Trial Outcomes & Findings for Study to Assess the Efficacy and Safety of Different Doses of BIM 23A760 in Patients With Carcinoid Syndrome (NCT NCT01018953)
NCT ID: NCT01018953
Last Updated: 2020-11-20
Results Overview
Patient satisfaction based on a Likert scale from 0-5 (0 being not satisfied and 5 being completely satisfied)
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
8 participants
Primary outcome timeframe
Week 24
Results posted on
2020-11-20
Participant Flow
This study was terminated prematurely. Only 8 patients were treated in part A and no patients participated in part B.
Patients screened were 15 and not treated were 7 (2 subjects were not included due to early termination of study by sponsor and 5 subjects failed to meet inclusion criteria).
Participant milestones
| Measure |
BIM 23A760
This dose adaptive study was planned to treat up to 20 patients in each starting dose cohort, with a maximum of three starting dose cohorts. The doses planned to be assessed were 1, 2, 4, 6 and 8 mg; however, the maximum starting dose was 4 mg. The starting dose of the first cohort was 1 mg and the first cohort would include at least five patients. After the first 15 patients were treated for 4 weeks, the results were to be reviewed by a Data Review Committee. An extension phase (Part B) was planned for those subjects completing the initial study and fulfilling specific eligibility criteria (symptoms control, willingness to participate, safety and tolerability).
BIM 23A760 was a solution at a concentration of 5 mg/mL ready for subcutaneous injection. BIM 23A760 doses of 1, 2, 4, 6 and 8 mg were to be given to the patient according to a dose escalation and titration process. Patients would have received 24 weekly injections of BIM 23A760 during the treatment period.
|
|---|---|
|
Overall Study
STARTED
|
8
|
|
Overall Study
BIM 23A760 1 mg
|
3
|
|
Overall Study
BIM 23A760 2 mg
|
2
|
|
Overall Study
BIM 23A760 4 mg
|
3
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
8
|
Reasons for withdrawal
| Measure |
BIM 23A760
This dose adaptive study was planned to treat up to 20 patients in each starting dose cohort, with a maximum of three starting dose cohorts. The doses planned to be assessed were 1, 2, 4, 6 and 8 mg; however, the maximum starting dose was 4 mg. The starting dose of the first cohort was 1 mg and the first cohort would include at least five patients. After the first 15 patients were treated for 4 weeks, the results were to be reviewed by a Data Review Committee. An extension phase (Part B) was planned for those subjects completing the initial study and fulfilling specific eligibility criteria (symptoms control, willingness to participate, safety and tolerability).
BIM 23A760 was a solution at a concentration of 5 mg/mL ready for subcutaneous injection. BIM 23A760 doses of 1, 2, 4, 6 and 8 mg were to be given to the patient according to a dose escalation and titration process. Patients would have received 24 weekly injections of BIM 23A760 during the treatment period.
|
|---|---|
|
Overall Study
Adverse Event
|
2
|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Study termination by sponsor
|
5
|
Baseline Characteristics
Study to Assess the Efficacy and Safety of Different Doses of BIM 23A760 in Patients With Carcinoid Syndrome
Baseline characteristics by cohort
| Measure |
BIM 23A760
n=8 Participants
This dose adaptive study was planned to treat up to 20 patients in each starting dose cohort, with a maximum of three starting dose cohorts. The doses planned to be assessed were 1, 2, 4, 6 and 8 mg; however, the maximum starting dose was 4 mg. The starting dose of the first cohort was 1 mg and the first cohort would include at least five patients. After the first 15 patients were treated for 4 weeks, the results were to be reviewed by a Data Review Committee. An extension phase (Part B) was planned for those subjects completing the initial study and fulfilling specific eligibility criteria (symptoms control, willingness to participate, safety and tolerability).
