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

Primary Purpose

Polycystic Liver Disease

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
CAM2029
Placebo
Sponsored by
Camurus AB
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Polycystic Liver Disease focused on measuring Polycystic liver, PLD, ADPLD, ADPKD, PCLD, height-adjusted total liver volume, htTLV, CAM2029, POSITANO

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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 ≥2500 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
  • 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

Sites / Locations

  • Mayo ClinicRecruiting
  • The New York Presbyterian HospitalRecruiting
  • Hospital of the University of PennsylvaniaRecruiting
  • University of Texas Southwestern Medical CenterRecruiting
  • Bon Secours Richmond Community HospitalRecruiting
  • Universitair Ziekenhuis GentRecruiting
  • University Hospitals KU LeuvenRecruiting
  • Hannover Medical SchoolRecruiting
  • Radboud UMC, Department of Gastroenterology and HepatologyRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

CAM2029 once weekly

CAM2029 once every 2 weeks

Placebo

Arm Description

0.5 mL CAM2029 10 mg, subcutaneous (SC) injection, once weekly

0.5 mL CAM2029 10 mg, SC injection, every 2 weeks and 0.5 mL placebo, SC injection, once every 2 weeks (alternating with CAM2029 dosing)

0.5 mL placebo, SC injection, once weekly

Outcomes

Primary Outcome Measures

Height-adjusted total liver volume (htTLV)
Change from baseline to Week 53 in htTLV as determined by MRI volumetry

Secondary Outcome Measures

PLD symptom (PLD-S) score
Key secondary endpoint. Change from baseline to Week 53 in the PLD-S measure score
htTLV
Change from baseline in htTLV as determined by MRI volumetry
PLD-S
Change from baseline in the PLD-S measure score
Height-adjusted total kidney volume (htTKV)
Change from baseline in htTKV as determined by MRI volumetry
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
PLD impact (PLD-I) score
Change from baseline in the PLD-I measure score
Clinical Global Impression of Severity (CGI-S) score
Change from baseline in the CGI-S score
Patient Global Impression of Severity (PGI-S) score
Change from baseline in the PGI-S score
Patient Global Impression of Change (PGI-C) score
Change from baseline in the PGI-C score
Short Form-36 (SF-36) score
Change from baseline in the SF-36 score
Polycystic Liver Disease Questionnaire (PLD-Q)
Change from baseline in the PLD-Q score
Adverse events (AEs)
Incidence of AEs

Full Information

First Posted
March 7, 2022
Last Updated
August 14, 2023
Sponsor
Camurus AB
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1. Study Identification

Unique Protocol Identification Number
NCT05281328
Brief Title
A Trial to Assess the Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With PLD
Acronym
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
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 28, 2022 (Actual)
Primary Completion Date
December 2024 (Anticipated)
Study Completion Date
June 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Camurus AB

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Polycystic Liver Disease
Keywords
Polycystic liver, PLD, ADPLD, ADPKD, PCLD, height-adjusted total liver volume, htTLV, CAM2029, POSITANO

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
69 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
CAM2029 once weekly
Arm Type
Experimental
Arm Description
0.5 mL CAM2029 10 mg, subcutaneous (SC) injection, once weekly
Arm Title
CAM2029 once every 2 weeks
Arm Type
Experimental
Arm Description
0.5 mL CAM2029 10 mg, SC injection, every 2 weeks and 0.5 mL placebo, SC injection, once every 2 weeks (alternating with CAM2029 dosing)
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
0.5 mL placebo, SC injection, once weekly
Intervention Type
Drug
Intervention Name(s)
CAM2029
Other Intervention Name(s)
octreotide subcutaneous depot
Intervention Description
SC injection using a pre-filled pen
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
SC injection using a pre-filled pen
Primary Outcome Measure Information:
Title
Height-adjusted total liver volume (htTLV)
Description
Change from baseline to Week 53 in htTLV as determined by MRI volumetry
Time Frame
From screening until treatment week 53
Secondary Outcome Measure Information:
Title
PLD symptom (PLD-S) score
Description
Key secondary endpoint. Change from baseline to Week 53 in the PLD-S measure score
Time Frame
From screening to week 53
Title
htTLV
Description
Change from baseline in htTLV as determined by MRI volumetry
Time Frame
From screening until treatment weeks 13, 25 and 77
Title
PLD-S
Description
Change from baseline in the PLD-S measure score
Time Frame
From screening to weeks 13, 21, 25, 39 and 77
Title
Height-adjusted total kidney volume (htTKV)
Description
Change from baseline in htTKV as determined by MRI volumetry
Time Frame
From screening until treatment weeks 13, 25, 53 and 77
Title
Estimated glomerular filtration rate (eGFR)
Description
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 and 77
Title
PLD impact (PLD-I) score
Description
Change from baseline in the PLD-I measure score
Time Frame
From screening to weeks 13, 21, 25, 39, 53 and 77
Title
Clinical Global Impression of Severity (CGI-S) score
Description
Change from baseline in the CGI-S score
Time Frame
From treatment week 1 to weeks 13, 21, 25, 53 and 77
Title
Patient Global Impression of Severity (PGI-S) score
Description
Change from baseline in the PGI-S score
Time Frame
From screening to weeks 13, 21, 25, 39, 53 and 77
Title
Patient Global Impression of Change (PGI-C) score
Description
Change from baseline in the PGI-C score
Time Frame
At treatment weeks 13, 21, 25, 39, 53 and 77
Title
Short Form-36 (SF-36) score
Description
Change from baseline in the SF-36 score
Time Frame
From treatment week 1 to weeks 25, 53 and 77
Title
Polycystic Liver Disease Questionnaire (PLD-Q)
Description
Change from baseline in the PLD-Q score
Time Frame
From treatment week 1 to weeks 25, 53 and 77
Title
Adverse events (AEs)
Description
Incidence of AEs
Time Frame
From screening to the safety follow-up, assessed up to approximately 21 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Camurus AB
Phone
+46 46 286 57 30
Email
medicalinfo@camurus.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Joost Drenth, MD
Organizational Affiliation
Department of Gastroenterology and Hepatology, Radboud UMC Nijmegen, The Netherlands
Official's Role
Principal Investigator
Facility Information:
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Marie Hogan, MD
First Name & Middle Initial & Last Name & Degree
Ryan Helland
Phone
507-422-3998
Email
helland.ryan@mayo.edu
Facility Name
The New York Presbyterian Hospital
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Individual Site Status
Recruiting
Facility Name
Hospital of the University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75380
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
William Lee, MD
Facility Name
Bon Secours Richmond Community Hospital
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23602
Country
United States
Individual Site Status
Recruiting
Facility Name
Universitair Ziekenhuis Gent
City
Gent
ZIP/Postal Code
9000
Country
Belgium
Individual Site Status
Recruiting
Facility Name
University Hospitals KU Leuven
City
Leuven
ZIP/Postal Code
B-3000
Country
Belgium
Individual Site Status
Recruiting
Facility Name
Hannover Medical School
City
Hannover
ZIP/Postal Code
30625
Country
Germany
Individual Site Status
Recruiting
Facility Name
Radboud UMC, Department of Gastroenterology and Hepatology
City
Nijmegen
ZIP/Postal Code
6525 GA
Country
Netherlands
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

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

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