This study is being conducted to evaluate efficacy and safety of Octreotide compared with Placebo via subcutaneous depot route in subjects with polycystic liver disease

Phase 1

• Conditions: Polycystic liver disease; Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

• Registration Number: EUCTR2021-003764-27-NL
• Lead Sponsor: Camurus AB

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-04-12
• Last Update Posted: 8 April 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 69

Inclusion Criteria

• Male or female patient, =18 years at screening
• Diagnosis of PLD (associated with ADPKD or isolated as in ADPLD) with htTLV =1800 mL/m at screening
• Presence of at least 1 of the following PLD-related symptoms within 2 weeks before screening: bloating, fullness in abdomen, lack of appetite, feeling full quickly after beginning to eat, acid reflux, nausea, rib cage pain or pressure, pain in side, abdominal pain, back pain, shortness of breath after physical exertion, limited in mobility, concern about abdomen getting larger, dissatisfied by the size of abdomen
• Not a candidate for, or not willing to undergo, surgical intervention for hepatic cysts during the trial

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 69
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion Criteria

• Surgical intervention for PLD within 3 months before screening. For
sclerotherapy patients, at least 6 months before screening
• Treatment with a somatostatin analogue (SSA) within 3 months before screening
• Non-responsive to previous treatment of PLD with an SSA as per the Investigator’s assessment
• Symptomatic cholelithiasis within 3 months before screening or previous medical history of cholelithiasis induced by SSAs unless treated with cholecystectomy
• Presence of extrahepatic cysts that, in the Investigator’s opinion, may prevent the patient from safely participating in the trial
• Severe kidney disease, as defined by eGFR <30 mL/min/1.73 m2
• Severe liver disease defined as liver cirrhosis of Child-Pugh class C
• Use of oral contraceptives or estrogen supplementation within 3 months before screening
• Poorly controlled diabetes (hemoglobin A1c =10%) at screening
• Patients with a known history of hypothyroidism, unless they have been on adequate and stable replacement thyroid hormone therapy for at least 3 months before the first dose of the IMP.
• Uncontrolled hypertension defined by a systolic blood pressure of >160 mmHg and/or diastolic blood pressure of >100 mmHg at screening
• History of significant cardiac disease or current diagnosis of cardiac disease indicating significant risk of safety for patients participating in the trial, such as uncontrolled or significant cardiac disease, including any of the following:
a. History of myocardial infarction, angina pectoris or coronary artery bypass graft within 6 months before screening
b. Clinically significant cardiac arrhythmias (e.g. ventricular tachycardia), complete left bundle branch block or high-grade atrioventricular block (e.g. bifascicular block, Mobitz type II and third-degree atrioventricular block)
c. Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome, or any of the following:
i. Risk factors for Torsades de Pointes including uncorrected hypokalemia or hypomagnesemia, history of cardiac failure or history of clinically significant/symptomatic bradycardia
ii. Treatment with concomitant medication(s) with a Known risk of Torsades de Pointes that cannot be discontinued or replaced by safe alternative medication at least 5 half-lives or 7 days (whichever is longer) before the first dose of IMP
iii. Patients with a baseline QTc interval corrected by Fridericia's formula >450 msec for males and >470 msec for females at screening
• Patients with vascular compromise, including, but not limited to, mesenteric thrombosis, portal hypertension and thrombocytopenia (platelet counts less than 100x109/L)
• Pregnant, lactating or planning to be pregnant during the trial
• History of solid organ transplantation. Exception for kidney transplant patients
• Contraindications to, or interference with, MRI assessments, as dictated by local hospital regulations

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified