Trial Outcomes & Findings for Clinical Study of BYM338 for the Treatment of Unintentional Weight Loss in Patients With Cancer of the Lung or the Pancreas (NCT NCT01433263)
NCT ID: NCT01433263
Last Updated: 2016-03-02
Results Overview
Thigh Muscle Volume (TMV) change was evaluated by a responder analysis. Patients whose loss of muscle TMV by MRI was no more than or equal to 2% at Week 8 was considered responders.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
57 participants
Primary outcome timeframe
Baseline, week 8
Results posted on
2016-03-02
Participant Flow
Core Phase single dose BYM338 30mg/kg i.v. active or placebo with 8week followup. Followup phase started Week 8 \& patients on placebo in the Core Phase were given BYM338 \& patients on BYM338 in Core Phase continued to be followed for an additional 8 weeks. Late BYM338 are patients who received Placebo during Core Phase and then BYM338 after Week 8.
Participant milestones
| Measure |
30mg/kg BYM338
|
Placebo / Late 30mg/kg BYM338
|
|---|---|---|
|
Overall Study
STARTED
|
29
|
28
|
|
Overall Study
COMPLETED
|
10
|
16
|
|
Overall Study
NOT COMPLETED
|
19
|
12
|
Reasons for withdrawal
| Measure |
30mg/kg BYM338
|
Placebo / Late 30mg/kg BYM338
|
|---|---|---|
|
Overall Study
Adverse Event
|
3
|
1
|
|
Overall Study
Death
|
5
|
3
|
|
Overall Study
Lost to Follow-up
|
0
|
1
|
|
Overall Study
Protocol Deviation
|
1
|
0
|
|
Overall Study
Withdrawal by Subject
|
10
|
7
|
Baseline Characteristics
Clinical Study of BYM338 for the Treatment of Unintentional Weight Loss in Patients With Cancer of the Lung or the Pancreas
Baseline characteristics by cohort
| Measure |
30mg/kg BYM338
n=29 Participants
|
Placebo / Late 30mg/kg BYM338
n=28 Participants
|
Total
n=57 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
62.8 years
STANDARD_DEVIATION 10.17 • n=5 Participants
|
61.5 years
STANDARD_DEVIATION 10.74 • n=7 Participants
|
62.1 years
STANDARD_DEVIATION 10.38 • n=5 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=5 Participants
|
22 Participants
n=7 Participants
|
42 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, week 8Population: Pharmacodynamics (PD) analysis set: Patients with evaluable PD parameter data. However, for a given time frame, analyzed participants had values at both baseline and the corresponding time frame, i.e. week 8
Thigh Muscle Volume (TMV) change was evaluated by a responder analysis. Patients whose loss of muscle TMV by MRI was no more than or equal to 2% at Week 8 was considered responders.
Outcome measures
| Measure |
30mg/kg BYM338
n=15 Participants
|
Placebo / Late 30mg/kg BYM338
n=22 Participants
|
|---|---|---|
|
Percentage Change From Baseline of Thigh Muscle Volume (TMV) by MRI Scan at Week 8
|
2.0 Percentage Change of TMV
Standard Deviation 8.094
|
0.65 Percentage Change of TMV
Standard Deviation 8.239
|
SECONDARY outcome
Timeframe: Baseline, Week 7 and Week 9Population: Pharmacodynamics (PD) analysis set: Patients with evaluable PD parameter data.
Percentage Change in body weight from baseline in killograms (kg) at week 7 and week 9
Outcome measures
| Measure |
30mg/kg BYM338
n=29 Participants
|
Placebo / Late 30mg/kg BYM338
n=28 Participants
|
|---|---|---|
|
Percentage Change in Body Weight From Baseline at Week 7 and Week 9
Week 7 (n= 15, 17)
|
-3.3 Percent Change of Weight (kg)
Standard Deviation 5.035
|
-0.68 Percent Change of Weight (kg)
Standard Deviation 4.457
|
|
Percentage Change in Body Weight From Baseline at Week 7 and Week 9
Week 9 (n=14,16)
|
-1.8 Percent Change of Weight (kg)
Standard Deviation 7.131
|
-0.32 Percent Change of Weight (kg)
Standard Deviation 3.271
|
SECONDARY outcome
Timeframe: 0, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96 hours post-dose on Day 1 and Week 8Population: Pharmacokinetics (PK) analysis set: Patients with evaluable PK data.
