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Evaluating RGLS8429 for treating Autosomal Dominant Polycystic Kidney Disease

A Phase 1b, Double-Blind, Placebo-Controlled, Multiple Ascending Dose and an Open-Label Fixed-Dose Study in Patients With Autosomal Dominant Polycystic Kidney Disease to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of RGLS8429

Phase 1 Interventional Regulus Therapeutics Inc. · NCT05521191

This study is testing a new drug called RGLS8429 to see if it is safe and helps people with Autosomal Dominant Polycystic Kidney Disease.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment70 (estimated)
Ages18 Years to 70 Years
SexAll
SponsorRegulus Therapeutics Inc. Industry-sponsored
Locations28 sites (Mesa, Arizona and 27 other locations)
Trial IDNCT05521191 on ClinicalTrials.gov

What this trial studies

This clinical trial assesses the safety and tolerability of RGLS8429 in patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD). It consists of two parts: Part A involves administering multiple ascending doses of RGLS8429 or a placebo to evaluate its pharmacodynamics and pharmacokinetics, while Part B involves a fixed dose of RGLS8429. The study also aims to measure the impact of the drug on ADPKD biomarkers and renal function, as well as height-adjusted total kidney volume.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 70 with a confirmed diagnosis of ADPKD and specific Mayo Imaging classifications.

Not a fit: Patients who have recently taken tolvaptan or have significant mental or emotional issues may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve kidney function and slow disease progression in patients with ADPKD.

How similar studies have performed: While this approach is novel, similar studies targeting ADPKD have shown promise in the past.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key Inclusion Criteria:

1. Male or female ADPKD patients, 18 to 70 years old
2. Class 1C, 1D, or 1E Mayo Imaging Classification of ADPKD (based upon either the MRI obtained during screening, or a prior MRI obtained within 5 years of screening with documented Mayo classification)
3. eGFR between 30 to 90 mL/min/1.73 m2
4. Body mass index (BMI) 18 to 35 kg/m2
5. Must understand and consent to the study procedures explained in the ICF and be willing and able to comply with the protocol

Key Exclusion Criteria:

1. Administration of tolvaptan in the 28 days before randomization
2. Subject is mentally incapacitated or has significant emotional problems
3. Any medical condition or social circumstance that, in the opinion of the Investigator, may make the subject unlikely to complete the study or comply with study procedures and requirements; or may pose a risk to the subject's safety
4. History or presence of alcoholism or drug abuse within the past 2 years prior to screening
5. Only one kidney or kidney transplant recipient
6. Participation in another clinical trial and/or exposure to any investigational drug or approved therapy for investigational use within 28 days or 5 half-lives of the investigational drug's dosing, whichever is longer, prior to dosing. The 28-day or 5-half-life windows will be calculated from the date of the last dosing in the previous study to Day 1 of the current study.

Where this trial is running

Mesa, Arizona and 27 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Autosomal Dominant Polycystic Kidney DiseaseADPKDPolycystic Kidney, Autosomal Dominant
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.