Clinical trial NCT06199700: Esketamine for the Treatment of Rett Syndrome
Official title: An Exploratory Trial of Esketamine for the Treatment of Rett Syndrome
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: EARLY_PHASE1
Conditions: Rett Syndrome
Keywords: Rett syndrome
Esketamine
efficacy
safety
Lead sponsor: Children's Hospital of Fudan University
Sponsor class: OTHER
First posted: 2024-01-10
Enrollment: 3
Sex: FEMALE
Minimum age (years): 5
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Efficacy of treatment with Esketamine in girls with RTT
timeFrame: baseline, week 5, and month 6
measure: Incidence of Adverse events
timeFrame: baseline, week 5
measure: Co-outcome for efficacy of treatment with Esketamine in girls with RTT
timeFrame: baseline, week 5, and month 6
Secondary outcomes:
measure: Autism Diagnostic Observation Schedule, Second Edition (ADOS-2)
timeFrame: baseline, week 5, and month 6
measure: Griffiths Scales of Child Development
timeFrame: baseline, week 5, and month 6
measure: Revised-Motor Behavior Assessment scale (R-MBA)
timeFrame: baseline, week 5, and month 6
measure: Rett Syndrome Severity Scale (RSSS)
timeFrame: baseline, week 5, and month 6
measure: Sleep improvement by Esketamine treatment
timeFrame: baseline, week 5, and month 6
measure: Behavior observation
timeFrame: baseline, week 5, and month 6
measure: Brain image change
timeFrame: baseline, week 5, and month 6
measure: Brain function change
timeFrame: baseline, week 5, and month 6
Other outcomes:
Locations:
city: Xiamen
country: China
facility: Xiamen Children's Hospital (Children's Hospital of Fudan University at Xiamen)
state: Fujian
Study officials:
affiliation: Children's Hospital of Fudan University
name: Huiping Li
role: PRINCIPAL_INVESTIGATOR
References: