2.3 to 6.5)
1.4
(1.2 to 3.0)
3.7
(2.6 to 4.3)
7.Secondary Outcome
Title Progression-free Survival at 4 Months
Hide Description 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
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Treated patients with disease measurement
Arm/Group Title Biliary Tract Carcinoma Cohort Carcinoma of Unknown Primary Site Cohort Endometrial Carcinoma Cohort Ewing's Family of Tumors Cohort Germ Cell Tumors Cohort Head and Neck Carcinoma Cohort BRCA1/2-associated Metastatic Breast Carcinoma Cohort Neuroendocrine Tumors Cohort Small Cell Lung Cancer Cohort
Hide Arm/Group Description:
Patients with Pathologically proven diagnosis of biliary tract carcinoma
Patients with pathologically proven diagnosis of carcinoma of unknown primary site
Patients with pathologically proven diagnosis of endometrial carcinoma
Patients with pathologically proven diagnosis of Ewing's Family of Tumors
Patients with pathologically proven diagnosis of Germ Cell Tumors, excluding immature teratoma, or teratoma with malignant transformation.
Patients with Pathologically proven diagnosis of Head and Neck Carcinoma. Salivary glands tumors were excluded.
Patients with pathologically proven diagnosis of BRCA1/2-associated metastatic breast carcinoma
Patients with Pathologically proven diagnosis of Neuroendocrine Tumors, grade 2 and 3 according to World Health Organization (WHO) classification.
Patients with pathologically proven diagnosis of small cell lung cancer
Overall Number of Participants Analyzed 18 19 71 28 23 13 21 31 105
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
13.7
(0.0 to 31.1)
38.9
(16.4 to 61.4)
39.7
(28.2 to 51.3)
46.2
(27.0 to 65.3)
30.7
(11.0 to 50.4)
15.4
(0.0 to 35.0)
57.1
(36.0 to 78.3)
30.0
(13.6 to 46.4)
46.5
(36.8 to 56.3)
8.Secondary Outcome
Title Progression-free Survival at 6 Months
Hide Description 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
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Hide Analysis Population Description
Treated patients with disease measurement
Arm/Group Title Biliary Tract Carcinoma Cohort Carcinoma of Unknown Primary Site Cohort Endometrial Carcinoma Cohort Ewing's Family of Tumors Cohort Germ Cell Tumors Cohort Head and Neck Carcinoma Cohort BRCA1/2-associated Metastatic Breast Carcinoma Cohort Neuroendocrine Tumors Cohort Small Cell Lung Cancer Cohort
Hide Arm/Group Description:
Patients with Pathologically proven diagnosis of biliary tract carcinoma
Patients with pathologically proven diagnosis of carcinoma of unknown primary site
Patients with pathologically proven diagnosis of endometrial carcinoma
Patients with pathologically proven diagnosis of Ewing's Family of Tumors
Patients with pathologically proven diagnosis of Germ Cell Tumors, excluding immature teratoma, or teratoma with malignant transformation.
Patients with Pathologically proven diagnosis of Head and Neck Carcinoma. Salivary glands tumors were excluded.
Patients with pathologically proven diagnosis of BRCA1/2-associated metastatic breast carcinoma
Patients with Pathologically proven diagnosis of Neuroendocrine Tumors, grade 2 and 3 according to World Health Organization (WHO) classification.
Patients with pathologically proven diagnosis of small cell lung cancer
Overall Number of Participants Analyzed 18 19 71 28 23 13 21 31 105
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
13.7
(0.0 to 31.1)
22.2
(3.0 to 41.4)
29.0
(18.2 to 39.8)
23.1
(5.9 to 40.3)
30.7
(11.0 to 50.4)
7.7
(0.0 to 22.2)
33.3
(13.2 to 53.5)
16.7
(3.3 to 30.0)
33.4
(24.1 to 42.6)
9.Secondary Outcome
Title Overall Survival (OS)
Hide Description 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
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Hide Analysis Population Description
Treated patients with disease measurement
Arm/Group Title Biliary Tract Carcinoma Cohort Carcinoma of Unknown Primary Site Cohort Endometrial Carcinoma Cohort Ewing's Family of Tumors Cohort Germ Cell Tumors Cohort Head and Neck Carcinoma Cohort BRCA1/2-associated Metastatic Breast Carcinoma Cohort Neuroendocrine Tumors Cohort Small Cell Lung Cancer Cohort
Hide Arm/Group Description:
Patients with Pathologically proven diagnosis of biliary tract carcinoma
Patients with pathologically proven diagnosis of carcinoma of unknown primary site
Patients with pathologically proven diagnosis of endometrial carcinoma
Patients with pathologically proven diagnosis of Ewing's Family of Tumors
Patients with pathologically proven diagnosis of Germ Cell Tumors, excluding immature teratoma, or teratoma with malignant transformation.
