Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT05281328

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Notify Me

Key information

Conditions

Polycystic Liver Disease Abnormalities, Multiple Ciliopathies Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Kidney Diseases Kidney Diseases, Cystic Male Urogenital Diseases Polycystic Kidney Diseases Polycystic Kidney, Autosomal Dominant Urogenital Diseases Urologic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

University Hospitals KU Leuven, Leuven, Belgium

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

CAM2029

Drug

SC injection using a pre-filled pen

Other names: octreotide subcutaneous depot

Placebo

Drug

SC injection using a pre-filled pen

Primary outcomes

  1. Height-adjusted Total Liver Volume (htTLV)

    Time frame: 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.

Secondary outcomes

  1. PLD Symptom (PLD-S) Score

    Time frame: From screening to Week 53

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

  2. htTLV

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

    Change from baseline in htTLV as determined by MRI volumetry

  3. PLD-S

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

    Change from baseline in the PLD-S measure score

  4. Height-adjusted Total Kidney Volume (htTKV)

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

    Change from baseline in htTKV as determined by MRI volumetry

  5. Total Liver Cyst Volume

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

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

  6. Estimated Glomerular Filtration Rate (eGFR)

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

    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

  7. PLD Impact (PLD-I) Score

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

    Change from baseline in the PLD-I measure score

  8. Clinical Global Impression of Severity (CGI-S) Score

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

    Change from baseline in the CGI-S score

  9. Patient Global Impression of Severity (PGI-S) Score

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

    Change from baseline in the PGI-S score

  10. Patient Global Impression of Change (PGI-C) Score

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

    Change from baseline in the PGI-C score

  11. Short Form-36 (SF-36) Score

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

    Change from baseline in the SF-36 score

  12. Polycystic Liver Disease Questionnaire (PLD-Q)

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

    Change from baseline in the PLD-Q score

  13. Adverse Events (AEs)

    Time frame: From screening to the safety follow-up, assessed up to approximately 47 months.

    Incidence of AEs

Sponsors and collaborators

Lead sponsor

Camurus AB

Industry

Registry information

Official study 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

Acronym: POSITANO

Important dates

Study start
2022
Primary completion
2025
Study completion
2027
First posted
Mar 16, 2022
Registry last updated
Jun 1, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.