Could an HIV drug help treat rett syndrome?

NCT ID NCT07640503

ENROLLING_BY_INVITATION Disease control Sponsor: Maria Denise Fernandes Carvalho de Andrade Source: ClinicalTrials.gov ↗

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.

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.

Rett syndrome

As listed by the trial registrant

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

RETT SYNDROME

Contacts and locations

Locations

GenClinics

Fortaleza, Ceará, Brazil

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