Patients with Pathologically proven diagnosis of Head and Neck Carcinoma. Salivary glands tumors were excluded.
Patients with pathologically proven diagnosis of BRCA1/2-associated metastatic breast carcinoma
Patients with Pathologically proven diagnosis of Neuroendocrine Tumors, grade 2 and 3 according to World Health Organization (WHO) classification.
Patients with pathologically proven diagnosis of small cell lung cancer
Overall Number of Participants Analyzed 18 19 71 28 23 13 21 31 105
Median (95% Confidence Interval)
Unit of Measure: months
7.3
(2.7 to 8.9)
7.7
(3.8 to 18.8)
9.3
(6.1 to 12.8)
12.0
(8.5 to 18.5)
9.2
(2.7 to 17.4)
5.7
(2.1 to 12.1)
16.1 [1] 
(8.7 to NA)
7.4
(3.4 to 16.2)
8.1
(6.5 to 10.9)
[1]
Not reached
10.Secondary Outcome
Title Overall Survival at 6 Months
Hide Description Overall Survival at 6 months defined as the probability of being alive after the first infusion at 6 months
Time Frame At 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Treated patients with disease measurement
Arm/Group Title Biliary Tract Carcinoma Cohort Carcinoma of Unknown Primary Site Cohort Endometrial Carcinoma Cohort Ewing's Family of Tumors Cohort Germ Cell Tumors Cohort Head and Neck Carcinoma Cohort BRCA1/2-associated Metastatic Breast Carcinoma Cohort Neuroendocrine Tumors Cohort Small Cell Lung Cancer Cohort
Hide Arm/Group Description:
Patients with Pathologically proven diagnosis of biliary tract carcinoma
Patients with pathologically proven diagnosis of carcinoma of unknown primary site
Patients with pathologically proven diagnosis of endometrial carcinoma
Patients with pathologically proven diagnosis of Ewing's Family of Tumors
Patients with pathologically proven diagnosis of Germ Cell Tumors, excluding immature teratoma, or teratoma with malignant transformation.
Patients with Pathologically proven diagnosis of Head and Neck Carcinoma. Salivary glands tumors were excluded.
Patients with pathologically proven diagnosis of BRCA1/2-associated metastatic breast carcinoma
Patients with Pathologically proven diagnosis of Neuroendocrine Tumors, grade 2 and 3 according to World Health Organization (WHO) classification.
Patients with pathologically proven diagnosis of small cell lung cancer
Overall Number of Participants Analyzed 18 19 71 28 23 13 21 31 105
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
58.2
(34.5 to 82.0)
55.6
(32.6 to 78.5)
62.8
(51.2 to 74.4)
88.2
(75.7 to 100.0)
55.0
(32.8 to 77.1)
38.5
(12.0 to 64.9)
79.2
(61.0 to 97.4)
52.1
(33.9 to 70.3)
68.6
(59.5 to 77.6)
11.Secondary Outcome
Title Overall Survival at 12 Months
Hide Description Overall Survival at 12 months defined as the probability of being alive after the first infusion at 12 months
Time Frame At 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
Treated patients with disease measurement
Arm/Group Title Biliary Tract Carcinoma Cohort Carcinoma of Unknown Primary Site Cohort Endometrial Carcinoma Cohort Ewing's Family of Tumors Cohort Germ Cell Tumors Cohort Head and Neck Carcinoma Cohort BRCA1/2-associated Metastatic Breast Carcinoma Cohort Neuroendocrine Tumors Cohort Small Cell Lung Cancer Cohort
Hide Arm/Group Description:
Patients with Pathologically proven diagnosis of biliary tract carcinoma
Patients with pathologically proven diagnosis of carcinoma of unknown primary site
Patients with pathologically proven diagnosis of endometrial carcinoma
Patients with pathologically proven diagnosis of Ewing's Family of Tumors
Patients with pathologically proven diagnosis of Germ Cell Tumors, excluding immature teratoma, or teratoma with malignant transformation.
