Esketamine for the Treatment of Rett Syndrome

NCT06199700 | Active Not Recruiting Early Phase 1

• 2 other identifiers: EskRTT2023XE2023-ETBJ-D01
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

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.

Conditions

Rett Syndrome

Keywords

Rett syndrome; Esketamine; efficacy; safety

Outcome Measures

Primary Outcomes (3)

• Efficacy of treatment with Esketamine in girls with RTT

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

  baseline, week 5, and month 6

• Incidence of Adverse events

  Safety of treatment with Esketamine in girls with RTT

  baseline, week 5

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

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

  baseline, week 5, and month 6

Secondary Outcomes (8)

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

  baseline, week 5, and month 6

• Griffiths Scales of Child Development

  baseline, week 5, and month 6

• Revised-Motor Behavior Assessment scale (R-MBA)

  baseline, week 5, and month 6

• Rett Syndrome Severity Scale (RSSS)

  baseline, week 5, and month 6

• Sleep improvement by Esketamine treatment

  baseline, week 5, and month 6

Study Arms (1)

Esketamine treatment

EXPERIMENTAL

Esketamine, diluted with 20ml Saline at a dose of 0.25mg/kg, intravenously for 40 minutes, once a week; 5 weeks in total.

Drug: Esketamine hydrochloride

Interventions

Esketamine hydrochloride DRUG

Intravenous infusion of Ketamine

Esketamine treatment

Eligibility Criteria

• Age: 5 Years - 10 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's Hospital of Fudan University: lead

Study Sites (1)

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

Xiamen, Fujian, 361006, China

Location

MeSH Terms

Conditions

Rett Syndrome

Condition Hierarchy (Ancestors)

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

Study Officials

• Huiping Li

  Children's Hospital of Fudan University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 21, 2023
• First Posted: January 10, 2024
• Study Start: November 15, 2023
• Primary Completion: April 4, 2024
• Study Completion: May 1, 2025
• Last Updated: February 18, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR, ANALYTIC CODE
• Time Frame: starting 6 months after publication，for one year

Locations

CN (1)