A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney Disease

May 28, 2025 updated by: Regulus Therapeutics Inc.

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

Primary Objectives

  • To assess the safety and tolerability of RGLS8429
  • To assess the impact of RGLS8429 on ADPKD biomarkers

Secondary Objectives

  • To assess the impact of RGLS8429 on height-adjusted total kidney volume (htTKV)
  • To characterize the pharmacokinetic (PK) properties of RGLS8429
  • To assess the impact of RGLS8429 on renal function

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

This is a randomized, double-blind, placebo-controlled multiple ascending dose and an open-label fixed-dose Phase 1b study consisting of two parts, Part A and Part B. In Part A, multiple ascending doses of RGLS8429 or placebo will be administered via subcutaneous injection to subjects with ADPKD to evaluate the safety, tolerability, pharmacodynamics and pharmacokinetics of RGLS8429. In Part B, a fixed-dose of RGLS8429 will be administered via subcutaneous injection to subjects with ADPKD to evaluate the safety, tolerability, pharmacodynamics and pharmacokinetics of RGLS8429.

Study Type

Interventional

Enrollment (Actual)

68

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Arizona
      • Mesa, Arizona, United States, 85206
        • Centricity Research Phoenix Multispecialty
    • California
      • Los Angeles, California, United States, 90022
        • Academic Medical Research Institute
    • Connecticut
      • New Haven, Connecticut, United States, 06510
        • Yale Nephrology Outpatient Clinic
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Mayo Clinic - Florida
      • Orlando, Florida, United States, 32806
        • ELIXIA
    • Georgia
      • Augusta, Georgia, United States, 30904
        • Southeastern Clinical Research Institute, LLC
    • Idaho
      • Boise, Idaho, United States, 83706
        • CARE Institute
      • Chubbuck, Idaho, United States, 83202
        • CARE Institute
      • Idaho Falls, Idaho, United States, 83404
        • The Idaho Kidney Institute
    • Illinois
      • Chicago, Illinois, United States, 60643
        • Research by Design, LLC
      • Chicago, Illinois, United States, 60617
        • University of Chicago
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center Jared Grantham Kidney Institute
      • Wichita, Kansas, United States, 67214
        • Witchita Nephrology Group, PA
    • Maryland
      • Baltimore, Maryland, United States, 21201
        • University of Maryland School of Medicine, Nephrology
    • Massachusetts
      • Boston, Massachusetts, United States, 02111
        • Tufts Medical Center
    • Michigan
      • Roseville, Michigan, United States, 48066
        • St. Clair Nephrology Research
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic - Rochester
    • Pennsylvania
      • Bethlehem, Pennsylvania, United States, 18017
        • Northeast Clinical Research Center
    • Texas
      • Dallas, Texas, United States, 75390
        • University of Texas Southwestern Medical Center
    • Washington
      • Seattle, Washington, United States, 98104
        • Swedish Center for Comprehensive Care

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 70 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: RGLS8429

The randomized,double blind part of the study (Part A) will consist of three sequential cohorts of approximately 12 subjects each randomized centrally to receive RGLS8429 or placebo by subcutaneous injection every other week (Q2W) x 7 doses (36 subjects total).

  • Cohort 1: first dose level of RGLS8429 or placebo
  • Cohort 2: second dose level of RGLS8429 or placebo
  • Cohort 3: third dose level of RGLS8429 or placebo
Drug: RGLS8429
Solution for subcutaneous injection
Experimental: Placebo

The randomized double blind part of the study (Part A) will consist of three sequential cohorts of approximately 12 subjects each randomized centrally to receive RGLS8429 or placebo by subcutaneous injection every other week (Q2W) x 7 doses (36 subjects total).

  • Cohort 1: first dose level of RGLS8429 or placebo
  • Cohort 2: second dose level of RGLS8429 or placebo
  • Cohort 3: third dose level of RGLS8429 or placebo
Drug: Placebo
Solution for subcutaneous injection
Experimental: Open Label Fixed Dose RGLS8429
The open-label fixed dose part of the study (Part B and Cohort 4) will consist of a single cohort of up to 30 subjects each receiving 300 mg RGLS8429.
Drug: RGLS8429
Solution for subcutaneous injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety and tolerability of RGLS4829: Incidence of AEs
Time Frame: Baseline to Day 113
Incidence of adverse events over time
Baseline to Day 113
Impact of RGLS8429 on ADPKD biomarkers
Time Frame: Baseline to Day 113
Change from baseline in PC1, PC2, NGAL, and KIM-1 urine biomarkers
Baseline to Day 113

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Impact of RGLS8429 on height-adjusted total kidney volume (htTKV)
Time Frame: Baseline to Day 113
Change from baseline in htTKV
Baseline to Day 113
Pharmacokinetic properties of RGLS8429: Cmax
Time Frame: Baseline to Day 113
Maximum observed concentration (Cmax) of RGLS8429
Baseline to Day 113
Pharmacokinetic properties of RGLS8429: Tmax
Time Frame: Baseline to Day 113
Time to maximum observed concentration (Tmax) of RGLS8429
Baseline to Day 113
Pharmacokinetic properties of RGLS8429: t½
Time Frame: Baseline to Day 113
Half-life of RGLS8429 (t½)
Baseline to Day 113

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Regulus Therapeutics Inc.

Investigators

  • Study Director: Rekha Garg, MD, Regulus Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 6, 2022

Primary Completion (Actual)

January 27, 2025

Study Completion (Actual)

March 27, 2025

Study Registration Dates

First Submitted

August 24, 2022

First Submitted That Met QC Criteria

August 25, 2022

First Posted (Actual)

August 30, 2022

Study Record Updates

Last Update Posted (Actual)

June 3, 2025

Last Update Submitted That Met QC Criteria

May 28, 2025

Last Verified

May 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RGLS8429-02

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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