Patients with Pathologically proven diagnosis of Head and Neck Carcinoma. Salivary glands tumors were excluded.
Patients with pathologically proven diagnosis of BRCA1/2-associated metastatic breast carcinoma
Patients with Pathologically proven diagnosis of Neuroendocrine Tumors, grade 2 and 3 according to World Health Organization (WHO) classification.
Patients with pathologically proven diagnosis of small cell lung cancer
Overall Number of Participants Analyzed 18 19 71 28 23 13 21 31 105
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
21.8
(0.4 to 43.3)
36.5
(13.1 to 59.8)
45.8
(33.8 to 75.9)
48.5
(27.8 to 69.2)
34.4
(11.3 to 57.4)
30.8
(5.7 to 55.9)
58.1
(35.9 to 80.2)
38.2
(20.5 to 55.9)
34.8
(25.5 to 44.1)
Time Frame From the date of first infusion to the date of death or last contact, up to an average of 5 years
Adverse Event Reporting Description

Safety in the entire trial population is a secondary objective defined in the protocol as all patients were exposed to the same unique dose of lurbinectedin. Consequently, adverse events are reported in a combined mode for all groups (cohorts) to provide a clear picture of the drug safety in the whole population.

Out of the 345 patients enrolled there were 10 patients who did not receive lurbinectedin treatment so patients at risk is 335 (345-10)
 
Arm/Group Title Lurbinectedin (PM01183)
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Lurbinectedin was administered over a minimum total volume of 100 mL of solution for infusion (either on 5% glucose or 0.9% sodium chloride), through a central catheter, or over a minimum total volume of 250 mL if administered through a peripheral line, always over one hour at a fixed infusion rate. Starting dose was 3.2 mg/m2. Dose was capped at body surface area (BSA) of 2.0 m2 (i.e., dose did not exceed 6.4 mg).

Patients received lurbinectedin intravenously (i.v.) as a one-hour infusion on Day 1 q3wk (three weeks = one treatment cycle).
All-Cause Mortality
Lurbinectedin (PM01183)
Affected / at Risk (%)
Total   261/335 (77.91%)    
Hide Serious Adverse Events
Lurbinectedin (PM01183)
Affected / at Risk (%) # Events
Total   136/335 (40.60%)    
Blood and lymphatic system disorders   
Anaemia * 1  10/335 (2.99%)  18
Febrile neutropenia * 1  20/335 (5.97%)  21
Leukopenia * 1  2/335 (0.60%)  4
Lymphopenia * 1  1/335 (0.30%)  1
Neutropenia * 1  15/335 (4.48%)  22
Thrombocytopenia * 1  12/335 (3.58%)  36
Cardiac disorders   
Tachycardia * 1  1/335 (0.30%)  1
Endocrine disorders   
Cushing's syndrome * 1  1/335 (0.30%)  1
Gastrointestinal disorders   
Abdominal pain * 1  11/335 (3.28%)  12
Colitis * 1  1/335 (0.30%)  1
Diarrhoea * 1  2/335 (0.60%)  2
Dysphagia * 1  2/335 (0.60%)  2
Gastrointestinal haemorrhage * 1  2/335 (0.60%)  2
Intestinal obstruction * 1  4/335 (1.19%)  5
Intra-abdominal haemorrhage * 1  1/335 (0.30%)  1
Nausea * 1  4/335 (1.19%)  4
Small intestinal obstruction * 1  2/335 (0.60%)  2
Upper gastrointestinal haemorrhage * 1  2/335 (0.60%)  3