Blood samples for pharmacokinetic (PK) evaluation were drawn on Day 1 30mg/kg BYM338 (Core)or week 8 Late 30mg/kg BYM338 (when placebo subjects were rolled over to active). PK parameters were calculated from plasma concentration-time data using non-compartmental methods.
Outcome measures
| Measure |
30mg/kg BYM338
n=29 Participants
|
Placebo / Late 30mg/kg BYM338
n=14 Participants
|
|---|---|---|
|
Maximum Observed Serum Concentration (Cmax)
|
422 ng/ml
Standard Deviation 142
|
408 ng/ml
Standard Deviation 78.4
|
SECONDARY outcome
Timeframe: 0, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96 hours post-dose on Day 1 and Week 8Population: Pharmacokinetics (PK) analysis set: Patients with evaluable PK data.
Blood samples for pharmacokinetic (PK) evaluation were drawn on Day 1 30mg/kg BYM338 (Core)or week 8 Late 30mg/kg BYM338 (when placebo subjects were rolled over to active). Tmax was directly determined from the raw serum concentration-time data.
Outcome measures
| Measure |
30mg/kg BYM338
n=29 Participants
|
Placebo / Late 30mg/kg BYM338
n=14 Participants
|
|---|---|---|
|
Time to Reach the Maximum Concentration After Drug Administration (Tmax)
|
2.05 hr
Inter-Quartile Range 142 • Interval 1.83 to 3.92
|
2.22 hr
Inter-Quartile Range 78.4 • Interval 2.0 to 4.0
|
SECONDARY outcome
Timeframe: Baseline, Week 8Population: Pharmacodynamics (PD) analysis set: Patients with evaluable PD parameter data. However, for a given time frame, analyzed participants had values at both baseline and the corresponding time frame, i.e. week 8
total lean body mass (LBM) is measured by dual energy x-ray absorptiometry (DXA).Percent Change = \[(LBM at Visit - LBM at Baseline) / LBM at Baseline\] \* 100.
Outcome measures
| Measure |
30mg/kg BYM338
n=19 Participants
|
Placebo / Late 30mg/kg BYM338
n=22 Participants
|
|---|---|---|
|
Percentage Change From Baseline in Total Lean Body Mass (LBM) by Dual-Energy X-ray Absorptiometery (DXA) Compared to Placebo: at Week 8
|
4.97 Percentage Change in LBM
Standard Deviation 7.537
|
2.41 Percentage Change in LBM
Standard Deviation 4.625
|
SECONDARY outcome
Timeframe: Baseline, Week 8Population: Pharmacodynamics (PD) analysis set: Patients with evaluable PD parameter data. However, for a given time frame, analyzed participants had values at both baseline and the corresponding time frame, i.e. week 8
Bone Mineral Density (BMD)is measured by dual energy x-ray absorptiometry (DXA).Percent Change = \[(BMD at Visit - BMD at Baseline) / BMD at Baseline\] \* 100.
Outcome measures
| Measure |
30mg/kg BYM338
n=19 Participants
|
Placebo / Late 30mg/kg BYM338
n=22 Participants
|
|---|---|---|
|
Percentage Change From Baseline of Bone Mineral Density (BMD) by Dual-Energy X-ray Absorptiometery (DXA) Compared to Placebo at Week 8
|
0.51 Percentage Change in BMD
Standard Deviation 3.712
|
0.14 Percentage Change in BMD
Standard Deviation 4.14
|
SECONDARY outcome
Timeframe: Baseline, Week 4 and Week 7Population: Pharmacodynamics (PD) analysis set: Patients with evaluable PD parameter data.
Each patient was required to wear the ActivPal™ for a span of 6 days at Week 4 and Week 7 for patient home activity recording. The ActivPal™ was given to patients in clinic to wear for 6 consecutive days. The ActivPAL™ records periods spent sitting, standing and walking, sit-to-stand transitions, step count and rate of stepping (cadence) over a maximum period of 10 days with a fully charged new battery.
