NCT05521191

A Study of RGLS8429 in Patients With 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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 68 (actual)
• Sponsor: Regulus Therapeutics Inc. · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

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

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.

Conditions

• Autosomal Dominant Polycystic Kidney Disease
• ADPKD
• Polycystic Kidney, Autosomal Dominant

Interventions

Type	Name	Description
DRUG	RGLS8429	Solution for subcutaneous injection
DRUG	Placebo	Solution for subcutaneous injection

Timeline

• Start date: 2022-10-06
• Primary completion: 2025-01-27
• Completion: 2025-03-27
• First posted: 2022-08-30
• Last updated: 2025-06-03

Locations

21 sites across 1 country: United States

Regulatory

• FDA-regulated drug study