Could HIV meds tame a rare immune disease in kids?
NCT ID NCT02363452
First seen Nov 01, 2025 · Last updated Jun 22, 2026 · Updated 40 times
Summary
This study tested whether HIV drugs (reverse transcriptase inhibitors) can lower abnormal immune activity in children with Aicardi-Goutières syndrome, a rare genetic disorder that affects the brain and immune system. Eleven children took the drugs to see if their interferon levels—a marker of immune overactivity—would return to normal. The goal was to find a new way to control the disease.
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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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Contacts and locations
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Locations
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Hôpital Necker - Enfants Malades
Paris, 75015, France
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
reverse transcriptase inhibitors (zidovudine, lamivudine, abacavir)
What this could lead to
If successful, this could point toward a treatment that controls the overactive immune response in Aicardi-Goutières syndrome, potentially reducing disease severity.
What could go wrong
This is a very small pilot study (11 children) with no control group, so results may not be conclusive. The drugs may not lower interferon levels enough or could cause side effects.
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.