Esketamine for the Treatment of Rett Syndrome

NCT ID: NCT06199700

Last Updated: 2025-02-18

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 3 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-11-15
• Study Completion Date: 2025-05-01

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Esketamine treatment

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

Group Type: EXPERIMENTAL

Esketamine hydrochloride

Intervention Type: DRUG

Intravenous infusion of Ketamine

Interventions

Esketamine hydrochloride

Intravenous infusion of Ketamine

Intervention Type: DRUG

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

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 10 Years
• Eligible Sex: FEMALE
• Accepts Healthy Volunteers: No

Sponsors

Children's Hospital of Fudan University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Huiping Li

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Fudan University

Locations

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

Xiamen, Fujian, China

Countries

China

Other Identifiers

XE2023-ETBJ-D01

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

EskRTT2023

Identifier Type: -

Identifier Source: org_study_id