- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT06199700
Esketamine for the Treatment of Rett Syndrome
December 28, 2023 updated by: Children's Hospital of Fudan University
An Exploratory Trial of Esketamine for the Treatment of Rett Syndrome
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.
Study Overview
Study Type
Interventional
Enrollment (Estimated)
3
Phase
- Early Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Shu Liu
- Phone Number: +86 17612101065
- Email: mickeyshu@126.com
Study Locations
-
-
Fujian
-
Xiamen, Fujian, China, 361006
- Recruiting
- Xiamen Children's Hospital (Children's Hospital of Fudan University at Xiamen)
-
Contact:
- Min Zhang
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
Accepts Healthy Volunteers
No
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: 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.
|
Intravenous infusion of Ketamine
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Efficacy of treatment with Esketamine in girls with RTT
Time Frame: baseline, week 5, and month 6
|
Rett Syndrome Behaviour Questionnaire (RSBQ) total score
|
baseline, week 5, and month 6
|
Incidence of Adverse events
Time Frame: baseline, week 5
|
Safety of treatment with Esketamine in girls with RTT
|
baseline, week 5
|
Co-outcome for efficacy of treatment with Esketamine in girls with RTT
Time Frame: baseline, week 5, and month 6
|
Clinical Global Impressions Scale-improvement (CGI-I) score
|
baseline, week 5, and month 6
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Autism Diagnostic Observation Schedule, Second Edition (ADOS-2)
Time Frame: baseline, week 5, and month 6
|
Total score
|
baseline, week 5, and month 6
|
Griffiths Scales of Child Development
Time Frame: baseline, week 5, and month 6
|
Development quotient
|
baseline, week 5, and month 6
|
Revised-Motor Behavior Assessment scale (R-MBA)
Time Frame: baseline, week 5, and month 6
|
Subscale score, 0-100, higher sores means a worse outcome
|
baseline, week 5, and month 6
|
Rett Syndrome Severity Scale (RSSS)
Time Frame: baseline, week 5, and month 6
|
Subscale score, 0-21, higher sores means a worse outcome
|
baseline, week 5, and month 6
|
Sleep improvement by Esketamine treatment
Time Frame: baseline, week 5, and month 6
|
Sleep record
|
baseline, week 5, and month 6
|
Behavior observation
Time Frame: baseline, week 5, and month 6
|
Unusual hand movements duration per hour
|
baseline, week 5, and month 6
|
Magnetic Resonance Imaging (MRI)
Time Frame: baseline, week 5, and month 6
|
Brain network connectivity
|
baseline, week 5, and month 6
|
Electroencephalogram (EEG)
Time Frame: baseline, week 5, and month 6
|
Abnormal brain-wave activity
|
baseline, week 5, and month 6
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Investigators
- Principal Investigator: Huiping Li, Children's Hospital of Fudan University
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
November 15, 2023
Primary Completion (Estimated)
October 31, 2024
Study Completion (Estimated)
May 1, 2025
Study Registration Dates
First Submitted
November 21, 2023
First Submitted That Met QC Criteria
December 28, 2023
First Posted (Actual)
January 10, 2024
Study Record Updates
Last Update Posted (Actual)
January 10, 2024
Last Update Submitted That Met QC Criteria
December 28, 2023
Last Verified
December 1, 2023
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Pathologic Processes
- Nervous System Diseases
- Neurologic Manifestations
- Neurobehavioral Manifestations
- Disease
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Mental Retardation, X-Linked
- Intellectual Disability
- Heredodegenerative Disorders, Nervous System
- Syndrome
- Rett Syndrome
- Psychotropic Drugs
- Antidepressive Agents
- Esketamine
Other Study ID Numbers
- EskRTT2023
- XE2023-ETBJ-D01 (Other Grant/Funding Number: Fujian Province Clinical Key Specialty Construction Project)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
YES
IPD Plan Description
all collected IPD, all IPD that underlie results in a publication
IPD Sharing Time Frame
starting 6 months after publication,for one year
IPD Sharing Supporting Information Type
- STUDY_PROTOCOL
- SAP
- ANALYTIC_CODE
- CSR
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Rett Syndrome
-
University of Alabama at BirminghamUniversity of Colorado, Denver; Eunice Kennedy Shriver National Institute of... and other collaboratorsCompletedRett Syndrome, Preserved Speech Variant | Mecp2 Duplication Syndrome | Rett-related DisordersUnited States
-
Children's Hospital of PhiladelphiaNational Institute of Neurological Disorders and Stroke (NINDS); Vanderbilt...RecruitingRett Syndrome | Rett Syndrome, Atypical | RTTUnited States
-
University of Alabama at BirminghamUniversity of Colorado, Denver; Eunice Kennedy Shriver National Institute of... and other collaboratorsCompletedRett Syndrome | MECP2 Duplication Disorder | Rett-related DisorderUnited States
-
International Rett Syndrome FoundationBaylor College of Medicine; Children's Hospital of Philadelphia; University of... and other collaboratorsRecruitingNervous System Diseases | Neurologic Manifestations | Neurobehavioral Manifestations | Genetic Diseases, X-Linked | Intellectual Disability | Neurodevelopmental Disorders | Neurologic Disorder | Rett Syndrome | Genetic Disease | Rett Syndrome, AtypicalUnited States
-
Assistance Publique - Hôpitaux de ParisCompleted
-
University Hospital, Basel, SwitzerlandNovartisCompletedRett's SyndromeSwitzerland
-
ACADIA Pharmaceuticals Inc.CompletedRett SyndromeUnited States
-
ACADIA Pharmaceuticals Inc.Completed
-
IRCCS Eugenio MedeaAssociazione Italiana Rett (AIRett) O.n.l.u.s.Completed
-
Rett Syndrome Research TrustDuke UniversityCompletedRett SyndromeUnited States
Clinical Trials on Esketamine hydrochloride
-
Janssen Research & Development, LLCCompleted
-
Janssen Research & Development, LLCCompleted
-
Wuhan Union Hospital, ChinaRecruitingSepsis | Inflammatory Response | Immunosuppression | EsketamineChina
-
bo xuNot yet recruiting
-
University Hospital, Basel, SwitzerlandCompleted
-
Tianjin Medical University General HospitalNot yet recruiting
-
Reinier de Graaf GroepRecruitingEndometriosis | Chronic Pelvic Pain SyndromeNetherlands
-
Second Affiliated Hospital, School of Medicine,...Unknown
-
Janssen Research & Development, LLCCompletedTreatment Resistant Depressive DisorderUnited States, Japan, Belgium
-
The Second Hospital of Anhui Medical UniversityNot yet recruiting