Trial Outcomes & Findings for Pasireotide LAR in Severe Polycystic Liver Disease (NCT NCT01670110)

NCT ID: NCT01670110

Last Updated: 2020-05-01

Results Overview

Percent change was calculated for liver volumes using the equation=\[(12 month value-baseline value)/baseline value\]\*100\*12/12 month

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 48 participants
• Primary outcome timeframe: baseline , 12 month
• Results posted on: 2020-05-01

Participant Flow

Participant milestones

Measure	Pasireotide LAR (SOM230) Active Pasireotide LAR Pasireotide LAR: Injectible, 60mg per month	Placebo Injection Placebo: To be injected once per month
Overall Study STARTED	33	15
Overall Study COMPLETED	29	12
Overall Study NOT COMPLETED	4	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pasireotide LAR in Severe Polycystic Liver Disease

Baseline characteristics by cohort

Measure	Pasireotide LAR (SOM230) n=33 Participants Active Pasireotide LAR Pasireotide LAR: Injectible, 60mg per month	Placebo Injection n=15 Participants Placebo: To be injected once per month	Total n=48 Participants Total of all reporting groups
Age, Continuous	50.16 years STANDARD_DEVIATION 8.63 • n=5 Participants	51.40 years STANDARD_DEVIATION 7.97 • n=7 Participants	50.55 years STANDARD_DEVIATION 8.37 • n=5 Participants
Sex: Female, Male Female	31 Participants n=5 Participants	12 Participants n=7 Participants	43 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Race/Ethnicity, Customized White	29 Participants n=5 Participants	15 Participants n=7 Participants	44 Participants n=5 Participants
Race/Ethnicity, Customized African American	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Other	3 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants
Region of Enrollment United States	33 participants n=5 Participants	15 participants n=7 Participants	48 participants n=5 Participants

PRIMARY outcome

Timeframe: baseline , 12 month

Percent change was calculated for liver volumes using the equation=[(12 month value-baseline value)/baseline value]*100*12/12 month

Outcome measures

Measure	Pasireotide LAR (SOM230) n=29 Participants Active Pasireotide LAR Pasireotide LAR: Injectible, 60mg per month	Placebo Injection n=12 Participants Placebo: To be injected once per month
Change in Liver Volume	-3.36 percentage of change Standard Deviation 7.33	6.29 percentage of change Standard Deviation 6.97

PRIMARY outcome

Timeframe: baseline to 12 months

Percent change was calculated for kidney volumes using the equation=[(12 month value-baseline value)/baseline value]*100*12/12 month

Outcome measures

Measure	Pasireotide LAR (SOM230) n=29 Participants Active Pasireotide LAR Pasireotide LAR: Injectible, 60mg per month	Placebo Injection n=12 Participants Placebo: To be injected once per month
Change in Kidney Volume	-1.37 percentage of change Standard Deviation 3.52	3.85 percentage of change Standard Deviation 4.49

SECONDARY outcome

Timeframe: Baseline, 12 months

eGFR was measured using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation. This value at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100

Outcome measures

Measure	Pasireotide LAR (SOM230) n=29 Participants Active Pasireotide LAR Pasireotide LAR: Injectible, 60mg per month	Placebo Injection n=12 Participants Placebo: To be injected once per month
Percentage Change in Estimated Glomerular Filtration Rate (eGFR)	-0.3 percentage of change Standard Deviation 14	-2.2 percentage of change Standard Deviation 17.7

SECONDARY outcome

Timeframe: Baseline, 12 months

Serum creatinine level at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100

Outcome measures

Measure	Pasireotide LAR (SOM230) n=29 Participants Active Pasireotide LAR Pasireotide LAR: Injectible, 60mg per month	Placebo Injection n=12 Participants Placebo: To be injected once per month
Percentage Change in Serum Creatinine	1.8 percentage of change Standard Deviation 12	3.4 percentage of change Standard Deviation 15.6

