Lamivudine as a Novel Clinical Effort for Rett Syndrome (LANCE)

June 6, 2026 updated by: Maria Denise Fernandes Carvalho de Andrade

Lamivudine in Individuals With Rett Syndrome: Clinical, Biochemical, and Cellular Evaluation

Rett syndrome (RTT) is a rare genetic neurodevelopmental disorder caused primarily by mutations in the MECP2 gene, leading to progressive impairments in motor function, communication, and behavior following an initial period of apparently typical development. Currently, there are no treatments that change the course of the disease, and clinical care is largely focused on managing symptoms. Loss of MeCP2 function has been associated with increased activity of the LINE-1 (L1) retroelement, which may contribute to neuroinflammation and cellular stress in the brain. Lamivudine, a nucleoside reverse transcriptase inhibitor widely used in antiviral therapy, can inhibit L1 reverse transcription and has shown beneficial effects in preclinical models of RTT, including reductions in inflammatory and oxidative stress markers and improvements in neurological and behavioral outcomes. This study aims to evaluate the safety and potential clinical and biological effects of lamivudine in individuals with Rett syndrome using a before-and-after treatment design. Participants will receive oral lamivudine and will undergo clinical assessments and laboratory testing before and after the treatment period to evaluate changes in symptom severity, functional status, quality of life, seizure activity, and biomarkers related to inflammation and neurodevelopment. Biological samples will also be collected to support translational laboratory studies aimed at improving understanding of disease mechanisms and treatment response in RTT. Results from this study may help determine whether lamivudine is a safe and promising therapeutic option and may guide future clinical research in this population.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Enrolling by invitation

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Rett syndrome (RTT) is a rare genetic neurodevelopmental disorder caused primarily by loss-of-function mutations in the MECP2 gene, leading to progressive impairments in motor, cognitive, and communicative function after an initial period of apparently typical development. Currently, there are no medications that change the course of the disease, and treatment is mainly focused on managing symptoms.

Functional MeCP2 represses the activity of the LINE-1 (L1) retroelement, and loss of this repression may result in increased L1 accumulation in glial cells in the brain, promoting genomic instability, oxidative stress, and neuroinflammation associated with RTT pathophysiology.

Lamivudine inhibits L1 reverse transcription and has shown beneficial effects in preclinical neuronal culture and MeCP2-deficient mouse models, including reduced inflammatory and oxidative stress markers, improved neurological and behavioral outcomes, and increased survival. In this study, each participant is evaluated before and after receiving treatment, allowing changes to be assessed within the same individual over time. Following screening and baseline assessments, participants enter a treatment phase during which oral lamivudine is administered for a defined period. Longitudinal clinical, laboratory, and safety evaluations are conducted at scheduled visits, followed by a post-treatment follow-up period to assess the persistence of effects.

A 24-week intervention period was selected as a feasible and acceptable duration for participants and caregivers and as an appropriate timeframe for an initial open-label evaluation of therapeutic potential. This duration is expected to provide sufficient exposure to lamivudine to allow detection of short-term clinical and biological changes suggested by preclinical studies, while also enabling systematic assessment of safety and tolerability in this population.

Participants may continue their usual medical treatments, including medications for seizures and other supportive therapies, as long as these treatments remain stable during the study, unless a change is medically necessary.

Caregivers receive standardized instructions on medication administration, and adherence is monitored throughout the treatment phase through caregiver reporting and visit-based verification to ensure accurate assessment of treatment exposure.

Clinical assessments employ validated clinician and caregiver-reported instruments to evaluate symptom severity, functional status, quality of life, and seizure activity, capturing changes across motor, behavioral, and daily functioning domains relevant to RTT. Safety is monitored continuously through adverse event surveillance, routine clinical evaluations, and laboratory testing, with predefined criteria for treatment interruption if clinically indicated. Venous blood samples are collected at baseline and during treatment to evaluate systemic biomarkers related to oxidative stress, inflammation, and neurodevelopment. These measures complement clinical outcomes and explore potential mechanistic effects of lamivudine treatment.

In addition to the biomarkers reported as outcome measures, a panel of exploratory, hypothesis-generating biomarkers will be analyzed, including markers of oxidative stress, neuroinflammation and neurodevelopment, mitochondrial and energy metabolism, and nutritional and micronutrient status. These analyses are not considered clinical endpoints and are not reported as outcome measures.

