Esketamine for the Treatment of Rett Syndrome

Rett Syndrome Clinical Trial

Official title:

An Exploratory Trial of Esketamine for the Treatment of Rett Syndrome

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06199700
• Other study ID #: EskRTT2023
• Secondary ID: XE2023-ETBJ-D01
• Status: Recruiting
• Phase: Early Phase 1
• Start date: November 15, 2023
• Est. completion date: May 1, 2025

Study information

• Verified date: December 2023
• Source: Children's Hospital of Fudan University
• Contact: Shu Liu
• Phone: +86 17612101065
• Email: mickeyshu[@]126.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 3
• Est. completion date: May 1, 2025
• Est. primary completion date: October 31, 2024
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 5 Years to 10 Years

Eligibility

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

Related Conditions & MeSH terms

• Rett Syndrome
• Syndrome

Intervention

Drug:
• Esketamine hydrochloride
Intravenous infusion of Ketamine

Locations

Country	Name	City	State
China	Xiamen Children's Hospital (Children's Hospital of Fudan University at Xiamen)	Xiamen	Fujian

Sponsors (1)

Lead Sponsor	Collaborator
Children's Hospital of Fudan University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Efficacy of treatment with Esketamine in girls with RTT	Rett Syndrome Behaviour Questionnaire (RSBQ) total score	baseline, week 5, and month 6
Primary	Incidence of Adverse events	Safety of treatment with Esketamine in girls with RTT	baseline, week 5
Primary	Co-outcome for efficacy of treatment with Esketamine in girls with RTT	Clinical Global Impressions Scale-improvement (CGI-I) score	baseline, week 5, and month 6
Secondary	Autism Diagnostic Observation Schedule, Second Edition (ADOS-2)	Total score	baseline, week 5, and month 6
Secondary	Griffiths Scales of Child Development	Development quotient	baseline, week 5, and month 6
Secondary	Revised-Motor Behavior Assessment scale (R-MBA)	Subscale score, 0-100, higher sores means a worse outcome	baseline, week 5, and month 6
Secondary	Rett Syndrome Severity Scale (RSSS)	Subscale score, 0-21, higher sores means a worse outcome	baseline, week 5, and month 6
Secondary	Sleep improvement by Esketamine treatment	Sleep record	baseline, week 5, and month 6
Secondary	Behavior observation	Unusual hand movements duration per hour	baseline, week 5, and month 6
Secondary	Magnetic Resonance Imaging (MRI)	Brain network connectivity	baseline, week 5, and month 6
Secondary	Electroencephalogram (EEG)	Abnormal brain-wave activity	baseline, week 5, and month 6