Rett Syndrome
Conditions
Keywords
Rett syndrome, Esketamine, efficacy, safety
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.
Interventions
Intravenous infusion of Ketamine
Sponsors
Children's Hospital of Fudan University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
FEMALE
Age
5 Years to 10 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy of treatment with Esketamine in girls with RTT | baseline, week 5, and month 6 | Rett Syndrome Behaviour Questionnaire (RSBQ) total score; Minimum value 0; Maximum values 90; higher scores mean a worse outcome. |
| Incidence of Adverse events | baseline, week 5 | Safety of treatment with Esketamine in girls with RTT |
| Co-outcome for efficacy of treatment with Esketamine in girls with RTT | baseline, week 5, and month 6 | Clinical Global Impressions Scale-improvement (CGI-I) score; Minimum value 0; Maximum values 7; higher scores mean a worse outcome. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Autism Diagnostic Observation Schedule, Second Edition (ADOS-2) | baseline, week 5, and month 6 | Total score，; Minimum value 0; Maximum values 28; higher scores mean a worse outcome. |
| Griffiths Scales of Child Development | baseline, week 5, and month 6 | Development quotient; Minimum value 0; Maximum values 96; higher scores mean a better outcome. |
| Revised-Motor Behavior Assessment scale (R-MBA) | baseline, week 5, and month 6 | Subscale score, 0-100, higher sores means a worse outcome |
| Rett Syndrome Severity Scale (RSSS) | baseline, week 5, and month 6 | Subscale score, 0-21, higher sores means a worse outcome |
| Sleep improvement by Esketamine treatment | baseline, week 5, and month 6 | Sleep record |
| Behavior observation | baseline, week 5, and month 6 | Unusual hand movements duration per hour |
| Brain image change | baseline, week 5, and month 6 | Magnetic Resonance Imaging (MRI)；brain network connectivity |
| Brain function change | baseline, week 5, and month 6 | Electroencephalogram (EEG); brain-wave activity |
Countries
China
Contacts
PRINCIPAL_INVESTIGATORHuiping Li
Children's Hospital of Fudan University
Outcome results
None listed