As a translational research component, skin punch biopsies from a subset of participants are used to establish a dermal fibroblast biobank, enabling the generation of patient-derived induced pluripotent stem cells and brain organoids. These models will be used in exploratory laboratory studies to investigate cellular and molecular mechanisms in RTT and responses to lamivudine, without being considered clinical endpoints. Based on preclinical evidence and the proposed mechanism of action, lamivudine treatment is expected to be safe and well-tolerated in individuals with Rett syndrome. Clinically, it may lead to improvements in motor function, social engagement, and overall symptom severity, while reducing seizure frequency and enhancing quality of life. Biologically, lamivudine is anticipated to modulate oxidative stress and inflammatory pathways, potentially restoring a more balanced neurodevelopmental environment. The combined clinical and laboratory observations from this study will provide critical insights into the therapeutic potential of lamivudine and inform future research directions in RTT.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
10

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Brazil
    • Ceará
      • Fortaleza, Ceará, Brazil
        • GenClinics

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Lamivudine Treatment
Approximately 10 participants aged ≥2 years with a confirmed clinical and molecular diagnosis of Rett syndrome will receive oral lamivudine using weight-based dosing for 24 weeks following baseline clinical and laboratory assessments. Participants will undergo regular monitoring for safety, tolerability, and efficacy, including clinical evaluations, laboratory testing, and assessment of neurological and functional outcomes. Dose adjustments or discontinuation may occur in response to adverse events. Blood samples will be collected for biomarker analyses, with an optional skin biopsy in a subset of participants for exploratory research.
Drug: Lamivudine
Lamivudine will be given orally to participants with Rett syndrome using weight-based dosing after baseline clinical and laboratory assessments. Children <14 kg will receive oral solution at 4 mg/kg twice daily (max 300 mg/day). Participants ≥14 kg will receive tablets by weight: 14-20 kg, 150 mg/day; 20-25 kg, 225 mg/day; >25 kg, 300 mg/day. Medication may be taken with or without food. Tablets should not be crushed unless swallowing is difficult. If needed, they may be crushed and mixed with liquid or soft food and taken immediately, or oral solution may be used. Caregivers will be instructed on dosing and concomitant medications. Treatment lasts 24 weeks with weekly safety, tolerability, and efficacy monitoring. Dose adjustment or discontinuation may occur if significant adverse events arise.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Change in Rett Syndrome Symptom Severity (RARS)
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in Rett syndrome symptom severity assessed using the Rett Assessment Rating Scale (RARS), comparing total scores obtained at baseline with scores obtained after treatment with lamivudine and at post-treatment follow-up. Total scores range from 0 to 128, with higher scores indicating greater symptom severity. A reduction in total score will be interpreted as clinical improvement.

Unit of Measure: Units on a scale (0-128)
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in Rett Syndrome Behavioral Symptoms
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in Rett syndrome behavioral symptoms assessed using the Rett Syndrome Behaviour Questionnaire (RSBQ), comparing total scores obtained at baseline with scores obtained at the end of treatment with lamivudine and at post-treatment follow-up. The RSBQ is a Rett syndrome-specific instrument used to assess behavioral, autonomic, and functional manifestations associated with Rett syndrome, including breathing abnormalities, irritability, anxiety, mood, repetitive hand movements, communication, and social interaction.

The RSBQ consists of 45 items scored from 0 to 2, where 0 indicates "not true," 1 indicates "somewhat or sometimes true," and 2 indicates "often true" or "very true." Total scores range from 0 to 90, with higher scores indicating greater frequency or severity of behavioral symptoms and worse clinical status. A reduction in total score after treatment will be interpreted as improvement in Rett syndrome behavioral symptoms.

Unit of Measure: Units on a scale (0-90)
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Safety of Lamivudine: Occurrence of Adverse Events
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Safety assessed by the number and proportion of participants experiencing at least one adverse event (serious or non-serious) during treatment with lamivudine, based on clinical assessment and laboratory monitoring.

Unit of Measure: Number and proportion of participants with adverse events
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Causality of Adverse Events (LCAT)
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Causality of adverse events related to lamivudine assessed using the Liverpool Adverse Drug Reaction Causality Assessment Tool (LCAT). The tool classifies the causal relationship between the drug and each adverse event as unlikely, possible, probable, or definite. Higher categories indicate a greater likelihood of a causal relationship between the adverse event and lamivudine.

Unit of Measure: Number and proportion of adverse events in each causality category
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in Health-Related Quality of Life: Pediatric Participants (PedsQL Neuromuscular Module)
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in health-related quality of life assessed primarily by the PedsQL 3.0 Neuromuscular Module (PedsQL NMM), caregiver-report adapted version, comparing baseline scores with scores at the end of treatment and at follow-up. The PedsQL NMM yields transformed scores from 0 to 100; higher scores indicate better health-related quality of life and lower symptom impact. An increase after treatment is interpreted as improvement.

Unit of Measure: Units on a scale (0-100)
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in Health-Related Quality of Life - Adult participants (SF-36)
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in health-related quality of life assessed by the Short Form (36) Health Survey (SF-36) in adult participants, comparing baseline scores with scores at the end of treatment and follow-up. SF-36 domain scores range from 0 to 100, with higher scores indicating better quality of life. An increase in score after treatment is interpreted as improvement.

Unit of Measure: Units on a scale (0-100)
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in Adaptive Behavior (Vineland Adaptive Behavior Scales - Adaptive Behavior Composite)
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in adaptive behavior assessed using the Vineland Adaptive Behavior Scales, reported as the Adaptive Behavior Composite standard score, comparing baseline with end-of-treatment and follow-up values. Standard scores have a mean of 100 and a standard deviation of 15, higher scores indicate better adaptive functioning.

