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

NCT05281328 | Active Not Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: HS-20-6772021-003764-27
• Study Type: interventional
• Target: 71
• Locations: 4 countries
• Sites: 11

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.

Conditions

Polycystic Liver Disease

Keywords

Polycystic liver; PLD; ADPLD; ADPKD; PCLD; height-adjusted total liver volume; htTLV; CAM2029; POSITANO

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (13)

• PLD symptom (PLD-S) score

  From screening to week 53

• htTLV

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

• PLD-S

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

• Height-adjusted total kidney volume (htTKV)

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

• Total liver cyst volume

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

Study Arms (3)

CAM2029 once weekly

EXPERIMENTAL

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

Drug: CAM2029

CAM2029 once every 2 weeks

EXPERIMENTAL

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)

Drug: CAM2029; Drug: Placebo

Placebo

PLACEBO COMPARATOR

0.5 mL placebo, SC injection, once weekly

Drug: Placebo

Interventions

Placebo DRUG

SC injection using a pre-filled pen

CAM2029 once every 2 weeks; Placebo

CAM2029 DRUG

SC injection using a pre-filled pen

Also known as: octreotide subcutaneous depot

CAM2029 once every 2 weeks; CAM2029 once weekly

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Camurus AB: lead

Study Sites (11)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Mount Sinai Hospital

New York, New York, 10029, United States

Location

The New York Presbyterian Hospital

New York, New York, 10065, United States

Location

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

University of Texas Southwestern Medical Center

Dallas, Texas, 75380, United States

Location

Bon Secours Richmond Community Hospital

Richmond, Virginia, 23602, United States

Location

University Hospitals KU Leuven

Leuven, B-3000, Belgium

Location

Hannover Medical School

Hanover, 30625, Germany

Location

Universitätsklinikum Leipzig

Leipzig, 04103, Germany

Location

Universitaetsklinikum Müenster

Münster, 48149, Germany

Location

Radboud UMC, Department of Gastroenterology and Hepatology

Nijmegen, 6525 GA, Netherlands

Location

Related Publications (1)

• St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3.

  PMID: 39356039 DERIVED

MeSH Terms

Conditions

Polycystic liver disease; Polycystic Kidney, Autosomal Dominant

Condition Hierarchy (Ancestors)

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

Study Officials

• Joost Drenth, MD

  Department of Gastroenterology and Hepatology, Radboud UMC Nijmegen, The Netherlands

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 7, 2022
• First Posted: March 16, 2022
• Study Start: June 28, 2022
• Primary Completion: February 5, 2025
• Study Completion (Estimated): August 1, 2027
• Last Updated: December 11, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

NL (1); DE (3); US (6); BE (1)