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RGLS8429

Phase 1

Autosomal Dominant Polycystic Kidney Disease | Small molecule | Nephrology |Regulus Therapeutics Inc.|Last Updated: Aug 27, 2025

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLED
Total Trials1
Total Enrollment68

FDA Designations

No designations recorded

Clinical trial landscape

RGLS8429 · 2 trials · 4 indications

Phase 1 2
NCT05521191A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney DiseaseAutosomal Dominant Polycystic Kidney Disease
COMPLETED68 Analytics
NCT05429073A Study of RGLS8429 in Healthy VolunteersHealthy Volunteers
COMPLETED32 Analytics
PHASE1COMPLETED
A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney Disease
Autosomal Dominant Polycystic Kidney DiseaseUnlock trial analytics
PHASE1COMPLETED
A Study of RGLS8429 in Healthy Volunteers
Healthy VolunteersUnlock trial analytics

Study Endpoints

Primary Endpoints

Safety and tolerability of RGLS4829: Incidence of AEs
Baseline to Day 113

Incidence of adverse events over time

Impact of RGLS8429 on ADPKD biomarkers
Baseline to Day 113

Change from baseline in PC1, PC2, NGAL, and KIM-1 urine biomarkers

Incidence of Adverse Events (AEs)
Baseline to Day 29

Incidence and severity of adverse events after administration of RGLS8429

Secondary Endpoints

Impact of RGLS8429 on height-adjusted total kidney volume (htTKV)
Baseline to Day 113
Pharmacokinetic properties of RGLS8429: Cmax
Baseline to Day 113
Pharmacokinetic properties of RGLS8429: Tmax
Baseline to Day 113
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
RGLS8429EXPERIMENTALThe 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
PlaceboEXPERIMENTALThe 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
Open Label Fixed Dose RGLS8429EXPERIMENTALThe 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.
RGLS8429, first dose levelEXPERIMENTALEligible participants will receive subcutaneous injection of the first dose level RGLS8429 or placebo
RGLS8429, second dose levelEXPERIMENTALEligible participants will receive subcutaneous injection of the second dose level RGLS8429 or placebo
RGLS8429, third dose levelEXPERIMENTALEligible participants will receive subcutaneous injection of the third dose level RGLS8429 or placebo
RGLS8429, fourth dose levelEXPERIMENTALEligible participants will receive subcutaneous injection of the fourth dose level RGLS8429 or placebo

Interventions

NameTypeDescription
RGLS8429DRUGSolution for subcutaneous injection
PlaceboDRUGSolution for subcutaneous injection
Placebo volume-matching RGLS8429 doseDRUGsolution for subcutaneous injection
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Eligibility Criteria

Age Range18 Years to 70 Years
SexALL
Healthy VolunteersNo
Study Sites21

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 ...

Countries:United States
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Frequently asked questions about RGLS8429

What is RGLS8429 used for?

RGLS8429 is an investigational small molecule being studied for Autosomal Dominant Polycystic Kidney Disease (ADPKD), a genetic disorder characterized by cyst growth in the kidneys. It has also been evaluated in healthy volunteers to assess its safety and tolerability. The drug is in Phase 1 clinical development and is not yet approved.

Who makes RGLS8429?

RGLS8429 is being developed by Regulus Therapeutics Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol RGLS. The company is conducting clinical trials of RGLS8429 in the United States for Autosomal Dominant Polycystic Kidney Disease and in healthy volunteers.

What phase is RGLS8429 in?

RGLS8429 is in Phase 1 clinical development. Two Phase 1 trials have been completed: one in healthy volunteers and one in patients with Autosomal Dominant Polycystic Kidney Disease. The drug is investigational and has not been approved by regulatory authorities.

What clinical trials is RGLS8429 in?

RGLS8429 has been studied in two completed Phase 1 trials. NCT05429073 evaluated the drug in 32 healthy volunteers in the United States. NCT05521191 evaluated the drug in 68 patients with Autosomal Dominant Polycystic Kidney Disease, also in the United States. Both trials were randomized, double-blind, and controlled.

Is RGLS8429 the same as other names?

RGLS8429 is the primary name for this investigational drug. No alternative names have been reported for this compound. It is a small molecule being developed by Regulus Therapeutics Inc. for the treatment of Autosomal Dominant Polycystic Kidney Disease.