A Trial to Assess the Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With PLD

Phase 3Active Not RecruitingNCT05281328

Camurus AB71 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Polycystic Liver Disease

Interventions

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (11)

Mayo Clinic

Rochester, Minnesota, United States

Mount Sinai Hospital

New York, New York, United States

The New York Presbyterian Hospital

New York, New York, United States

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Bon Secours Richmond Community Hospital

Richmond, Virginia, United States

University Hospitals KU Leuven

Leuven, Belgium

Hannover Medical School

Hanover, Germany

Universitätsklinikum Leipzig

Leipzig, Germany

Universitaetsklinikum Müenster

Münster, Germany

...and 1 more locations

Outcomes

Primary Outcomes

Height-adjusted total liver volume (htTLV)

Change from baseline to Week 53 in htTLV as determined by MRI volumetry

Time frame: From screening until treatment week 53

Secondary Outcomes

PLD symptom (PLD-S) score

Key secondary endpoint. Change from baseline to Week 53 in the PLD-S measure score

Time frame: From screening to week 53

htTLV

Change from baseline in htTLV as determined by MRI volumetry

Time frame: From screening until treatment weeks 13, 25, 77, 125 and 173

PLD-S

Change from baseline in the PLD-S measure score

Time frame: From screening to weeks 13, 21, 25, 39, 77, 101, 125, 149 and 173

Height-adjusted total kidney volume (htTKV)

Change from baseline in htTKV as determined by MRI volumetry

Time frame: From screening until treatment weeks 13, 25, 53, 77, 125 and 173

Total liver cyst volume

Change from baseline in total liver cyst volume determined by MRI volumetry

Time frame: From screening to treatment weeks 13, 25, 53, 77, 125 and 173

Estimated glomerular filtration rate (eGFR)

Change from baseline in eGFR, assessed by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) cystatin C equation using serum concentrations of creatinine and cystatin C

Time frame: From treatment week 1 to weeks 13, 25, 53, 65, 77, 101, 125, 149 and 173

PLD impact (PLD-I) score

Change from baseline in the PLD-I measure score

Time frame: From screening to weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173

Clinical Global Impression of Severity (CGI-S) score

Change from baseline in the CGI-S score

Time frame: From treatment week 1 to weeks 13, 21, 25, 53, 77, 101, 125, 149 and 173

Patient Global Impression of Severity (PGI-S) score

Change from baseline in the PGI-S score

Time frame: From screening to weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173

Patient Global Impression of Change (PGI-C) score

Change from baseline in the PGI-C score

Time frame: At treatment weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173

Short Form-36 (SF-36) score

Change from baseline in the SF-36 score

Time frame: From treatment week 1 to weeks 25, 53, 77, 101, 125, 149 and 173

Polycystic Liver Disease Questionnaire (PLD-Q)

Change from baseline in the PLD-Q score

Time frame: From treatment week 1 to weeks 25, 53, 77, 101, 125, 149 and 173

Adverse events (AEs)

Incidence of AEs

Time frame: From screening to the safety follow-up, assessed up to approximately 43 months