The purpose of the trial is to compare the effectiveness and safety of 2 treatment regimens of CAM2029 (given weekly or every 2 weeks) to placebo in participants with symptomatic PLD, either isolated as in autosomal dominant PLD (ADPLD) or associated with autosomal dominant polycystic kidney disease (ADPKD).
In the Treatment Period of the trial, participants will be allocated at random to 1 of the 3 treatment arms in a 1:1:1 ratio. After completing the Treatment Period (53 weeks) participants may proceed to a 120-week open-label extension part of the trial and then only receive the same CAM2029 treatment.
The active ingredient in CAM2029, octreotide, is administered as a subcutaneous depot using Camurus' FluidCrystal® technology.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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 ≥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
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
* Systematic 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
* 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
Primary outcome measure(s)
Height-adjusted Total Liver Volume (htTLV) — From screening until treatment week 53 Magnetic resonance imaging (MRI) was used to measure the total liver volume (TLV) at baseline and at Week 53 and was adjusted to the participant's height. The change from baseline to Week 53 was calculated as the difference between htTLV values at Week 53 and baseline. The difference in treatment effect between the groups was presented as a ratio expressed in percentage.
Trial sites (11)
Facility
City
Region
Status
Mayo Clinic
Rochester
Minnesota
Mount Sinai Hospital
New York
New York
The New York Presbyterian Hospital
New York
New York
Hospital of the University of Pennsylvania
Philadelphia
Pennsylvania
University of Texas Southwestern Medical Center
Dallas
Texas
Bon Secours Richmond Community Hospital
Richmond
Virginia
University Hospitals KU Leuven
Leuven
Belgium
Hannover Medical School
Hanover
Germany
Universitätsklinikum Leipzig
Leipzig
Germany
Universitaetsklinikum Müenster
Münster
Germany
Radboud UMC, Department of Gastroenterology and Hepatology
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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