Could an HIV drug help treat rett syndrome?
NCT ID NCT07640503
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 2 trial tests whether lamivudine, an antiviral drug, can improve symptoms in people with Rett syndrome, a rare genetic disorder that affects movement, communication, and behavior. Ten participants will take the drug by mouth for a set period, and researchers will measure changes in symptom severity, behavior, and safety. The study also looks at blood markers to understand how the drug works in the body.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Lamivudine (an antiviral drug, given orally)
What this could lead to
If it works, this could point toward a treatment that reduces symptoms and inflammation in Rett syndrome, potentially improving quality of life.
What could go wrong
This is a very small early-phase trial with only 10 people and no placebo group, so results may not be reliable or generalizable. Lamivudine may cause side effects or show no benefit.
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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Locations
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GenClinics
Fortaleza, Ceará, Brazil