SECONDARY outcome

Timeframe: Baseline, 12 months

Blood glucose (mg/dLb) level at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100

Outcome measures

Measure	Pasireotide LAR (SOM230) n=29 Participants Active Pasireotide LAR Pasireotide LAR: Injectible, 60mg per month	Placebo Injection n=12 Participants Placebo: To be injected once per month
Percent Change in Blood Glucose	39 percentage of change Standard Deviation 30	2.2 percentage of change Standard Deviation 15.2

SECONDARY outcome

Timeframe: Baseline, 12 months

Hemoglobin A1C level at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100

Outcome measures

Measure	Pasireotide LAR (SOM230) n=29 Participants Active Pasireotide LAR Pasireotide LAR: Injectible, 60mg per month	Placebo Injection n=12 Participants Placebo: To be injected once per month
Percentage Change in Hemoglobin A1C	18 percentage of change Standard Deviation 11	1.6 percentage of change Standard Deviation 3.3

SECONDARY outcome

Timeframe: Baseline, 12 months

Heart rate, measured in beats per minute (BPM), at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100

Outcome measures

Measure	Pasireotide LAR (SOM230) n=29 Participants Active Pasireotide LAR Pasireotide LAR: Injectible, 60mg per month	Placebo Injection n=12 Participants Placebo: To be injected once per month
Percentage Change in Heart Rate	-0.15 percentage of change Standard Deviation 0.2	-0.1 percentage of change Standard Deviation 0.1

SECONDARY outcome

Timeframe: Baseline, 12 months

Population: Data for 19 participants in the pasireotide LAR (SOM230) group and 9 participants from the placebo injection group was available for analysis.

Measured using the SF-36 health survey, which consist of eight subscales each scored on a range of 0 to 100 (0=worst imaginable, 100=best imaginable). Change calculated from baseline = 12 month value-baseline value

Outcome measures

Measure	Pasireotide LAR (SOM230) n=19 Participants Active Pasireotide LAR Pasireotide LAR: Injectible, 60mg per month	Placebo Injection n=9 Participants Placebo: To be injected once per month
Change in Quality of Life Physical functioning	4.7 units on a scale Standard Deviation 16	-1 units on a scale Standard Deviation 8
Change in Quality of Life Physical role	7.9 units on a scale Standard Deviation 36	-3 units on a scale Standard Deviation 38
Change in Quality of Life Bodily pain	5.5 units on a scale Standard Deviation 22	7 units on a scale Standard Deviation 12
Change in Quality of Life General health	-6.2 units on a scale Standard Deviation 14	2 units on a scale Standard Deviation 15
Change in Quality of Life Vitality	4.5 units on a scale Standard Deviation 14	-2 units on a scale Standard Deviation 17
Change in Quality of Life Social functioning	0.0 units on a scale Standard Deviation 18	-3 units on a scale Standard Deviation 8
Change in Quality of Life Role emotional	0.0 units on a scale Standard Deviation 22	11 units on a scale Standard Deviation 5
Change in Quality of Life Mental health	1.5 units on a scale Standard Deviation 12	-1.8 units on a scale Standard Deviation 11

Adverse Events

Pasireotide LAR (SOM230)

