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| ID | Type | Description | Link |
|---|---|---|---|
| XE2023-ETBJ-D01 | Other Grant/Funding Number | Fujian Province Clinical Key Specialty Construction Project |
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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:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Esketamine hydrochloride | Drug | Intravenous infusion of Ketamine |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Autism Diagnostic Observation Schedule, Second Edition (ADOS-2) | Total score,; Minimum value 0; Maximum values 28; higher scores mean a worse outcome. | baseline, week 5, and month 6 |
| Griffiths Scales of Child Development |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Huiping Li | Children's Hospital of Fudan University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Xiamen Children's Hospital (Children's Hospital of Fudan University at Xiamen) | Xiamen | Fujian | 361006 | China |
all collected IPD, all IPD that underlie results in a publication
starting 6 months after publication,for one year
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| ID | Term |
|---|---|
| D015518 | Rett Syndrome |
| ID | Term |
|---|---|
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
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Development quotient; Minimum value 0; Maximum values 96; higher scores mean a better outcome.
| baseline, week 5, and month 6 |
| Revised-Motor Behavior Assessment scale (R-MBA) | Subscale score, 0-100, higher sores means a worse outcome | baseline, week 5, and month 6 |
| Rett Syndrome Severity Scale (RSSS) | Subscale score, 0-21, higher sores means a worse outcome | baseline, week 5, and month 6 |
| Sleep improvement by Esketamine treatment | Sleep record | baseline, week 5, and month 6 |
| Behavior observation | Unusual hand movements duration per hour | baseline, week 5, and month 6 |
| Brain image change | Magnetic Resonance Imaging (MRI);brain network connectivity | baseline, week 5, and month 6 |
| Brain function change | Electroencephalogram (EEG); brain-wave activity | baseline, week 5, and month 6 |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020271 | Heredodegenerative Disorders, Nervous System |