Clinical trial NCT05281328: A Trial to Assess the Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With PLD
Official title: 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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
PHASE3
Conditions: Polycystic Liver Disease
Keywords: Polycystic liver
PLD
ADPLD
ADPKD
PCLD
height-adjusted total liver volume
htTLV
CAM2029
POSITANO
Lead sponsor: Camurus AB
Sponsor class: INDUSTRY
First posted: 2022-03-16
Enrollment: 71
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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 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 ≥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 outcomes:
measure: Height-adjusted total liver volume (htTLV)
timeFrame: From screening until treatment week 53
Secondary outcomes:
measure: PLD symptom (PLD-S) score
timeFrame: From screening to week 53
measure: htTLV
timeFrame: From screening until treatment weeks 13, 25, 77, 125 and 173
measure: PLD-S
timeFrame: From screening to weeks 13, 21, 25, 39, 77, 101, 125, 149 and 173
measure: Height-adjusted total kidney volume (htTKV)
timeFrame: From screening until treatment weeks 13, 25, 53, 77, 125 and 173
measure: Total liver cyst volume
timeFrame: From screening to treatment weeks 13, 25, 53, 77, 125 and 173
measure: Estimated glomerular filtration rate (eGFR)
timeFrame: From treatment week 1 to weeks 13, 25, 53, 65, 77, 101, 125, 149 and 173
measure: PLD impact (PLD-I) score
timeFrame: From screening to weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173
measure: Clinical Global Impression of Severity (CGI-S) score
timeFrame: From treatment week 1 to weeks 13, 21, 25, 53, 77, 101, 125, 149 and 173
measure: Patient Global Impression of Severity (PGI-S) score
timeFrame: From screening to weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173
measure: Patient Global Impression of Change (PGI-C) score
timeFrame: At treatment weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173
measure: Short Form-36 (SF-36) score
timeFrame: From treatment week 1 to weeks 25, 53, 77, 101, 125, 149 and 173
measure: Polycystic Liver Disease Questionnaire (PLD-Q)
timeFrame: From treatment week 1 to weeks 25, 53, 77, 101, 125, 149 and 173
measure: Adverse events (AEs)
timeFrame: From screening to the safety follow-up, assessed up to approximately 43 months
Other outcomes:
Locations:
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: New York
country: United States
facility: Mount Sinai Hospital
state: New York
city: New York
country: United States
facility: The New York Presbyterian Hospital
state: New York
city: Philadelphia
country: United States
facility: Hospital of the University of Pennsylvania
state: Pennsylvania
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Richmond
country: United States
facility: Bon Secours Richmond Community Hospital
state: Virginia
city: Leuven
country: Belgium
facility: University Hospitals KU Leuven
city: Hannover
country: Germany
facility: Hannover Medical School
city: Leipzig
country: Germany
facility: Universitätsklinikum Leipzig
city: Münster
country: Germany
facility: Universitaetsklinikum Müenster
city: Nijmegen
country: Netherlands
facility: Radboud UMC, Department of Gastroenterology and Hepatology
Study officials:
affiliation: Department of Gastroenterology and Hepatology, Radboud UMC Nijmegen, The Netherlands
name: Joost Drenth, MD
role: PRINCIPAL_INVESTIGATOR
References: