This research study is for men and women who have been diagnosed with polycystic liver disease (PLD), a rare genetic disorder of the liver. This study will evaluate a experimental/investigational drug named CAM2029, which may reduce liver growth in individuals with PLD. CAM 2029 is considered investigational meaning it has not yet been approved by the U.S. Food and Drug Administration (FDA).
CAM2029 contains an active ingredient called octreotide. Octreotide is already approved by the FDA for treating other diseases but is not approved to treat PLD.
CAM2029 injection is new in the way octreotide is given. The technology is called FluidCrystal® and has been approved by the FDA for use since 2018. With the FluidCrystal® technology, the study drug will be released in the body over a longer period of time than pills. Because the study drug is in the body for longer, this may reduce the number of times the study drug is given.
Participants will be randomized into one of three study groups. Randomization is like flipping a coin and there is an equal chance of being assigned to each group.
Group 1 will receive a CAM2029 injection once a week, Group 2 will receive a CAM2029 injection once every 2 weeks with placebo given on the other weeks in-between, and Group 3 will receive a placebo injection once a week.
The placebo injection will look like the study drug CAM2029 but will not contain active medicine/ingredients.
Study participation will last approximately 20 months and participants will be compensated for their time.
Detailed eligibility reviewed when you contact the study team.