Vomiting * 1  7/335 (2.09%)  9
General disorders   
Asthenia * 1  2/335 (0.60%)  2
Gait disturbance * 1  2/335 (0.60%)  2
General physical health deterioration * 1  15/335 (4.48%)  21
Non-cardiac chest pain * 1  1/335 (0.30%)  1
Oedema * 1  1/335 (0.30%)  1
Oedema peripheral * 1  1/335 (0.30%)  1
Pain * 1  1/335 (0.30%)  1
Pyrexia * 1  6/335 (1.79%)  8
Hepatobiliary disorders   
Cholangitis * 1  3/335 (0.90%)  4
Hyperbilirubinaemia * 1  1/335 (0.30%)  1
Jaundice cholestatic * 1  1/335 (0.30%)  1
Infections and infestations   
Bacteraemia * 1  1/335 (0.30%)  1
Biliary tract infection * 1  1/335 (0.30%)  1
Bronchitis * 1  1/335 (0.30%)  1
Device related infection * 1  1/335 (0.30%)  1
Escherichia urinary tract infection * 1  1/335 (0.30%)  1
Lung infection * 1  3/335 (0.90%)  3
Peritonitis * 1  1/335 (0.30%)  1
Pneumonia * 1  8/335 (2.39%)  10
Pseudomonal bacteraemia * 1  1/335 (0.30%)  1
Respiratory tract infection * 1  1/335 (0.30%)  1
Sepsis * 1  3/335 (0.90%)  3
Septic shock * 1  2/335 (0.60%)  2
Skin infection * 1  2/335 (0.60%)  2
Upper respiratory tract infection * 1  4/335 (1.19%)  4
Urinary tract infection * 1  4/335 (1.19%)  4
Injury, poisoning and procedural complications   
Compression fracture * 1  1/335 (0.30%)  1
Spinal fracture * 1  1/335 (0.30%)  1
Vascular access complication * 1  1/335 (0.30%)  1
Investigations   
Alanine aminotransferase increased * 1  1/335 (0.30%)  1
Aspartate aminotransferase increased * 1  1/335 (0.30%)  1
Blood calcium decreased * 1  1/335 (0.30%)  2
Blood creatinine increased * 1  2/335 (0.60%)  2
Blood phosphorus decreased * 1  1/335 (0.30%)  1
Neutrophil count decreased * 1  2/335 (0.60%)  3
Platelet count decreased * 1  2/335 (0.60%)  5
Metabolism and nutrition disorders   
Decreased appetite * 1  1/335 (0.30%)  1
Dehydration * 1  4/335 (1.19%)  4
Hypoalbuminaemia * 1  2/335 (0.60%)  2
Hyponatraemia * 1  7/335 (2.09%)  11
Musculoskeletal and connective tissue disorders   
Back pain * 1  4/335 (1.19%)  5
Bone pain * 1  1/335 (0.30%)  1
Intervertebral disc compression * 1  1/335 (0.30%)  1
Muscular weakness * 1  2/335 (0.60%)  2
Musculoskeletal pain * 1  1/335 (0.30%)  1
Pain in extremity * 1  1/335 (0.30%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Tumour pain * 1  2/335 (0.60%)  3
Nervous system disorders   
Aphasia * 1  1/335 (0.30%)  1
Cerebrovascular accident * 1  1/335 (0.30%)  1
Cognitive disorder * 1  1/335 (0.30%)  1
Dizziness * 1  2/335 (0.60%)  2
Facial paralysis * 1  1/335 (0.30%)  1
Headache * 1  2/335 (0.60%)  2
Hemiplegia * 1  2/335 (0.60%)  2
Neuralgia * 1  2/335 (0.60%)  2
Seizure * 1  1/335 (0.30%)  1
Spinal cord compression * 1  2/335 (0.60%)  2
Subacute combined cord degeneration * 1  1/335 (0.30%)  1
Syncope * 1  1/335 (0.30%)  1
Product Issues   
Device malfunction * 1  1/335 (0.30%)  1
Psychiatric disorders   
Confusional state * 1  2/335 (0.60%)  2
Mental status changes * 1  1/335 (0.30%)  1
Renal and urinary disorders   
Haematuria * 1  4/335 (1.19%)  5
Hydronephrosis * 1  1/335 (0.30%)  1
