Polycystic liver disease
Conditions
Interventions
CAM2029
During the Treatment Period, CAM2029 10 mg (0.5 mL) will be administered as an
SC injection using a pre-filled pen once weekly in treatment arm 1 and every 2
weeks in treatment arm 2 (weekly
Sponsors
Camurus AB
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: - Male or female patient, >=18 years at screening - Diagnosis of PLD (associated with ADPKD or isolated as in ADPLD) as defined by htTLV >=2500 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 - 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
Exclusion criteria: • Surgical intervention for PLD within 3 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 • 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 =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 • Contraindications to, or interference with, MRI assessments, as dictated by local hospital regulations
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline to Week 53 in height-adjusted total liver volume (htTLV) as determined by magnetic resonance imaging (MRI) volumetry | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline to Week 53 in the Polycystic Liver Disease Symptoms (PLD-S) measure score | — |
Countries
Netherlands
Outcome results
None listed