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Could an HIV drug help treat rett syndrome?

NCT ID NCT07640503

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 10 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2025

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 years and older

Sex

Female participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Clinical and molecular diagnosis of Rett syndrome; * Age 2 years or older at the time of enrollment; * Ability to swallow liquid medication; * Stable clinical condition, as determined by the study investigator; * Availability of a parent or legal guardian able to provide informed consent and comply with study procedures; * Willingness of the participant and/or legal guardian to comply with study visits and assessments. Exclusion Criteria: * Known hypersensitivity or contraindication to lamivudine; * Severe hepatic or renal impairment that, in the investigator's judgment, would preclude safe participation; * Use of investigational drugs or participation in another clinical trial within a defined washout period before enrollment; * Presence of any medical condition or acute illness that could interfere with study participation or outcome assessment, as determined by the investigator; * Inability to undergo blood collection or skin biopsy procedures required for the study; * Any condition that, in the opinion of the investigator, would place the participant at undue risk or compromise adherence to the study protocol.

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • GenClinics

    Fortaleza, Ceará, Brazil

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