NCT05691361

Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients

A Phase 1, Randomized, Placebo-Controlled, Double-Blind Study in Healthy Volunteers and a Phase 2a, Open-Label Study in Patients With Hereditary Angioedema to Evaluate the Safety, Tolerability, PK and PD of ADX-324

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 53 (estimated)
• Sponsor: ADARx Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Accepted

Summary

The first-in-human Phase 1 study will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-324 in healthy volunteers (HV) and in patients with Hereditary Angioedema (HAE).

Detailed description

The clinical study described in this protocol is a Phase 1, single-center study evaluating safety, tolerability, PK, and PD of ADX-324. The study consists of 2 parts: * Randomized, double-blind, placebo-controlled, parallel group, single ascending dose (SAD) in HV with up to 6 dose cohorts. For SAD cohorts and planned dosing; and, * Expansion cohort in participants with Hereditary Angioedema (HAE) at selected dose from Part A and will be open label.

Conditions

• Hereditary Angioedema

Interventions

Type	Name	Description
DRUG	ADX-324	siRNA duplex oligonucleotide
DRUG	Placebo	saline

Timeline

• Start date: 2022-12-14
• Primary completion: 2027-12-30
• Completion: 2027-12-30
• First posted: 2023-01-20
• Last updated: 2026-03-02

Locations

1 site across 1 country: Australia