Polycystic Liver DiseasePhase 2/3POSITANONCT05281328
A Trial to Assess the Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With PLD
A Phase 2/3 study testing CAM2029 in people with Polycystic Liver Disease, run by Camurus AB at 11 sites in 4 countries.
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Polycystic Liver Disease
- What is being tested?
- CAM2029
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 71 participants
- Where
- 11 sitesin 4 countries
- Study dates
- 28 Jun 2022to Aug 2027
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
11 sites in 4 countries.
11 sites in 4 countries
United States6 sites
University of Texas Southwestern Medical Center
Dallas, Texas, 75380-8887
Mount Sinai Hospital
New York, New York, 10029
The New York Presbyterian Hospital
New York, New York, 10065
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Bon Secours Richmond Community Hospital
Richmond, Virginia, 23602
Mayo Clinic
Rochester, Minnesota, 55905
Germany3 sites
Hannover Medical School
Hanover, 30625
Universitätsklinikum Leipzig
Leipzig, 04103
Universitaetsklinikum Müenster
Münster, 48149
Netherlands1 site
Radboud UMC, Department of Gastroenterology and Hepatology
Nijmegen, 6525 GA
About this study
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
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
- Being tested
- CAM2029, Placebo
- Keywords
- Polycystic liver, PLD, ADPLD, ADPKD, PCLD, height-adjusted total liver volume, htTLV, CAM2029, POSITANO
- Medical subject headings
- Polycystic liver disease, Polycystic Kidney, Autosomal Dominant
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CAM2029Drug
Also called octreotide subcutaneous depot
SC injection using a pre-filled pen
PlaceboDrug
SC injection using a pre-filled pen
| Study group | Role | What they receive |
|---|---|---|
| CAM2029 once weekly | Experimental | 0.5 mL CAM2029 10 mg, subcutaneous (SC) injection, once weekly Receives: 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) Receives: Drug: CAM2029; Drug: Placebo |
| Placebo | Placebo comparator | 0.5 mL placebo, SC injection, once weekly Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Height-adjusted Total Liver Volume (htTLV)
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.
Measured: From screening until treatment week 53
Secondary outcomes (13)
PLD Symptom (PLD-S) Score
Change from baseline to Week 53 in the PLD-S measure score.
Measured: From screening to Week 53
htTLV
Change from baseline in htTLV as determined by MRI volumetry
Measured: From screening until treatment weeks 13, 25, 77, 125 and 173
PLD-S
Change from baseline in the PLD-S measure score
Measured: From screening to weeks 13, 21, 25, 39, 77, 101, 125, 149 and 173
Height-adjusted Total Kidney Volume (htTKV)
Change from baseline in htTKV as determined by MRI volumetry
Measured: From screening until treatment weeks 13, 25, 53, 77, 125 and 173
Total Liver Cyst Volume
Change from baseline in total liver cyst volume determined by MRI volumetry
Measured: From screening to treatment weeks 13, 25, 53, 77, 125 and 173
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
Measured: From treatment week 1 to weeks 13, 25, 53, 65, 77, 101, 125, 149 and 173
PLD Impact (PLD-I) Score
Change from baseline in the PLD-I measure score
Measured: From screening to weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173
Clinical Global Impression of Severity (CGI-S) Score
Change from baseline in the CGI-S score
Measured: From treatment week 1 to weeks 13, 21, 25, 53, 77, 101, 125, 149 and 173
Patient Global Impression of Severity (PGI-S) Score
Change from baseline in the PGI-S score
Measured: From screening to weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173
Patient Global Impression of Change (PGI-C) Score
Change from baseline in the PGI-C score
Measured: At treatment weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173
Short Form-36 (SF-36) Score
Change from baseline in the SF-36 score
Measured: From treatment week 1 to weeks 25, 53, 77, 101, 125, 149 and 173
Polycystic Liver Disease Questionnaire (PLD-Q)
Change from baseline in the PLD-Q score
Measured: From treatment week 1 to weeks 25, 53, 77, 101, 125, 149 and 173
Adverse Events (AEs)
Incidence of AEs
Measured: From screening to the safety follow-up, assessed up to approximately 47 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2022
- Study start28 Jun 2022
- Primary completion5 Feb 2025
- Study completionAug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Polycystic Liver DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 71 participants (actual)
Sponsor and investigators
- Lead sponsor
- Camurus AB
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Joost Drenth, MD, Principal investigator, Department of Gastroenterology and Hepatology, Radboud UMC Nijmegen, The Netherlands
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05281328
- Sponsor's protocol ID
- HS-20-677
- Other IDs
- 2021-003764-27
- Acronym
- POSITANO
- Status last verified
- Mar 2026
- First submitted
- 7 Mar 2022
- Last updated
- 1 Jun 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 39356039
Study at a glance
From the registry record, last updated by the sponsor on 1 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05281328
- Acronym / sponsor ID
- POSITANO / HS-20-677
- Conditions
- Polycystic Liver Disease
- Keywords
- Polycystic liver, PLD, ADPLD, ADPKD, PCLD, height-adjusted total liver volume, htTLV, CAM2029
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 71 participants (actual)
- Lead sponsor
- Camurus ABIndustry (pharma, biotech, devices)
- Study start
- 28 Jun 2022 (actual)
- Primary completion
- 5 Feb 2025 (actual)
- Study completion
- Aug 2027 (estimated)
- First posted
- 16 Mar 2022 (actual)
- Results
- Posted 1 Jun 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| CAM2029 once weekly | Experimental | Drug: CAM20290.5 mL CAM2029 10 mg, subcutaneous (SC) injection, once weekly |
| CAM2029 once every 2 weeks | Experimental | Drug: CAM2029, Drug: Placebo0.5 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 comparator | Drug: Placebo0.5 mL placebo, SC injection, once weekly |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CAM2029Also: octreotide subcutaneous depotSC injection using a pre-filled pen | Drug | CAM2029 once every 2 weeks, CAM2029 once weekly |
| PlaceboSC injection using a pre-filled pen | Drug | CAM2029 once every 2 weeks, Placebo |
Primary outcome
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.
13 secondary outcomes
PLD Symptom (PLD-S) Score
Time frame: From screening to Week 53
htTLV
Time frame: From screening until treatment weeks 13, 25, 77, 125 and 173
PLD-S
Time frame: From screening to weeks 13, 21, 25, 39, 77, 101, 125, 149 and 173
Height-adjusted Total Kidney Volume (htTKV)
Time frame: From screening until treatment weeks 13, 25, 53, 77, 125 and 173
Total Liver Cyst Volume
Time frame: From screening to treatment weeks 13, 25, 53, 77, 125 and 173
Estimated Glomerular Filtration Rate (eGFR)
Time frame: From treatment week 1 to weeks 13, 25, 53, 65, 77, 101, 125, 149 and 173
PLD Impact (PLD-I) Score
Time frame: From screening to weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173
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
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
Patient Global Impression of Change (PGI-C) Score
Time frame: At treatment weeks 13, 21, 25, 39, 53, 77, 101, 125, 149 and 173
Short Form-36 (SF-36) Score
Time frame: From treatment week 1 to weeks 25, 53, 77, 101, 125, 149 and 173
Polycystic Liver Disease Questionnaire (PLD-Q)
Time frame: From treatment week 1 to weeks 25, 53, 77, 101, 125, 149 and 173
Adverse Events (AEs)
Time frame: From screening to the safety follow-up, assessed up to approximately 47 months.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jun 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39356039St 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Joost Drenth, MD principal investigator
Department of Gastroenterology and Hepatology, Radboud UMC Nijmegen, The Netherlands
11 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing CAM2029 in people with Polycystic Liver Disease, run by Camurus AB at 11 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 1 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, Germany, Belgium, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 Jun 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Height-adjusted Total Liver Volume (htTLV) (time frame: From screening until treatment week 53). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05281328, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jun 2026.