Serious events: 4 serious events

Other events: 33 other events

Deaths: 0 deaths

Placebo Injection

Serious events: 1 serious events

Other events: 15 other events

Deaths: 1 deaths

Serious adverse events

Measure	Pasireotide LAR (SOM230) n=33 participants at risk Active Pasireotide LAR Pasireotide LAR: Injectible, 60mg per month	Placebo Injection n=15 participants at risk Placebo: To be injected once per month
Hepatobiliary disorders Ruptured liver cyst	3.0% 1/33 • Number of events 1 • Adverse Events were collected from baseline to 12 months	0.00% 0/15 • Adverse Events were collected from baseline to 12 months
Hepatobiliary disorders Elevated alkaline phosphatase	3.0% 1/33 • Number of events 1 • Adverse Events were collected from baseline to 12 months	0.00% 0/15 • Adverse Events were collected from baseline to 12 months
Cardiac disorders Prolonged QT>480msec	3.0% 1/33 • Number of events 1 • Adverse Events were collected from baseline to 12 months	0.00% 0/15 • Adverse Events were collected from baseline to 12 months
General disorders Right upper quadrant pain	3.0% 1/33 • Number of events 1 • Adverse Events were collected from baseline to 12 months	0.00% 0/15 • Adverse Events were collected from baseline to 12 months
Gastrointestinal disorders Abdominal pain	3.0% 1/33 • Number of events 1 • Adverse Events were collected from baseline to 12 months	0.00% 0/15 • Adverse Events were collected from baseline to 12 months
General disorders Ascites	3.0% 1/33 • Number of events 1 • Adverse Events were collected from baseline to 12 months	0.00% 0/15 • Adverse Events were collected from baseline to 12 months
Surgical and medical procedures Abdominal hernia repair	0.00% 0/33 • Adverse Events were collected from baseline to 12 months	6.7% 1/15 • Number of events 1 • Adverse Events were collected from baseline to 12 months

Other adverse events

Measure	Pasireotide LAR (SOM230) n=33 participants at risk Active Pasireotide LAR Pasireotide LAR: Injectible, 60mg per month	Placebo Injection n=15 participants at risk Placebo: To be injected once per month
Metabolism and nutrition disorders Hyperglycemia	78.8% 26/33 • Number of events 26 • Adverse Events were collected from baseline to 12 months	26.7% 4/15 • Number of events 4 • Adverse Events were collected from baseline to 12 months
Gastrointestinal disorders Diarrhea	51.5% 17/33 • Number of events 17 • Adverse Events were collected from baseline to 12 months	53.3% 8/15 • Number of events 8 • Adverse Events were collected from baseline to 12 months
Nervous system disorders Dizziness	30.3% 10/33 • Number of events 10 • Adverse Events were collected from baseline to 12 months	20.0% 3/15 • Number of events 3 • Adverse Events were collected from baseline to 12 months
Gastrointestinal disorders Pain abdomen	33.3% 11/33 • Number of events 11 • Adverse Events were collected from baseline to 12 months	6.7% 1/15 • Number of events 1 • Adverse Events were collected from baseline to 12 months
General disorders Fatigue	39.4% 13/33 • Number of events 13 • Adverse Events were collected from baseline to 12 months	20.0% 3/15 • Number of events 3 • Adverse Events were collected from baseline to 12 months
Endocrine disorders Diabetes	59.4% 19/32 • Number of events 19 • Adverse Events were collected from baseline to 12 months	6.7% 1/15 • Number of events 1 • Adverse Events were collected from baseline to 12 months
Gastrointestinal disorders Nausea	27.3% 9/33 • Number of events 9 • Adverse Events were collected from baseline to 12 months	20.0% 3/15 • Number of events 3 • Adverse Events were collected from baseline to 12 months
Cardiac disorders Bradycardia	30.3% 10/33 • Number of events 10 • Adverse Events were collected from baseline to 12 months	6.7% 1/15 • Number of events 1 • Adverse Events were collected from baseline to 12 months
General disorders Alopecia	27.3% 9/33 • Number of events 9 • Adverse Events were collected from baseline to 12 months	0.00% 0/15 • Adverse Events were collected from baseline to 12 months
Nervous system disorders Headache	15.2% 5/33 • Number of events 5 • Adverse Events were collected from baseline to 12 months	20.0% 3/15 • Number of events 3 • Adverse Events were collected from baseline to 12 months

Additional Information

Marie C. Hogan, M.D., Ph.D.

Mayo Clinic

Phone: 507-284-3479

Email: Hogan.Marie@mayo.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place