A Study of RGLS8429 in Healthy Volunteers

A Phase 1, Double-Blind, Placebo-Controlled, Single Ascending Dose Study in Healthy Volunteers to Evaluate the Safety, Tolerability, and Pharmacokinetics of RGLS8429

Primary Objective

• To assess the safety and tolerability of single ascending doses of RGLS8429

Secondary Objectives

• To identify dose-limiting toxicity (DLT) and to determine the maximum tolerated dose (MTD) of a single SC dose of RGLS8429
• To characterize the pharmacokinetic (PK) properties of RGLS8429

Study Overview

• Status: Completed
• Conditions: Healthy Volunteers
• Intervention / Treatment: Drug: RGLS8429; Drug: Placebo volume-matching RGLS8429 dose

Detailed Description

In this randomized, double-blind, placebo-controlled Phase 1 study, a single ascending dose of RGLS8429 or placebo will be administered via subcutaneous (SC) injection to healthy volunteers to evaluate the safety, tolerability, and PK of RGLS8429.

• Study Type: Interventional
• Enrollment (Actual): 32
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Texas
    • San Antonio, Texas, United States, 78209
      • ICON Early Phase Services

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult)
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

1. Male or female, 18 to 55 years of age
2. Body mass index (BMI) 18 to 35 kg/m2
3. Medically healthy, with no clinically significant medical history in the opinion of the Investigator
4. Estimated glomerular filtration rate (eGFR) ≥ 90 mL/min/1.73 m2
5. Must understand and consent to the study procedures explained in the informed consent form (ICF) and be willing and able to comply with the protocol

Exclusion Criteria:

1. Subject is mentally incapacitated or has significant emotional problems
2. 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
3. History or presence of alcoholism or drug abuse within the past 2 years prior to Screening
4. 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 and 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

How is the study designed?

Design Details

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

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: RGLS8429, first dose level; Eligible participants will receive subcutaneous injection of the first dose level RGLS8429 or placebo	Drug: RGLS8429; solution for subcutaneous injection; Drug: Placebo volume-matching RGLS8429 dose; solution for subcutaneous injection
Experimental: RGLS8429, second dose level; Eligible participants will receive subcutaneous injection of the second dose level RGLS8429 or placebo	Drug: RGLS8429; solution for subcutaneous injection; Drug: Placebo volume-matching RGLS8429 dose; solution for subcutaneous injection
Experimental: RGLS8429, third dose level; Eligible participants will receive subcutaneous injection of the third dose level RGLS8429 or placebo	Drug: RGLS8429; solution for subcutaneous injection; Drug: Placebo volume-matching RGLS8429 dose; solution for subcutaneous injection
Experimental: RGLS8429, fourth dose level; Eligible participants will receive subcutaneous injection of the fourth dose level RGLS8429 or placebo	Drug: RGLS8429; solution for subcutaneous injection; Drug: Placebo volume-matching RGLS8429 dose; solution for subcutaneous injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Adverse Events (AEs)	Incidence and severity of adverse events after administration of RGLS8429	Baseline to Day 29

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of DLT and Determination of MTD	Identify dose limiting toxicity and the maximum tolerated dose after RGLS8429 administration	Baseline to Day 29
Plasma Half-life (T1/2) of RGLS8429	Measure plasma elimination half-life after RGLS8429 administration	Baseline to Day 15
Plasma Clearance of RGLS8429	Measure plasma clearance after RGLS8429 administration	Baseline to Day 15
Area Under the Plasma Concentration vs Time Curve (AUC) of RGLS8429	Measure area under the plasma concentration vs time curve after RGLS8429 administration	Baseline to Day 15
Amount of RGLS8429 Excreted in Urine	Measure the amount of RGLS8429 excreted in urine after RGLS8429 administration	Baseline to 24-hour

Collaborators and Investigators

• Sponsor: Regulus Therapeutics Inc.
• Investigators: Study Director: Rekha Garg, MD, Regulus Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): June 10, 2022
• Primary Completion (Actual): September 8, 2022
• Study Completion (Actual): September 8, 2022

Study Registration Dates

• First Submitted: May 26, 2022
• First Submitted That Met QC Criteria: June 17, 2022
• First Posted (Actual): June 23, 2022

Study Record Updates

• Last Update Posted (Estimated): August 27, 2025
• Last Update Submitted That Met QC Criteria: August 25, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Keywords: ADPKD; Autosomal Dominant Polycystic Kidney Disease; RGLS8429
• Additional Relevant MeSH Terms: Ciliopathies; Urogenital Diseases; Male Urogenital Diseases; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Genetic Diseases, Inborn; Congenital Abnormalities; Abnormalities, Multiple; Kidney Diseases, Cystic; Polycystic Kidney Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Polycystic Kidney, Autosomal Dominant
• Other Study ID Numbers: RGLS8429-01

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