Unit of Measure: Standard score
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Presence of Epileptic Seizures
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Presence of epileptic seizures during treatment with lamivudine, assessed by the number of participants experiencing at least one epileptic seizure after the start of the intervention. Data obtained from caregiver records and clinical assessment by the research team.

Unit of Measure: Participants
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Frequency of Epileptic Seizures
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in the frequency of epileptic seizures, comparing the mean number of seizures per participant at baseline with the mean number recorded during treatment with lamivudine. Information obtained from a caregiver-completed seizure diary, confirmed at follow-up visits.

Unit of Measure: Number of seizures per month
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Need for Medical Intervention Related to Epileptic Seizures
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Need for medical intervention related to epileptic seizures during treatment with lamivudine, assessed by the number of participants requiring emergency care, hospitalization, or rescue medication due to an epileptic seizure.

Unit of Measure: Participants
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in Caregiver Burden (Zarit Burden Interview, ZBI-22)
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in caregiver burden assessed using the Zarit Burden Interview (ZBI-22), comparing baseline scores with scores at the end of treatment and at follow-up. Total scores range from 0 to 88, with higher scores indicating greater caregiver burden. A reduction in total score after treatment is interpreted as improvement.

Unit of Measure: Units on a scale (0-88)
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in Global Clinical Severity (CGI-S)
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in global clinical severity assessed by the Clinical Global Impression Severity (CGI-S) scale, comparing the score at baseline with the score at the end of treatment. The CGI-S is a 7-point clinician-rated scale ranging from 1 to 7, where 1 = normal (not at all ill) and 7 = among the most extremely ill patients. Higher scores indicate greater global clinical severity. A reduction after treatment is interpreted as clinical improvement.

Unit of Measure: Units on a scale (1-7)
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Global Clinical Improvement (CGI-I)
Time Frame: Week 24 and Week 52

Global clinical improvement assessed using the Clinical Global Impression-Improvement scale (CGI-I) at the end of lamivudine treatment and during post-treatment follow-up. Scores range from 1 ("very much improved") to 7 ("very much worse"), with lower scores indicating greater improvement.

Unit of Measure: Units on a scale (1-7)
Week 24 and Week 52
Change in serum C-reactive protein (CRP)
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum C-reactive protein (CRP) concentrations, comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: mg/L
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Monocyte chemoattractant protein-1 (MCP-1)
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum Monocyte chemoattractant protein-1 (MCP-1), comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: pg/mL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Ferritin
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum Ferritin, comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: ng/mL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Interleukin-1 beta (IL-1β)
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum Interleukin-1 beta (IL-1β), comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: pg/mL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Interleukin-6 (IL-6)
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum Interleukin-6 (IL-6), comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: pg/mL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Interleukin-8 (IL-8)
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum Interleukin-8 (IL-8), comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: pg/mL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Interleukin-10 (IL-10)
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum Interleukin-10 (IL-10), comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: pg/mL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Tumor Necrosis Factor alpha (TNF-α)
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum Tumor Necrosis Factor alpha (TNF-α), comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: pg/mL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Aspartate aminotransferase (AST/TGO)
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum Aspartate aminotransferase (AST/TGO) levels comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: U/L
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Alanine aminotransferase (ALT/TGP)
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum Alanine aminotransferase (ALT/TGP) levels comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: U/L
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Alkaline phosphatase
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum Alkaline phosphatase levels comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: U/L
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Urea
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum Urea levels comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: mg/dL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Creatinine
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum Creatinine levels comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: mg/dL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Glucose
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum Glucose levels comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: mg/dL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Uric acid
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum Uric acid levels comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: mg/dL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Total bilirubin
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum total bilirubin levels comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: mg/dL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Direct bilirubin
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum direct bilirubin levels comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: mg/dL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in serum Albumin
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in serum albumin levels comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: g/dL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in Hemoglobin
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in hemoglobin comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: g/dL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in Hematocrit
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in hematocrit comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: %
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in Leukocyte count
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in leukocyte count comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: cells/µL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)
Change in Platelet count
Time Frame: Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Change in platelet count comparing values at baseline with values at the end of treatment and at follow-up.

Unit of Measure: cells/µL
Baseline (Week 0), Week 24 (end of treatment), and Week 52 (post-treatment follow-up)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Maria Denise Fernandes Carvalho de Andrade

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
University of California, San Diego

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Maria Denise Fernandes Carvalho de Andrade, MD, PhD, Universidade Estadual do Ceara

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

  • Martins AMA, Nakashima H, Macia A, et al. Dormant viral pathways underlie space-induced neural senescence: a neuroprotective strategy for spaceflight and neurological diseases. bioRxiv. 2025. doi:10.1101/2025.11.02.686043

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 5, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 1, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 12, 2026

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 6, 2026

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 10, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 10, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 6, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 88455225.8.0000.5534

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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