A randomized, placebo-controlled, double-blind, multi-center trial to assess efficacy and safety of octreotide subcutaneous depot (CAM2029) in patients with symptomatic polycystic liver disease

Phase 1

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

• Registration Number: CTIS2023-505313-24-00
• Lead Sponsor: Camurus AB

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-06-16
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

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

Inclusion Criteria

Voluntary and valid written informed consent to participate in the trial provided by the patient before any trial related procedures are performed, Mannelijke of vrouwelijke patiënt, = 18 jaar bij de keuring, Diagnose polycysteuze leverziekte (geassocieerd met ADPKD of geïsoleerd zoals bij ADPLD) met htTLV = 1800 ml/m bij de keuring, Aanwezigheid van ten minste één van de volgende symptomen gerelateerd aan polycysteuze leverziekte binnen twee weken vóór de keuring: opgeblazen gevoel, vol gevoel in de buik, gebrek aan eetlust, snel nadat wordt begonnen met eten een vol gevoel krijgen, zure oprispingen, misselijkheid, pijn of druk in de ribbenkast, pijn in de zij, buikpijn, rugpijn, kortademigheid na lichamelijke inspanning, beperkte mobiliteit, bezorgdheid over het dikker worden van de buik, ontevredenheid over de omvang van de buik, Not a candidate for, or not willing to undergo, surgical intervention for hepatic cysts during the trial, Female patients of childbearing potential must be willing to use an acceptable method of contraception from screening and during the entire trial, Male patients must be willing to use condom as method of contraception from screening and throughout the trial unless they have been sterilized by vasectomy (with an appropriate post-vasectomy documentation of the absence of sperm in the ejaculate)

Exclusion Criteria

Surgical intervention for PLD within 3 months before screening. For sclerotherapy patients, at least 6 months before 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 per www.crediblemeds.org 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 (QTcF) >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, Clinically significant laboratory abnormalities, which in the opinion of the Investigator may prevent the patient from safely participating in the trial, History of solid organ transplantation. Exception for kidney transplant patients, Any known allergy, hypersensitivity or intolerance to octreotide or any related drug, or other components of CAM2029, or history of any drug hypersensitivity or intolerance that, in the opinion of the Investigator, would compromise the safety of the patient, Contraindications to, or interference with, MRI assessments, as dictated by local hospital regulations, Previously treated/randomized in the current clinical trial, Treatment with an Somatostatin analogue (SSA) within 3 months before screening, Participation in any other clinical trial to test an investigational drug or device within the last 30 days before screening or during the trial, Any other contraindicated serious medical condition that, in the Investigator’s opinion, may prevent the patient from safely participating in the trial, Any other current or prior medical condition that may interfere with the conduct of the trial or the evaluation of its results in the opinion of the Investigator, Unwilling or unable to comply with the requirements of the protocol or in a situation or condition that, in the opinion of the Investigator, may interfere with participation in the trial, On the staff, affiliated with, or a family member of the personnel directly involved with this trial, Non-responsive to pr

Study & Design

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