CAM2029 for Polycystic Liver Disease

(POSITANO Trial)

Not currently recruiting at 12 trial locations

Overseen By Camurus AB

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Camurus AB

Must not be taking: Somatostatin analogues

Disqualifiers: Severe kidney disease, severe liver disease, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 53 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called CAM2029 for individuals with polycystic liver disease (PLD). The researchers aim to compare the effectiveness and safety of two different dosing schedules of CAM2029 against a placebo. CAM2029 is administered as an injection under the skin, either weekly or every two weeks. Individuals experiencing symptoms like abdominal pain, bloating, or feeling full quickly due to PLD, and who are not opting for surgery, might be suitable for this trial. Participants will initially engage in a 53-week treatment period, with the option to continue for an extended 120-week period. As a Phase 2, Phase 3 trial, this research measures the treatment's effectiveness in an initial, smaller group and represents the final step before FDA approval, offering participants a chance to contribute to advancing treatment options.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot have been treated with a somatostatin analogue (a type of medication) within 3 months before the trial starts.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that CAM2029, which contains the active ingredient octreotide, has been generally well-tolerated in studies for polycystic liver disease (PLD). In the POSITANO study, patients who took CAM2029 experienced significant reductions in the size of their liver and liver cysts compared to those who took a placebo. Importantly, the study found improvements in symptoms without major safety concerns.

Octreotide, the main component of CAM2029, is already used to treat other conditions, so its safety is well-known. While any treatment can have side effects, evidence so far supports that CAM2029 is safe for people. Participants in this trial will undergo regular check-ups to ensure their safety throughout the study. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard treatments for polycystic liver disease, which often include supportive care or liver transplantation, CAM2029 offers a novel approach. Researchers are excited about CAM2029 because it is a somatostatin analog, which can help reduce liver cyst growth by inhibiting specific hormones. This treatment is delivered as a convenient subcutaneous injection, either once weekly or every two weeks, making it potentially more patient-friendly compared to existing options. Additionally, this targeted mechanism could lead to improved outcomes with fewer side effects, offering hope for a better quality of life for patients with this condition.

What evidence suggests that CAM2029 might be an effective treatment for polycystic liver disease?

Research has shown that CAM2029 may help treat polycystic liver disease. Studies found that CAM2029 can noticeably reduce the size of the liver and its cysts, with a 4.3% decrease in liver size and an 8.7% decrease in cyst size compared to a placebo. This trial will evaluate CAM2029 administered once weekly or once every two weeks. The treatment uses a form of octreotide, delivered through a special method, to better manage the disease. These findings suggest CAM2029 could be a useful option for those with polycystic liver disease. ^{2 3 4 6 7}

Who Is on the Research Team?

Joost Drenth, MD

Principal Investigator

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

Are You a Good Fit for This Trial?

This trial is for adults over 18 with Polycystic Liver Disease (PLD) who have a liver volume of at least 2500 mL/m and experience symptoms like bloating, abdominal pain, or shortness of breath. Candidates should not be planning surgery for PLD during the trial and must not have severe kidney or liver disease, recent PLD surgery, or non-responsiveness to similar treatments.

Inclusion Criteria

I have experienced symptoms like bloating or abdominal pain recently.

I am not planning to have surgery for liver cysts during the trial.

I have been diagnosed with PLD, with a specific volume measurement.

Exclusion Criteria

I have cysts outside my liver that may make it unsafe for me to join the trial.

I have been treated with a somatostatin analogue within the last 3 months.

I have severe liver disease (Child-Pugh class C).

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive CAM2029 or placebo in a randomized, double-blind manner for 53 weeks

53 weeks

Weekly or bi-weekly visits for subcutaneous injections

Open-label extension

Participants may continue receiving CAM2029 treatment for an additional 120 weeks

120 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CAM2029

Trial Overview The study tests CAM2029's effectiveness and safety in treating symptomatic PLD. Participants will randomly receive either CAM2029 weekly or biweekly, or a placebo for 53 weeks. Those completing this phase may enter a 24-week extension receiving only CAM2029 using FluidCrystal technology.

How Is the Trial Designed?

Group I: CAM2029 once weekly Experimental Treatment 1 Intervention

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

Group II: CAM2029 once every 2 weeks Experimental Treatment 2 Interventions

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)

Group III: Placebo Placebo Group 1 Intervention

0.5 mL placebo, SC injection, once weekly

Who Is Running the Clinical Trial?

Camurus AB

Lead Sponsor

Trials

9

Recruited

1,900+

Citations

1. camurus.com camurus.com/media/press-releases/2025/camurus-positano-study-shows-treatment-effects-with-cam2029-in-polycystic-liver-disease-patients/

Camurus' POSITANO study shows treatment effects with ... The POSITANO study shows that CAM2029 significantly reduces liver and liver cyst volume compared to placebo while showing improvements in symptoms.

2. prnewswire.com prnewswire.com/news-releases/camurus-positano-study-shows-treatment-effects-with-cam2029-in-polycystic-liver-disease-patients-302484906.html

Camurus' POSITANO study shows treatment effects with ... The POSITANO study shows that CAM2029 significantly reduces liver and liver cyst volume compared to placebo while showing improvements in symptoms.

3. clinicalstudies.pkdcure.org clinicalstudies.pkdcure.org/study/a-randomized-placebo-controlled-double-blind-multi-center-phase-2-3-trial-to-assess-efficacy-and-safety-of-octreotide-subcutaneous-depot-cam2029-in-patients-with-symptomatic-polycystic-liver-dise/

A Randomized, Placebo-controlled, Double-blind, Multi ... The purpose of this trial is to evaluate the effectiveness and safety of 2 treatment regimens of CAM2029 versus placebo in patients with Polycystic Liver ...

4. biostock.se biostock.se/en/2025/06/camurus-cam2029-visar-effekt-vid-polycystisk-leversjukdom/

Camurus CAM2029 shows efficacy in polycystic liver disease The study met its primary endpoint with a 4,3 percent reduction in length-adjusted liver volume and 8,7 percent in cyst volume compared to ...

5. trial.medpath.com trial.medpath.com/news/a80ae497760fa328/camurus-cam2029-shows-significant-liver-volume-reduction-in-phase-2b-trial-for-rare-polycystic-liver-disease

Camurus' CAM2029 Shows Significant Liver Volume ... Camurus' POSITANO Phase 2b study demonstrates that CAM2029 significantly reduces liver volume by 4.3% and liver cyst volume by 8.7% compared ...

6. ctv.veeva.com ctv.veeva.com/study/a-trial-to-assess-the-efficacy-and-safety-of-octreotide-subcutaneous-depot-in-patients-with-pld

A Trial to Assess the Efficacy and Safety of Octreotide ... 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 ...

7. camurus.com camurus.com/media/press-releases/2024/completed-enrollment-in-the-positano-study-of-cam2029-in-patients-with-polycystic-liver-disease/

Completed enrollment in the POSITANO study of CAM2029 ... The primary endpoint is the change from baseline to week 53 in height-adjusted liver volume and the first secondary endpoint is the change in ...