A clinical study to learn more about the safety and effects of Farabursen in people with autosomal dominant polycystic kidney disease

This clinical study investigates an experimental drug called Farabursen in adults with autosomal dominant polycystic kidney disease (ADPKD), a genetic condition where cysts form in the kidneys over time. These cysts can lead to kidney enlargement and eventually impair kidney function. The aim of the study is to determine whether Farabursen can slow the progression of the disease by reducing kidney growth and preserving kidney function. Participants will be randomly assigned to receive either Farabursen or a placebo (a treatment without an active ingredient). The study is double-blind, meaning that neither the participants nor the investigators know which treatment each participant is receiving. The treatment will be administered as a subcutaneous injection every two weeks. Participants will regularly attend study visits where health checks, laboratory tests, imaging studies (such as MRI), and questionnaires about their health status and quality of life will be conducted. The study will last approximately 2 years. The results are expected to show whether Farabursen is safe and effective as a potential new treatment for ADPKD.

Participants will receive either:
• Farabursen (experimental investigational drug), or
• Placebo (an inactive comparator treatment)

Farabursen will be administered as a subcutaneous injection (under the skin) every two weeks. Two different dosing levels will be investigated. The study will compare how well Farabursen slows the progression of kidney disease compared to placebo. At the same time, safety and tolerability will also be assessed.

Autosomal dominant polycystic kidney disease (ADPKD)