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New siRNA drug ADX-038 enters human trials for PNH anemia

NCT ID NCT05876312

Recruiting now Disease control Sponsor: ADARx Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a new drug called ADX-038 in healthy volunteers and people with PNH who still have low red blood cells despite treatment. The goal is to check safety and how the drug works in the body. It is an early-stage trial with 50 participants.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
ADX-038 (siRNA drug)
What this could lead to
If successful, this could lead to a new treatment option for people with PNH who still have anemia despite current therapy.
What could go wrong
This is a very early, first-in-human study with only 50 participants. The drug may not work as hoped or could have unexpected side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Hemoglobinuria, Paroxysmal paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PAROXYSMAL NOCTURNAL HEMOGLOBINURIA, PNH

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Nucleus Network Brisbane

    COMPLETED

    Brisbane, Queensland, 4006, Australia

  • Peter MacCallum Cancer Centre

    COMPLETED

    Melbourne, Victoria, 3000, Australia

  • Richmond Pharmacology Ltd

    COMPLETED

    London, London, SE1 1YR, United Kingdom

  • Royal Melbourne Hospital

    RECRUITING

    Parkville, Victoria, 3052, Australia

More trials for these conditions

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