Outcome measures
| Measure |
30mg/kg BYM338
n=27 Participants
|
Placebo / Late 30mg/kg BYM338
n=28 Participants
|
|---|---|---|
|
Percentage Change From Baseline of Physical Activity Levels (Using the ActivPAL™ Device) Number of Steps Taken Compared to Placebo at Week 4 and 7
Week 4 (n=18, 23)
|
917.78 percentage change in number of steps
Standard Deviation 3720.491
|
63.59 percentage change in number of steps
Standard Deviation 130.913
|
|
Percentage Change From Baseline of Physical Activity Levels (Using the ActivPAL™ Device) Number of Steps Taken Compared to Placebo at Week 4 and 7
Week 7 (n=13, 22)
|
-17.37 percentage change in number of steps
Standard Deviation 80.350
|
35.80 percentage change in number of steps
Standard Deviation 119.486
|
SECONDARY outcome
Timeframe: Baseline, Week 4 and Week 7Population: Pharmacodynamics (PD) analysis set: Patients with evaluable PD parameter data.
Each patient was required to wear the ActivPal™ for a span of 6 days at Week 4 and Week 7 for patient home activity recording. The ActivPal™ was given to patients in clinic to wear for 6 consecutive days. The ActivPAL™ records periods spent sitting, standing and walking, sit-to-stand transitions, step count and rate of stepping (cadence) over a maximum period of 10 days with a fully charged new battery.
Outcome measures
| Measure |
30mg/kg BYM338
n=27 Participants
|
Placebo / Late 30mg/kg BYM338
n=28 Participants
|
|---|---|---|
|
Percentage Change From Baseline of Physical Activity Levels (Using the ActivPAL™ Device) Time Sedentary Taken Compared to Placebo at Week 4 and 7
Week 4 (n=18, 23)
|
-0.05 percentage change in time (minutes)
Standard Deviation 10.049
|
52.25 percentage change in time (minutes)
Standard Deviation 207.403
|
|
Percentage Change From Baseline of Physical Activity Levels (Using the ActivPAL™ Device) Time Sedentary Taken Compared to Placebo at Week 4 and 7
Week 7 (n=13, 22)
|
107.85 percentage change in time (minutes)
Standard Deviation 280.791
|
60.25 percentage change in time (minutes)
Standard Deviation 222.366
|
SECONDARY outcome
Timeframe: Baseline, Week 4 and Week 7Population: Pharmacodynamics (PD) analysis set: Patients with evaluable PD parameter data.
Each patient was required to wear the ActivPal™ for a span of 6 days at Week 4 and Week 7 for patient home activity recording. The ActivPal™ was given to patients in clinic to wear for 6 consecutive days. The ActivPAL™ records periods spent sitting, standing and walking, sit-to-stand transitions, step count and rate of stepping (cadence) over a maximum period of 10 days with a fully charged new battery.
Outcome measures
| Measure |
30mg/kg BYM338
n=27 Participants
|
Placebo / Late 30mg/kg BYM338
n=28 Participants
|
|---|---|---|
|
Percentage Change From Baseline of Physical Activity Levels (Using the ActivPAL™ Device) Time Standing Compared to Placebo at Week 4 and 7
Week 4 (n=18, 23)
|
1.82 percentage change in time (minutes)
Standard Deviation 78.364
|
38.17 percentage change in time (minutes)
Standard Deviation 111.361
|
|
Percentage Change From Baseline of Physical Activity Levels (Using the ActivPAL™ Device) Time Standing Compared to Placebo at Week 4 and 7
Week 7 (n=13, 22)
|
41.90 percentage change in time (minutes)
Standard Deviation 251.291
|
23.76 percentage change in time (minutes)
Standard Deviation 99.268
|
SECONDARY outcome
Timeframe: Baseline, Week 4 and Week 7Population: Pharmacodynamics (PD) analysis set: Patients with evaluable PD parameter data.
