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

Polycystic Liver Disease Clinical Trial

— POSITANO  

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05281328
• Other study ID #: HS-20-677
• Secondary ID: 2021-003764-27
• Status: Active, not recruiting
• Phase: Phase 2/Phase 3
• Start date: June 28, 2022
• Est. completion date: August 2025

Study information

• Verified date: February 2024
• Source: Camurus AB
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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 24-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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 71
• Est. completion date: August 2025
• Est. primary completion date: February 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cysts
• Liver Diseases
• Polycystic Liver Disease

Intervention

Drug:
• CAM2029
SC injection using a pre-filled pen
• Placebo
SC injection using a pre-filled pen

Locations

Country	Name	City	State
Belgium	University Hospitals KU Leuven	Leuven
Germany	Hannover Medical School	Hannover
Germany	Universitätsklinikum Leipzig	Leipzig
Germany	Universitaetsklinikum Müenster	Münster
Netherlands	Radboud UMC, Department of Gastroenterology and Hepatology	Nijmegen
United States	University of Texas Southwestern Medical Center	Dallas	Texas
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	Bon Secours Richmond Community Hospital	Richmond	Virginia
United States	Mayo Clinic	Rochester	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Camurus AB

Countries where clinical trial is conducted

United States,  Belgium,  Germany,  Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Height-adjusted total liver volume (htTLV)	Change from baseline to Week 53 in htTLV as determined by MRI volumetry	From screening until treatment week 53
Secondary	PLD symptom (PLD-S) score	Key secondary endpoint. Change from baseline to Week 53 in the PLD-S measure score	From screening to week 53
Secondary	htTLV	Change from baseline in htTLV as determined by MRI volumetry	From screening until treatment weeks 13, 25 and 77
Secondary	PLD-S	Change from baseline in the PLD-S measure score	From screening to weeks 13, 21, 25, 39 and 77
Secondary	Height-adjusted total kidney volume (htTKV)	Change from baseline in htTKV as determined by MRI volumetry	From screening until treatment weeks 13, 25, 53 and 77
Secondary	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	From treatment week 1 to weeks 13, 25, 53, 65 and 77
Secondary	PLD impact (PLD-I) score	Change from baseline in the PLD-I measure score	From screening to weeks 13, 21, 25, 39, 53 and 77
Secondary	Clinical Global Impression of Severity (CGI-S) score	Change from baseline in the CGI-S score	From treatment week 1 to weeks 13, 21, 25, 53 and 77
Secondary	Patient Global Impression of Severity (PGI-S) score	Change from baseline in the PGI-S score	From screening to weeks 13, 21, 25, 39, 53 and 77
Secondary	Patient Global Impression of Change (PGI-C) score	Change from baseline in the PGI-C score	At treatment weeks 13, 21, 25, 39, 53 and 77
Secondary	Short Form-36 (SF-36) score	Change from baseline in the SF-36 score	From treatment week 1 to weeks 25, 53 and 77
Secondary	Polycystic Liver Disease Questionnaire (PLD-Q)	Change from baseline in the PLD-Q score	From treatment week 1 to weeks 25, 53 and 77
Secondary	Adverse events (AEs)	Incidence of AEs	From screening to the safety follow-up, assessed up to approximately 21 months