A Study of RGLS8429 in Healthy Volunteers

NCT05429073 | Completed Phase 1 Results FDA Drug

• 1 other identifier: RGLS8429-01
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Healthy Volunteers

Keywords

RGLS8429; Autosomal Dominant Polycystic Kidney Disease; ADPKD

Outcome Measures

Primary Outcomes (1)

• Incidence of Adverse Events (AEs)

  Incidence and severity of adverse events after administration of RGLS8429

  Baseline to Day 29

Secondary Outcomes (5)

• Incidence of DLT and Determination of MTD

  Baseline to Day 29

• Plasma Half-life (T1/2) of RGLS8429

  Baseline to Day 15

• Plasma Clearance of RGLS8429

  Baseline to Day 15

• Area Under the Plasma Concentration vs Time Curve (AUC) of RGLS8429

  Baseline to Day 15

• Amount of RGLS8429 Excreted in Urine

  Baseline to 24-hour

Study Arms (4)

RGLS8429, first dose level

EXPERIMENTAL

Eligible participants will receive subcutaneous injection of the first dose level RGLS8429 or placebo

Drug: RGLS8429; Drug: Placebo volume-matching RGLS8429 dose

RGLS8429, second dose level

EXPERIMENTAL

Eligible participants will receive subcutaneous injection of the second dose level RGLS8429 or placebo

Drug: RGLS8429; Drug: Placebo volume-matching RGLS8429 dose

RGLS8429, third dose level

EXPERIMENTAL

Eligible participants will receive subcutaneous injection of the third dose level RGLS8429 or placebo

Drug: RGLS8429; Drug: Placebo volume-matching RGLS8429 dose

RGLS8429, fourth dose level

EXPERIMENTAL

Eligible participants will receive subcutaneous injection of the fourth dose level RGLS8429 or placebo

Drug: RGLS8429; Drug: Placebo volume-matching RGLS8429 dose

Interventions

RGLS8429 DRUG

solution for subcutaneous injection

RGLS8429, first dose level; RGLS8429, fourth dose level; RGLS8429, second dose level; RGLS8429, third dose level

Placebo volume-matching RGLS8429 dose DRUG

solution for subcutaneous injection

RGLS8429, first dose level; RGLS8429, fourth dose level; RGLS8429, second dose level; RGLS8429, third dose level

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Male or female, 18 to 55 years of age
• Body mass index (BMI) 18 to 35 kg/m2
• Medically healthy, with no clinically significant medical history in the opinion of the Investigator
• Estimated glomerular filtration rate (eGFR) ≥ 90 mL/min/1.73 m2
• 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

You may not qualify if:

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

Sponsors & Collaborators

• Regulus Therapeutics Inc.: lead

Study Sites (1)

ICON Early Phase Services

San Antonio, Texas, 78209, United States

Location

MeSH Terms

Conditions

Polycystic Kidney, Autosomal Dominant

Condition Hierarchy (Ancestors)

Polycystic Kidney Diseases; Kidney Diseases, Cystic; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ciliopathies; Genetic Diseases, Inborn

Results Point of Contact

• Title: Kristen Gillotti, Senior Director Clinical Operations
• Organization: Regulus Therapeutics

kgillotti@regulusrx.com

203-313-8185

Study Officials

• Rekha Garg, MD

  Regulus Therapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: This will be a double-blinded, randomized, placebo-controlled study.
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 26, 2022
• First Posted: June 23, 2022
• Study Start: June 10, 2022
• Primary Completion: September 8, 2022
• Study Completion: September 8, 2022
• Last Updated: August 27, 2025
• Results First Posted: August 27, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)