NCT05984992

The First-in-human Study of SRN-001 in Healthy Participants

A Randomized, Double-blinded, Placebo-controlled, Single Ascending Dose Study to Assess the Safety, Tolerability and Pharmacokinetics of SRN-001 in Healthy Participants

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 25 (actual)
• Sponsor: siRNAgen Therapeutics Inc. · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Accepted

Summary

SRN-001 is a novel small interfering RNA (siRNA) drug being developed to treat fibrosis using Self Assembled Micelle inhibitory ribonucleic acid (SAMiRNA™) technology. Amphiregulin (AREG) is a growth factor involved in fibroblast proliferation and myofibroblast transformation which is the hallmark of fibrosis in lung and kidney tissues. AREG is a downstream gene overexpressed by Transforming growth factor-β (TGF-β) during fibrosis, promoting fibroblast to myofibroblast transition (FMT). SRN-001 is designed to downregulate generating amphiregulin by RNA interference (RNAi). The goal of this clinical trial is to evaluate safety, tolerability, and pharmacokinetics in healthy participants. This trial is first-in-human clinical trial to develop SAMiRNA™ to utilize as therapeutic use.

Detailed description

Participants with part in consent will be enrolled in a phase 1a study of SRN-001. Prior to initiation of treatment, participants will undergo several screening test for checking their condition of health. There is no specific test comparing with the general other clinical trial in healthy volunteers. They will be randomized into two groups, active drug and inactive placebo(normal saline) as ratio 2:1. Starting dose is planned 15mg. For confirming maximal tolerable dose, dose will be escalated when no dose-limiting toxicity (DLT) confirmed. Each cohort will take single dose and for 4 weeks, safety observation will be taken. If safety abnormality will be retained in 4 weeks, the participant's safety observation will be prolonged by the end of the adverse event once 2 weeks.

Conditions

• Idiopathic Pulmonary Fibrosis

Interventions

Type	Name	Description
DRUG	SRN-001	siRNA therapeutics, Self Assembled Micelle inhibitory RNA platform utilized
DRUG	Placebo	0.9% Sodium Chloride(Normal saline)

Timeline

• Start date: 2023-09-08
• Primary completion: 2024-03-15
• Completion: 2024-09-25
• First posted: 2023-08-09
• Last updated: 2024-11-04

Locations

1 site across 1 country: Australia