Renal failure * 1  2/335 (0.60%)  2
Respiratory, thoracic and mediastinal disorders   
Acute respiratory failure * 1  1/335 (0.30%)  1
Aspiration * 1  1/335 (0.30%)  1
Chronic obstructive pulmonary disease * 1  1/335 (0.30%)  2
Dyspnoea * 1  8/335 (2.39%)  13
Haemoptysis * 1  1/335 (0.30%)  1
Lung infiltration * 1  1/335 (0.30%)  1
Pleural effusion * 1  1/335 (0.30%)  1
Pneumonitis * 1  3/335 (0.90%)  3
Pneumothorax * 1  2/335 (0.60%)  2
Pulmonary arterial hypertension * 1  1/335 (0.30%)  1
Pulmonary embolism * 1  4/335 (1.19%)  4
Respiratory failure * 1  2/335 (0.60%)  2
Surgical and medical procedures   
Cementoplasty * 1  1/335 (0.30%)  1
Vascular disorders   
Deep vein thrombosis * 1  1/335 (0.30%)  1
Embolism * 1  2/335 (0.60%)  2
Superior vena cava occlusion * 1  1/335 (0.30%)  1
Superior vena cava syndrome * 1  2/335 (0.60%)  2
1
Term from vocabulary, MedDRA (21.0)
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Lurbinectedin (PM01183)
Affected / at Risk (%) # Events
Total   330/335 (98.51%)    
Blood and lymphatic system disorders   
Anaemia * 1  59/335 (17.61%)  100
Neutropenia * 1  89/335 (26.57%)  174
Gastrointestinal disorders   
Abdominal pain * 1  54/335 (16.12%)  83
Abdominal pain upper * 1  19/335 (5.67%)  21
Constipation * 1  116/335 (34.63%)  189
Diarrhoea * 1  62/335 (18.51%)  104
Nausea * 1  167/335 (49.85%)  276
Vomiting * 1  76/335 (22.69%)  126
General disorders   
Asthenia * 1  129/335 (38.51%)  335
Chest pain * 1  23/335 (6.87%)  28
Fatigue * 1  122/335 (36.42%)  242
Mucosal inflammation * 1  19/335 (5.67%)  23
Oedema peripheral * 1  30/335 (8.96%)  35
Pyrexia * 1  48/335 (14.33%)  56
Investigations   
Weight decreased * 1  25/335 (7.46%)  33
Metabolism and nutrition disorders   
Decreased appetite * 1  102/335 (30.45%)  144
Hypoalbuminaemia * 1  18/335 (5.37%)  31
Musculoskeletal and connective tissue disorders   
Arthralgia * 1  23/335 (6.87%)  28
Back pain * 1  51/335 (15.22%)  71
Musculoskeletal pain * 1  20/335 (5.97%)  25
Pain in extremity * 1  23/335 (6.87%)  32
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Tumour pain * 1  28/335 (8.36%)  34
Nervous system disorders   
Headache * 1  25/335 (7.46%)  28
Neuropathy peripheral * 1  19/335 (5.67%)  27
Psychiatric disorders   
Insomnia * 1  34/335 (10.15%)  37
Respiratory, thoracic and mediastinal disorders   
Cough * 1  58/335 (17.31%)  71
Dyspnoea * 1  77/335 (22.99%)  99
1
Term from vocabulary, MedDRA (21.0)
*
Indicates events were collected by non-systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Clinical Development, Department of PharmaMar´s Oncology., Business Unit.
Organization: Pharma Mar S.A.
Phone: 0034 91846 60 00
EMail: clinicaltrials@pharmamar.com
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: PharmaMar
ClinicalTrials.gov Identifier: NCT02454972    
Other Study ID Numbers: PM1183-B-005-14
First Submitted: May 6, 2015
First Posted: May 27, 2015
Results First Submitted: September 17, 2021
Results First Posted: November 22, 2021
Last Update Posted: March 2, 2023