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Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

NCT05876312 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 50

Last updated 2026-04-22

No results posted yet for this study

Summary

The first-in-human Phase 1/Phase 2a study described herein will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-038 in both healthy participants (HP) and in patients with paroxysmal nocturnal hemoglobinuria (PNH).

Conditions

  • Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions

DRUG

ADX-038

siRNA duplex oligonucleotide

DRUG

Placebo

Saline

Sponsors & Collaborators

  • ADARx Australia Pty Ltd

    collaborator UNKNOWN

  • Novotech (Australia) Pty Limited

    collaborator INDUSTRY

  • ADARx Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2023-08-07
Primary Completion
2028-03-31
Completion
2028-07-31

Countries

  • Australia
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05876312 on ClinicalTrials.gov