Clinical TrialNeurocognitive DisordersEsketamineActive not recruiting

Esketamine for the Treatment of Rett Syndrome

This interventional trial (n=3) will assess the efficacy and safety of esketamine for the treatment of Rett Syndrome (RTT) in children aged 5 to 10 years.

Target Enrollment
3 participants
Study Type
Phase I interventional
Design
Non-randomized

Detailed Description

This single-group interventional study will evaluate the efficacy and safety of intravenous esketamine (0.25 mg/kg) given weekly for five weeks in children aged 5–10 years with classic Rett syndrome caused by MECP2 mutation.

Participants receive five once-weekly 40-minute IV infusions (esketamine diluted in 20 ml saline). Outcomes include disease severity measures and safety assessments; stable seizure status is required at enrolment.

Study Protocol

Preparation

sessions

Dosing

5 sessions
40 min each

Integration

sessions

Study Arms & Interventions

Esketamine

experimental

IV esketamine 0.25 mg/kg weekly for 5 weeks

Interventions

  • Esketamine0.25 mg/kg
    via IV• weekly• 5 doses total

    Diluted in 20 ml saline; 40-minute infusion

Participants

Ages
5 – 10
Sexes
female

Inclusion Criteria

  • Classic/typical Rett syndrome
  • Causing mutation in MECP2 gene
  • Stable pattern of seizures, or no seizures for at least 8 weeks

Exclusion Criteria

  • Use of other drugs that interact with esketamine (e.g., thyroxine, meglumine diatrizoate, aminophylline, diazepam or midazolam, antihypertensives or central nervous depressants, halogenated general anesthetics, tubocurarine, atracurium)
  • Conditions with hypertension, elevated intracranial pressure, or high intraocular pressure
  • Unstable systemic illness other than Rett syndrome (clinically significant cardiovascular, endocrine, renal, hepatic, respiratory, or gastrointestinal disease) or major surgery planned during the study
  • Clinically important variations in medication use

Study Details

  • Status
    Active not recruiting
  • Phase
    Phase I
  • Type
    interventional
  • Design
    Non-randomized
  • Target Enrollment3 participants
  • Timeline
    Start: 2023-11-15
    End: 2025-05-01
  • Compound
    Esketamine
  • Topic
    Neurocognitive Disorders

Locations

Xiamen Children's Hospital (Children's Hospital of Fudan University at Xiamen) — Xiamen, Fujian, China

Your Library