Each patient was required to wear the ActivPal™ for a span of 6 days at Week 4 and Week 7 for patient home activity recording. The ActivPal™ was given to patients in clinic to wear for 6 consecutive days. The ActivPAL™ records periods spent sitting, standing and walking, sit-to-stand transitions, step count and rate of stepping (cadence) over a maximum period of 10 days with a fully charged new battery.
Outcome measures
| Measure |
30mg/kg BYM338
n=27 Participants
|
Placebo / Late 30mg/kg BYM338
n=28 Participants
|
|---|---|---|
|
Percentage Change From Baseline of Physical Activity Levels (Using the ActivPAL™ Device) Time Stepping Compared to Placebo at Week 4 and 7
Week 4 (n=18, 22)
|
1446.69 percentage change in time (minutes)
Standard Deviation 6011.324
|
85.30 percentage change in time (minutes)
Standard Deviation 159.949
|
|
Percentage Change From Baseline of Physical Activity Levels (Using the ActivPAL™ Device) Time Stepping Compared to Placebo at Week 4 and 7
Week 7 (n=13, 21)
|
-31.64 percentage change in time (minutes)
Standard Deviation 67.180
|
33.39 percentage change in time (minutes)
Standard Deviation 129.218
|
Adverse Events
Core - 30mg/kg BYM338
Serious events: 18 serious events
Other events: 19 other events
Deaths: 0 deaths
Core - Placebo
Serious events: 4 serious events
Other events: 19 other events
Deaths: 0 deaths
Follow-up - 30mg/kg BYM338
Serious events: 7 serious events
Other events: 12 other events
Deaths: 0 deaths
Follow-up - Placebo
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Follow-up - 30mg/kg BYM338 Late
Serious events: 7 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Core - 30mg/kg BYM338
n=29 participants at risk
Core - 30mg/kg BYM338
|
Core - Placebo
n=28 participants at risk
Core - Placebo
|
Follow-up - 30mg/kg BYM338
n=19 participants at risk
Follow-up - 30mg/kg BYM338
|
Follow-up - Placebo
n=3 participants at risk
Follow-up - Placebo
|
Follow-up - 30mg/kg BYM338 Late
n=21 participants at risk
Follow-up - 30mg/kg BYM338 Late
|
|---|---|---|---|---|---|
|
Infections and infestations
Klebsiella sepsis
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Infections and infestations
Pneumonia
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Infections and infestations
Urinary tract infection
|
6.9%
2/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Metabolism and nutrition disorders
Dehydration
|
10.3%
3/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Pancreatic carcinoma
|
0.00%
0/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
4.8%
1/21
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Tumour pain
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Nervous system disorders
Cerebral infarction
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Nervous system disorders
Cerebrovascular accident
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Nervous system disorders
Dizziness
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Nervous system disorders
Hemiparesis
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Metabolism and nutrition disorders
Diabetes mellitus inadequate control
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Metabolism and nutrition disorders
Failure to thrive
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Nervous system disorders
Ischaemic stroke
|
0.00%
0/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
4.8%
1/21
|
|
Nervous system disorders
Presyncope
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Nervous system disorders
Syncope
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Metabolism and nutrition disorders
Malnutrition
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Lung neoplasm malignant
|
0.00%
0/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
4.8%
1/21
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Malignant neoplasm progression
|
20.7%
6/29
|
0.00%
0/28
|
15.8%
3/19
|
0.00%
0/3
|
4.8%
1/21
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastases to central nervous system
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
4.8%
1/21
|
|
Blood and lymphatic system disorders
Anaemia
|
6.9%
2/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Blood and lymphatic system disorders
Febrile bone marrow aplasia
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Eye disorders
Diplopia
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/29
|
3.6%
1/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Gastrointestinal disorders
Ascites
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Gastrointestinal disorders
Gastric haemorrhage
|
0.00%
0/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
4.8%
1/21
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Gastrointestinal disorders
Intestinal obstruction
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/29
|
0.00%
0/28
|
0.00%
0/19
|
33.3%
1/3
|
0.00%
0/21
|
|
Gastrointestinal disorders
Obstruction gastric
|
0.00%
0/29
|
3.6%
1/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
0.00%
0/29
|
3.6%
1/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/29
|
0.00%
0/28
|
0.00%
0/19
|
33.3%
1/3
|
0.00%
0/21
|
|
General disorders
Asthenia
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
General disorders
Disease progression
|
0.00%
0/29
|
3.6%
1/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
General disorders
Oedema peripheral
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Hepatobiliary disorders
Biliary dilatation
|
0.00%
0/29
|
3.6%
1/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Hepatobiliary disorders
Cholestasis
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Hepatobiliary disorders
Hepatic failure
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Infections and infestations
Bacterial sepsis
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Infections and infestations
Infection
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Infections and infestations
Klebsiella infection
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/29
|
3.6%
1/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Renal and urinary disorders
Renal failure
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Renal and urinary disorders
Renal failure acute
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
0.00%
0/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
4.8%
1/21
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Vascular disorders
Angiopathy
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Vascular disorders
Deep vein thrombosis
|
6.9%
2/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Vascular disorders
Peripheral ischaemia
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
Other adverse events
| Measure |
Core - 30mg/kg BYM338
n=29 participants at risk
Core - 30mg/kg BYM338
|
Core - Placebo
n=28 participants at risk
Core - Placebo
|
Follow-up - 30mg/kg BYM338
n=19 participants at risk
Follow-up - 30mg/kg BYM338
|
Follow-up - Placebo
n=3 participants at risk
Follow-up - Placebo
|
Follow-up - 30mg/kg BYM338 Late
n=21 participants at risk
Follow-up - 30mg/kg BYM338 Late
|
|---|---|---|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
13.8%
4/29
|
21.4%
6/28
|
10.5%
2/19
|
33.3%
1/3
|
14.3%
3/21
|
|
Blood and lymphatic system disorders
Leukopenia
|
10.3%
3/29
|
3.6%
1/28
|
5.3%
1/19
|
0.00%
0/3
|
4.8%
1/21
|
|
Blood and lymphatic system disorders
Neutropenia
|
10.3%
3/29
|
10.7%
3/28
|
10.5%
2/19
|
0.00%
0/3
|
4.8%
1/21
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
6.9%
2/29
|
7.1%
2/28
|
5.3%
1/19
|
0.00%
0/3
|
4.8%
1/21
|
|
Gastrointestinal disorders
Abdominal pain
|
6.9%
2/29
|
10.7%
3/28
|
0.00%
0/19
|
0.00%
0/3
|
4.8%
1/21
|
|
Gastrointestinal disorders
Ascites
|
6.9%
2/29
|
3.6%
1/28
|
5.3%
1/19
|
33.3%
1/3
|
4.8%
1/21
|
|
Gastrointestinal disorders
Constipation
|
6.9%
2/29
|
3.6%
1/28
|
0.00%
0/19
|
33.3%
1/3
|
0.00%
0/21
|
|
Gastrointestinal disorders
Diarrhoea
|
17.2%
5/29
|
10.7%
3/28
|
5.3%
1/19
|
0.00%
0/3
|
23.8%
5/21
|
|
Gastrointestinal disorders
Dysphagia
|
0.00%
0/29
|
3.6%
1/28
|
0.00%
0/19
|
33.3%
1/3
|
4.8%
1/21
|
|
Gastrointestinal disorders
Nausea
|
17.2%
5/29
|
3.6%
1/28
|
0.00%
0/19
|
0.00%
0/3
|
9.5%
2/21
|
|
Gastrointestinal disorders
Stomatitis
|
3.4%
1/29
|
10.7%
3/28
|
10.5%
2/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Gastrointestinal disorders
Vomiting
|
13.8%
4/29
|
3.6%
1/28
|
0.00%
0/19
|
0.00%
0/3
|
9.5%
2/21
|
|
General disorders
Asthenia
|
6.9%
2/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
General disorders
Fatigue
|
20.7%
6/29
|
10.7%
3/28
|
21.1%
4/19
|
33.3%
1/3
|
4.8%
1/21
|
|
General disorders
Oedema peripheral
|
10.3%
3/29
|
10.7%
3/28
|
5.3%
1/19
|
0.00%
0/3
|
4.8%
1/21
|
|
General disorders
Pyrexia
|
3.4%
1/29
|
7.1%
2/28
|
0.00%
0/19
|
0.00%
0/3
|
4.8%
1/21
|
|
Hepatobiliary disorders
Jaundice
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Infections and infestations
Candida infection
|
0.00%
0/29
|
7.1%
2/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Infections and infestations
