A clinical study to learn more about the safety and effects of Farabursen in people with autosomal dominant polycystic kidney disease
Summary description of the study
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.
(BASEC)
Intervention under investigation
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.
(BASEC)
Disease under investigation
Autosomal dominant polycystic kidney disease (ADPKD)
(BASEC)
Participants must:
1. Be adults (18 years or older) with a confirmed diagnosis of ADPKD
2. Have a risk for disease progression based on imaging (MRI) and kidney function
3. Meet certain criteria regarding kidney function (eGFR) and disease classification.
(BASEC)
Exclusion criteria
Participants cannot participate in the study if they:
1. Have received approved treatments for ADPKD (e.g., Tolvaptan) within three months prior to screening.
2. Have previously received Farabursen or participated in another Farabursen study.
3. Have other serious kidney diseases or severe medical conditions that could affect the study outcomes.
(BASEC)
Trial sites
Basel, Bern, Lausanne, Zurich
(BASEC)
Sponsor
Novartis Pharma Schweiz AG
(BASEC)
Contact
Contact Person Switzerland
Medizinische Auskünfte
Tel: +41 800 633 463
swiss.medinfo@clutternovartis.comNovartis Pharma Schweiz AG Medical Information
(BASEC)
Scientific Information
not available
Name of the authorising ethics committee (for multicentre studies, only the lead committee)
Ethics Committee northwest/central Switzerland EKNZ
(BASEC)
Date of authorisation
06.10.2026
(BASEC)
ICTRP Trial ID
not available
Official title (approved by ethics committee)
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study in Patients with Autosomal Dominant Polycystic Kidney Disease to Evaluate the Efficacy and Safety of Farabursen (BASEC)
Academic title
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Public title
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Disease under investigation
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Intervention under investigation
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Type of trial
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Trial design
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Inclusion/Exclusion criteria
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Primary and secondary end points
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Registration date
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Incorporation of the first participant
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Secondary sponsors
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Additional contacts
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Secondary trial IDs
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Results-Individual Participant Data (IPD)
not available
Further information on the trial
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Results of the trial
Results summary
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Link to the results in the primary register
not available