Reduction of polycystic liver volume by treating with octreotide, whether or not combined with everolimus. Assessing whether combination therapy of everolimus and octreotide gives a bigger reduction of polycystic liver volume than octreotide…
ID
Source
Brief title
Condition
- Hepatic and hepatobiliary disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Reduction of liver volume from baseline to 48 weeks, as determined by CT scan.
Secondary outcome
Change in symptoms, as determined by GI symptom questionnaire, proportion of
patients that have any reduction of liver volume after 48 weeks, and all
adverse events during these 48 weeks.
Background summary
Polycystic lvier disease is a disorder which is accompanied by the presence of
at least 20 liver cysts and sometimes a positive family history for liver
cysts. Until recent, no noninvasive treatment was available, but recent trials
have shown that somatostatin analogues reduce liver volume with 2-5% every
year. In trials with polycystic liver and kidney patients that received a
kidney transplant, was seen that sirolimus, a mTOR inhibitor that is given for
rejection prevention, reduced polycystic livers with almost 15%. In this trial
we want to investigate whether the combination of everolimus and octreotide
gives a bigger reduction of liver volume than octreotide monotherapy.
Study objective
Reduction of polycystic liver volume by treating with octreotide, whether or
not combined with everolimus. Assessing whether combination therapy of
everolimus and octreotide gives a bigger reduction of polycystic liver volume
than octreotide monotherapy.
Study design
This will be a randomized, open-label, parallel, clinical trial.
Intervention
The patients will be randomized to either the combination
(everolimus+octreotide) or the monotherapy (octreotide) group. All patients
will receive octreotide 40 mg every 28 days. If they are in the combination
group, they will also receive an initial dose of 2.5 mg of everolimus daily,
which will be adjusted upon trough levels of everolimus if necessary.
Study burden and risks
The treatment and CT scans go with certain risks, but in earlier trials with
these drugs, the side effects were relatively mild and patients had a
subjective reduction of their complaints. In conclusion, treatment with
somatostatin analogues seems an elegant alternative to surgical procedures that
go with considerable morbidity.
Postbus 9101
6500 HB Nijmegen
NL
Postbus 9101
6500 HB Nijmegen
NL
Listed location countries
Age
Inclusion criteria
• 18 - 70 years of age
• Polycystic liver disease (PCLD), defined as >= 20 liver cysts and total liver volume must be at least 2500 mL.
• Informed consent, patients are willing and able to comply with the study drug regimen and all other study requirements.
Exclusion criteria
- Use of estrogens
- Pregnancy or breastfeeding at baseline/during study
- Severe cardiac or pulmonary disease
- Intervention < 3 months before baseline
- Kidney transplantation
- Symptomatic gallstones
- Untreated hypercholesterolemie (fasting cholesterol > 8 mmol/L)
- Granulocytopenia (white blood cell < 3,000/mm3) or thrombocytopenia (platelets < 100,000/mm3)
- Infection with hepatitis B, hepatitis C, HIV or TBC
- Mental illness that interferes with ability to comply with study protocol
- Drug or alcohol abuse within one year before baseline
- Co-medication that interferes with study medication
- Hypersensitivity to everolimus or octreotide
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2009-016640-39-NL |
| CCMO | NL30160.091.09 |