New siRNA drug ADX-038 enters human trials for PNH anemia
NCT ID NCT05876312
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Nucleus Network Brisbane
COMPLETEDBrisbane, Queensland, 4006, Australia
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Peter MacCallum Cancer Centre
COMPLETEDMelbourne, Victoria, 3000, Australia
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Richmond Pharmacology Ltd
COMPLETEDLondon, London, SE1 1YR, United Kingdom
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Royal Melbourne Hospital
RECRUITINGParkville, Victoria, 3052, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••