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Completed

NCT Number: NCT06199700

Esketamine for the Treatment of Rett Syndrome

The goal of this interventional study is to learn about the efficacy and safety of Esketamine for treating children with Rett syndrome (RTT).

The main questions it aims to answer are:

* whether Esketamine treatment is effective in improving symptom severity for RTT. * whether Esketamine is safe in the treatment of RTT. Participants will receive a weekly intravenous infusion of Esketamine for five weeks and will be assessed for disease severity and drug safety.

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Key information

Conditions

Rett Syndrome Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations X-Linked Intellectual Disability

Age range

5 year–10 year

Sex eligibility

Female

Study type

Interventional

Phase

Early Phase 1

Primary location

Xiamen Children's Hospital (Children's Hospital of Fudan University at Xiamen)

Xiamen, Fujian, 361006, China

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Classic/typical RTT
  • Causing mutation in MECP2 gene
  • Stable pattern of seizures, or has had no seizures at least 8 weeks

Exclusion criteria

  • Use of other drugs that interact with Esketamine: thyroxine, meglumine diatrizoate, aminophylline, diazepam or midazolam, drugs for antihypertension or central nervous depressants, halogenated general anesthetics (e.g., halothane), tubocurarine, atracurium
  • Condition with hypertension, high cerebrospinal fluid pressure, and high intraocular pressure
  • Unstable systemic illness other than Rett syndrome: current clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, type 1 diabetes, or uncontrolled type 2 diabetes), renal, hepatic, respiratory, or gastrointestinal disease (such as celiac disease or inflammatory bowel disease), or major surgery planned during the study
  • Clinically important variations in medication use

Treatment and study plan

Esketamine hydrochloride

Drug

Intravenous infusion of Ketamine

Primary outcomes

  1. Efficacy of treatment with Esketamine in girls with RTT

    Time frame: baseline, week 5, and month 6

    Rett Syndrome Behaviour Questionnaire (RSBQ) total score; Minimum value 0; Maximum values 90; higher scores mean a worse outcome.

  2. Incidence of Adverse events

    Time frame: baseline, week 5

    Safety of treatment with Esketamine in girls with RTT

  3. Co-outcome for efficacy of treatment with Esketamine in girls with RTT

    Time frame: baseline, week 5, and month 6

    Clinical Global Impressions Scale-improvement (CGI-I) score; Minimum value 0; Maximum values 7; higher scores mean a worse outcome.

Secondary outcomes

  1. Autism Diagnostic Observation Schedule, Second Edition (ADOS-2)

    Time frame: baseline, week 5, and month 6

    Total score，; Minimum value 0; Maximum values 28; higher scores mean a worse outcome.

  2. Griffiths Scales of Child Development

    Time frame: baseline, week 5, and month 6

    Development quotient; Minimum value 0; Maximum values 96; higher scores mean a better outcome.

  3. Revised-Motor Behavior Assessment scale (R-MBA)

    Time frame: baseline, week 5, and month 6

    Subscale score, 0-100, higher sores means a worse outcome

  4. Rett Syndrome Severity Scale (RSSS)

    Time frame: baseline, week 5, and month 6

    Subscale score, 0-21, higher sores means a worse outcome

  5. Sleep improvement by Esketamine treatment

    Time frame: baseline, week 5, and month 6

    Sleep record

  6. Behavior observation

    Time frame: baseline, week 5, and month 6

    Unusual hand movements duration per hour

  7. Brain image change

    Time frame: baseline, week 5, and month 6

    Magnetic Resonance Imaging (MRI)；brain network connectivity

  8. Brain function change

    Time frame: baseline, week 5, and month 6

    Electroencephalogram (EEG); brain-wave activity

Sponsors and collaborators

Lead sponsor

Children's Hospital of Fudan University

Other

Registry information

Official study title

An Exploratory Trial of Esketamine for the Treatment of Rett Syndrome

Important dates

Study start
2023
Primary completion
2025
Study completion
2025
First posted
Jan 10, 2024
Registry last updated
May 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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