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

Phase 2

Active, not recruiting

• Conditions: Polycystic Liver Disease
• Interventions: Drug: CAM2029; Drug: Placebo

• Registration Number: NCT05281328
• Lead Sponsor: Camurus AB

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-03-07
• Study First Submitted QC: 2022-03-07
• Study First Posted: 2022-03-16
• Study Start: 2022-06-28
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-20
• Last Update Posted: 2024-08-22
• Primary Completion: 2025-02
• Study Completion: 2027-08

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 71

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
CAM2029 once every 2 weeks	Placebo	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)
Placebo	Placebo	0.5 mL placebo, SC injection, once weekly
CAM2029 once weekly	CAM2029	0.5 mL CAM2029 10 mg, subcutaneous (SC) injection, once weekly
CAM2029 once every 2 weeks	CAM2029	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)

Primary Outcome Measures

Name	Time	Method
Height-adjusted total liver volume (htTLV)	From screening until treatment week 53	Change from baseline to Week 53 in htTLV as determined by MRI volumetry

Secondary Outcome Measures

Name	Time	Method
Patient Global Impression of Severity (PGI-S) score	From screening to weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173	Change from baseline in the PGI-S score
Height-adjusted total kidney volume (htTKV)	From screening until treatment weeks 13, 25, 53, 77, 125 and 173	Change from baseline in htTKV as determined by MRI volumetry
Polycystic Liver Disease Questionnaire (PLD-Q)	From treatment week 1 to weeks 25, 53, 77, 101, 125, 149 and 173	Change from baseline in the PLD-Q score
PLD-S	From screening to weeks 13, 21, 25, 39, 77, 101, 125, 149 and 173	Change from baseline in the PLD-S measure score
Total liver cyst volume	From screening to treatment weeks 13, 25, 53, 77, 125 and 173	Change from baseline in total liver cyst volume determined by MRI volumetry
PLD impact (PLD-I) score	From screening to weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173	Change from baseline in the PLD-I measure score
Patient Global Impression of Change (PGI-C) score	At treatment weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173	Change from baseline in the PGI-C score
htTLV	From screening until treatment weeks 13, 25, 77, 125 and 173	Change from baseline in htTLV as determined by MRI volumetry
PLD symptom (PLD-S) score	From screening to week 53	Key secondary endpoint. Change from baseline to Week 53 in the PLD-S measure score
Estimated glomerular filtration rate (eGFR)	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
Short Form-36 (SF-36) score	From treatment week 1 to weeks 25, 53, 77, 101, 125, 149 and 173	Change from baseline in the SF-36 score
Clinical Global Impression of Severity (CGI-S) score	From treatment week 1 to weeks 13, 21, 25, 53, 77, 101, 125, 149 and 173	Change from baseline in the CGI-S score
Adverse events (AEs)	From screening to the safety follow-up, assessed up to approximately 43 months	Incidence of AEs

Trial Locations

Locations (11)

Hospital of the University of Pennsylvania; 🇺🇸 Philadelphia, Pennsylvania, United States

University of Texas Southwestern Medical Center; 🇺🇸 Dallas, Texas, United States

The New York Presbyterian Hospital; 🇺🇸 New York, New York, United States

Universitätsklinikum Leipzig; 🇩🇪 Leipzig, Germany

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States

Mount Sinai Hospital; 🇺🇸 New York, New York, United States

Bon Secours Richmond Community Hospital; 🇺🇸 Richmond, Virginia, United States

Hannover Medical School; 🇩🇪 Hannover, Germany

Universitaetsklinikum Müenster; 🇩🇪 Münster, Germany

University Hospitals KU Leuven; 🇧🇪 Leuven, Belgium

Radboud UMC, Department of Gastroenterology and Hepatology; 🇳🇱 Nijmegen, Netherlands