A randomized, double-blind, placebo-controlled clinical trial assessing the efficacy of combining pasireotide with aspiration sclerotherapy to improve volume reduction of dominant hepatic cysts

Liver cysts are fluid filled cavities located in the liver. They are present
in 2-11% of the general population, typically not causing any symptoms or
complications. However, in a small subset of patients complaints of pain,
abdominal fullness and distension, dyspnea and nausea occur. Currently,
aspiration and sclerotherapy is the treatment of choice in symptomatic patients
with a large dominant liver cyst. However, studies reported early fluid
reaccumulation and relative high recurrence rates of cyst growth after
aspiration sclerotherapy ultimately leading to re-interventions.

In this respect, somatostatin analogues are promising agents known for its
volume reducing effect in patients with polycystic liver disease. In this study
we want to evaluate the effect of combining aspiration sclerotherapy with the
multi-receptor binding, long-acting somatostatin analogue pasireotide. We
hypothesize that administrating pasireotide before and after aspiration
sclerotherapy could prevent early fluid reaccumulation and thereby result in a
greater reduction of cyst diameter. Moreover, we expect a lower rate of cyst
recurrence and subsequently lower need for re-interventions.

The primary objective is to minimize fluid reaccumulation in the hepatic cyst
after aspiration sclerotherapy in order to reduce cyst size. The secondary
objectives are to reduce symptoms, improve health-related quality of life
(HRQL), and reduce cyst recurrence to prevent re-interventions.

Randomized, double-blind, placebo-controlled intervention study.

The subjects will be randomized (1:1) into two groups. Both groups will undergo
aspiration sclerotherapy following the standard procedure. The intervention
group will additionally receive two injections of 60 mg pasireotide long-acting
release (LAR) intramuscularly: the first injection 14 days before and the
second injection 14 days after the intervention. Patients in the placebo arm
will receive two injections of saline solution corresponding to the scheme of
the intervention group.

Aspiration sclerotherapy and the accompanying physical examination,
ultrasonography and blood samples prior to the procedure are regarded as the
indicated standard procedure of dominant symptomatic liver cysts. When compared
to aspiration sclerotherapy alone, the burden and risks associated with
participation of this study are:
- Possible side effects or adverse events related to the administration of
long-acting pasireotide.
- Two to three additional site visits in comparison to standard care after
aspiration sclerotherapy
- Electrocardiogram (ECG)
- Three questionnaires (gastro-intestinal and HRQL) are assessed at baseline,
4, 12 and 24 weeks after aspiration sclerotherapy. Importantly, these
questionnaires are relatively short and are not considered having emotional
impact on the patient.

The potential benefit of participation is that combining pasireotide with
aspiration sclerotherapy might reduce the cyst diameter more effectively than
the conventional aspiration sclerotherapy. We expect that this will
subsequently reduce symptoms and improve HRQL. Moreover, the rate of cyst
recurrence is expected to be lower in the treatment arm and thereby
re-treatment can be postponed or even avoided.