Gingival infection
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Infections and infestations
Herpes zoster
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Infections and infestations
Septic shock
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/29
|
0.00%
0/28
|
10.5%
2/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Infections and infestations
Wound infection staphylococcal
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Injury, poisoning and procedural complications
Craniocerebral injury
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Injury, poisoning and procedural complications
Fall
|
3.4%
1/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Injury, poisoning and procedural complications
Laceration
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Investigations
Amylase increased
|
0.00%
0/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
9.5%
2/21
|
|
Investigations
Blood bilirubin increased
|
3.4%
1/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Investigations
Gamma-glutamyltransferase increased
|
3.4%
1/29
|
3.6%
1/28
|
5.3%
1/19
|
0.00%
0/3
|
4.8%
1/21
|
|
Investigations
Liver function test abnormal
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Investigations
Weight decreased
|
3.4%
1/29
|
0.00%
0/28
|
10.5%
2/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Metabolism and nutrition disorders
Decreased appetite
|
10.3%
3/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
4.8%
1/21
|
|
Metabolism and nutrition disorders
Dehydration
|
13.8%
4/29
|
3.6%
1/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
6.9%
2/29
|
3.6%
1/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
3.4%
1/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
6.9%
2/29
|
7.1%
2/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
6.9%
2/29
|
3.6%
1/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
10.3%
3/29
|
3.6%
1/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Metabolism and nutrition disorders
Lactic acidosis
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Metabolism and nutrition disorders
Malnutrition
|
6.9%
2/29
|
3.6%
1/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/29
|
7.1%
2/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
6.9%
2/29
|
3.6%
1/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
10.3%
3/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Benign neoplasm
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Nervous system disorders
Dizziness
|
6.9%
2/29
|
0.00%
0/28
|
0.00%
0/19
|
33.3%
1/3
|
4.8%
1/21
|
|
Nervous system disorders
Headache
|
0.00%
0/29
|
7.1%
2/28
|
0.00%
0/19
|
0.00%
0/3
|
9.5%
2/21
|
|
Nervous system disorders
Hepatic encephalopathy
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Nervous system disorders
Myoclonus
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Nervous system disorders
Neuropathy peripheral
|
3.4%
1/29
|
3.6%
1/28
|
0.00%
0/19
|
0.00%
0/3
|
9.5%
2/21
|
|
Nervous system disorders
Tremor
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Psychiatric disorders
Insomnia
|
6.9%
2/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Renal and urinary disorders
Bladder irritation
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Renal and urinary disorders
Urinary incontinence
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/29
|
3.6%
1/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
0.00%
0/29
|
7.1%
2/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
3.4%
1/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
4.8%
1/21
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Respiratory, thoracic and mediastinal disorders
Rales
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Skin and subcutaneous tissue disorders
Erythema
|
3.4%
1/29
|
0.00%
0/28
|
0.00%
0/19
|
33.3%
1/3
|
0.00%
0/21
|
|
Skin and subcutaneous tissue disorders
Rash
|
3.4%
1/29
|
7.1%
2/28
|
0.00%
0/19
|
0.00%
0/3
|
4.8%
1/21
|
|
Skin and subcutaneous tissue disorders
Swelling face
|
0.00%
0/29
|
0.00%
0/28
|
5.3%
1/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Vascular disorders
Deep vein thrombosis
|
10.3%
3/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
0.00%
0/21
|
|
Vascular disorders
Hot flush
|
0.00%
0/29
|
0.00%
0/28
|
0.00%
0/19
|
0.00%
0/3
|
9.5%
2/21
|
Additional Information
Study Director
Novartis Pharmaceuticals
Phone: 862-778-8300
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial
- Publication restrictions are in place
Restriction type: OTHER