BIM 23A760 was a solution at a concentration of 5 mg/mL ready for subcutaneous injection. BIM 23A760 doses of 1, 2, 4, 6 and 8 mg were to be given to the patient according to a dose escalation and titration process. Patients would have received 24 weekly injections of BIM 23A760 during the treatment period.
|
|---|---|
|
Age, Continuous
|
62.1 years
STANDARD_DEVIATION 6.88 • n=5 Participants
|
|
Age, Customized
Between 18 and 75 years
|
8 participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Caucasian/ White
|
8 participants
n=5 Participants
|
|
Region of Enrollment
Russian Federation
|
1 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Netherlands
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Belgium
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Ireland
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Finland
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
1 participants
n=5 Participants
|
|
Diabetic status
Diabetic
|
0 participants
n=5 Participants
|
|
Diabetic status
Nondiabetic
|
8 participants
n=5 Participants
|
|
Post-menopausal status
Yes
|
6 participants
n=5 Participants
|
|
Post-menopausal status
No
|
0 participants
n=5 Participants
|
|
Post-menopausal status
N/A - Not applicable
|
2 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Week 24Population: Study was prematurely terminated and no data was collected/analyzed for this outcome measure.
Patient satisfaction based on a Likert scale from 0-5 (0 being not satisfied and 5 being completely satisfied)
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to week 24Population: Study was prematurely terminated and no data was collected/analyzed for this outcome measure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 24Population: Study was prematurely terminated and no data was collected/analyzed for this outcome measure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 24Population: Study was prematurely terminated and no data was collected/analyzed for this outcome measure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to week 26Population: Both ITT (Intent-To-Treat) and safety populations were the same analysis group. Treatment emergent adverse events (TEAE) reported by 2 or more patients (safety population) by primary system organ class.
Outcome measures
| Measure |
BIM 23A760
n=8 Participants
This dose adaptive study was planned to treat up to 20 patients in each starting dose cohort, with a maximum of three starting dose cohorts. The doses planned to be assessed were 1, 2, 4, 6 and 8 mg; however, the maximum starting dose was 4 mg. The starting dose of the first cohort was 1 mg and the first cohort would include at least five patients. After the first 15 patients were treated for 4 weeks, the results were to be reviewed by a Data Review Committee. An extension phase (Part B) was planned for those subjects completing the initial study and fulfilling specific eligibility criteria (symptoms control, willingness to participate, safety and tolerability).
BIM 23A760 was a solution at a concentration of 5 mg/mL ready for subcutaneous injection. BIM 23A760 doses of 1, 2, 4, 6 and 8 mg were to be given to the patient according to a dose escalation and titration process. Patients would have received 24 weekly injections of BIM 23A760 during the treatment period.
|
|---|---|
|
Number of Subjects Reported Adverse Events, Including Any Findings From an Examination of the Injection Site(s)
Nervous System Disorders
|
3 Participants
|
|
Number of Subjects Reported Adverse Events, Including Any Findings From an Examination of the Injection Site(s)
General Disorders & Administration Site Condition
|
5 Participants
|
|
Number of Subjects Reported Adverse Events, Including Any Findings From an Examination of the Injection Site(s)
Gastrointestinal Disorder
|
4 Participants
|
|
Number of Subjects Reported Adverse Events, Including Any Findings From an Examination of the Injection Site(s)
Infections and Infestations
|
2 Participants
|
|
Number of Subjects Reported Adverse Events, Including Any Findings From an Examination of the Injection Site(s)
Metabolism and Nutritional Disorders
|
2 Participants
|
|
Number of Subjects Reported Adverse Events, Including Any Findings From an Examination of the Injection Site(s)
Neoplasms Benign, Malignant and unspecified
|
2 Participants
|
|
Number of Subjects Reported Adverse Events, Including Any Findings From an Examination of the Injection Site(s)
Reproductive System and Breast Disorders
|
2 Participants
|
SECONDARY outcome
Timeframe: At 9 timepoints up to 1 week after 24th administration in week 24Population: Study was prematurely terminated and no data was collected/analyzed for this outcome measure.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At 8 timepoints up to week 24Population: Study was prematurely terminated and no data was collected/analyzed for this outcome measure.
