Lamivudine as a Novel Clinical Effort for Rett Syndrome

Plain-language summary

This study evaluates the effect of the drug Lamivudine in people with Rett syndrome. People aged 2 and over with a clinical and molecular diagnosis of Rett syndrome who can swallow liquid medication and are in a stable clinical condition may take part. The study is in Phase 2 and enrolls participants by invitation.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 2

Status

Enrolling by invitation

Sponsor

Maria Denise Fernandes Carvalho de Andrade

Intervention

Lamivudine

Start date

11/5/2025

Estimated completion

12/1/2027

Countries

Brazil

Related genes

Locations

• Fortaleza, Brazil

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.