The purpose of this study is to compare SOM230 treatment to placebo. The investigators will also assess the efficacy and safety of SOM230 in reducing total liver volume and improving quality of life.
Pasireotide (SOM230) is a novel multi-receptor-targeted analog that has high affinity for four of the five SST receptor subtypes (SSTr1, SSTr2, SSTr3 and SSTr5); it has a 40-fold higher affinity and 158-fold higher functional activity for the SST5 receptor than octreotide. Because of its broad receptor binding profile, pasireotide may be more potent in Polycystic Liver Disease (PLD) than octreotide. In this randomized double blind placebo controlled trial the investigators will compare SOM230 treatment to placebo for 12 months in patients with PLD. The primary endpoints will be assessed at 12 months and patients receiving placebo then crossed over to SOM230, permitting all participants to receive SOM230 for the subsequent two years. Magnetic resonance imaging (MRI) will be used to assess liver volume - the primary endpoint, which will be assessed at baseline, end of years 1 and 3. This study will assess the efficacy and safety of SOM230 in reducing total liver volume and improving quality of life over 12 months. (The investigators will not be assessing efficacy at 24 months.) The therapy way be effective in PLD but also may prove to be effective for many more patients with Polycystic Kidney Disease (PKD) which will be evaluated using eGFR and kidney volume using MRI. The investigators plan to add other sub-sites in other locations.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
48
Injectible, 60mg per month
To be injected once per month
Mayo Clinic
Rochester, Minnesota, United States
Change in Liver Volume
Percent change was calculated for liver volumes using the equation=\[(12 month value-baseline value)/baseline value\]\*100\*12/12 month
Time frame: baseline , 12 month
Change in Kidney Volume
Percent change was calculated for kidney volumes using the equation=\[(12 month value-baseline value)/baseline value\]\*100\*12/12 month
Time frame: baseline to 12 months
Percentage Change in Estimated Glomerular Filtration Rate (eGFR)
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
Time frame: Baseline, 12 months
Percentage Change in Serum Creatinine
Serum creatinine level at baseline and 12 months was used to calculate the percentage change by \[12 month value-baseline value)/baseline value\]\*100
Time frame: Baseline, 12 months
Percent Change in Blood Glucose
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
Time frame: Baseline, 12 months
Percentage Change in Hemoglobin A1C
Hemoglobin A1C level at baseline and 12 months was used to calculate the percentage change by \[12 month value-baseline value)/baseline value\]\*100
Time frame: Baseline, 12 months
Percentage Change in Heart Rate
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
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
Time frame: Baseline, 12 months
Change in Quality of Life
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
Time frame: Baseline, 12 months