Outcome measures
Outcome data not reported
Adverse Events
BIM 23A760
Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
BIM 23A760
n=8 participants at risk
This dose adaptive study was planned to treat up to 20 patients in each starting dose cohort, with a maximum of three starting dose cohorts. The doses planned to be assessed were 1, 2, 4, 6 and 8 mg; however, the maximum starting dose was 4 mg. The starting dose of the first cohort was 1 mg and the first cohort would include at least five patients. After the first 15 patients were treated for 4 weeks, the results were to be reviewed by a Data Review Committee. An extension phase (Part B) was planned for those subjects completing the initial study and fulfilling specific eligibility criteria (symptoms control, willingness to participate, safety and tolerability).
BIM 23A760 was a solution at a concentration of 5 mg/mL ready for subcutaneous injection. BIM 23A760 doses of 1, 2, 4, 6 and 8 mg were to be given to the patient according to a dose escalation and titration process. Patients would have received 24 weekly injections of BIM 23A760 during the treatment period.
|
|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasm progression
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
Other adverse events
| Measure |
BIM 23A760
n=8 participants at risk
This dose adaptive study was planned to treat up to 20 patients in each starting dose cohort, with a maximum of three starting dose cohorts. The doses planned to be assessed were 1, 2, 4, 6 and 8 mg; however, the maximum starting dose was 4 mg. The starting dose of the first cohort was 1 mg and the first cohort would include at least five patients. After the first 15 patients were treated for 4 weeks, the results were to be reviewed by a Data Review Committee. An extension phase (Part B) was planned for those subjects completing the initial study and fulfilling specific eligibility criteria (symptoms control, willingness to participate, safety and tolerability).
BIM 23A760 was a solution at a concentration of 5 mg/mL ready for subcutaneous injection. BIM 23A760 doses of 1, 2, 4, 6 and 8 mg were to be given to the patient according to a dose escalation and titration process. Patients would have received 24 weekly injections of BIM 23A760 during the treatment period.
|
|---|---|
|
General disorders
Injection site erythema
|
25.0%
2/8 • Number of events 2 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
General disorders
Fatigue
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
General disorders
Injection site inflammation
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
General disorders
Injection site pain
|
12.5%
1/8 • Number of events 3 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
General disorders
Injection site pruritus
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
General disorders
Injection site reaction
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
General disorders
Oedema
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Gastrointestinal disorders
Nausea
|
37.5%
3/8 • Number of events 3 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Gastrointestinal disorders
Abdominal pain
|
25.0%
2/8 • Number of events 2 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Gastrointestinal disorders
Constipation
|
25.0%
2/8 • Number of events 2 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Gastrointestinal disorders
Vomiting
|
25.0%
2/8 • Number of events 3 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Gastrointestinal disorders
Abdominal distension
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Gastrointestinal disorders
Diarrhoea
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Gastrointestinal disorders
Dyspepsia
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Gastrointestinal disorders
Mucous stools
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Nervous system disorders
Dizziness
|
25.0%
2/8 • Number of events 2 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Nervous system disorders
Headache
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Nervous system disorders
Lethargy
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Infections and infestations
Nasopharyngitis
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Infections and infestations
Sinusitis
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
25.0%
2/8 • Number of events 3 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastases to liver
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastases to peripheral vascular system
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Reproductive system and breast disorders
Breast pain
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Reproductive system and breast disorders
Pelvic floor muscle weakness
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Reproductive system and breast disorders
Vulvovaginal dryness
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Eye disorders
Vision blurred
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Musculoskeletal and connective tissue disorders
Groin pain
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Musculoskeletal and connective tissue disorders
Systemic lupus erythematosus
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Psychiatric disorders
Anxiety
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Skin and subcutaneous tissue disorders
Night sweats
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
|
Vascular disorders
Flushing
|
12.5%
1/8 • Number of events 1 • Up to week 26
Dose received prior to AE onset. Two serious adverse events (SAEs) 'Confusional State' and 'Nausea' were not included as they occurred during screening phase, before starting study drug.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place