Rett SyndromeEarly Phase 1NCT06199700
Esketamine for the Treatment of Rett Syndrome
A Early Phase 1 study testing Esketamine hydrochloride in people with Rett Syndrome, run by Children's Hospital of Fudan University at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 to 10 years, women only
- Conditions the study is for
- Rett Syndrome
- What is being tested?
- Esketamine hydrochloride
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteChina
- Study dates
- 15 Nov 2023to 30 Dec 2025
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 10 years
- Women only
- No healthy volunteers
- Child
Who may be able to join
- Classic/typical RTT
- Causing mutation in MECP2 gene
- Stable pattern of seizures, or has had no seizures at least 8 weeks
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
China1 site
Xiamen Children's Hospital (Children's Hospital of Fudan University at Xiamen)
Xiamen, Fujian, 361006
About this study
Official title: 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.
- Conditions
- Rett Syndrome
- Being tested
- Esketamine hydrochloride
- Keywords
- Rett syndrome, Esketamine, efficacy, safety
- Medical subject headings
- Rett Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Esketamine hydrochlorideDrug
Intravenous infusion of Ketamine
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Esketamine hydrochloride |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: baseline, week 5, and month 6
Incidence of Adverse events
Safety of treatment with Esketamine in girls with RTT
Measured: 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.
Measured: baseline, week 5, and month 6
Secondary outcomes (8)
Autism Diagnostic Observation Schedule, Second Edition (ADOS-2)
Total score，; Minimum value 0; Maximum values 28; higher scores mean a worse outcome.
Measured: baseline, week 5, and month 6
Griffiths Scales of Child Development
Development quotient; Minimum value 0; Maximum values 96; higher scores mean a better outcome.
Measured: baseline, week 5, and month 6
Revised-Motor Behavior Assessment scale (R-MBA)
Subscale score, 0-100, higher sores means a worse outcome
Measured: baseline, week 5, and month 6
Rett Syndrome Severity Scale (RSSS)
Subscale score, 0-21, higher sores means a worse outcome
Measured: baseline, week 5, and month 6
Sleep improvement by Esketamine treatment
Sleep record
Measured: baseline, week 5, and month 6
Behavior observation
Unusual hand movements duration per hour
Measured: baseline, week 5, and month 6
Brain image change
Magnetic Resonance Imaging (MRI)；brain network connectivity
Measured: baseline, week 5, and month 6
Brain function change
Electroencephalogram (EEG); brain-wave activity
Measured: baseline, week 5, and month 6
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2024
- Study start15 Nov 2023
- Primary completion30 Dec 2025
- Study completion30 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rett SyndromeStudies of the same condition, open and under way.
- More trials run by Children's Hospital of Fudan UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Fudan University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Huiping Li, Principal investigator, Children's Hospital of Fudan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06199700
- Sponsor's protocol ID
- EskRTT2023
- Other IDs
- XE2023-ETBJ-D01
- Status last verified
- May 2026
- First submitted
- 21 Nov 2023
- Last updated
- 13 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: all collected IPD, all IPD that underlie results in a publication
Study at a glance
From the registry record, last updated by the sponsor on 13 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Trial of Esketamine for the Treatment of Rett Syndrome
- ClinicalTrials.gov ID
- NCT06199700
- Acronym / sponsor ID
- EskRTT2023
- Conditions
- Rett Syndrome
- Keywords
- Rett syndrome, Esketamine, efficacy, safety
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Children's Hospital of Fudan UniversityUniversity, hospital or other
- Study start
- 15 Nov 2023 (actual)
- Primary completion
- 30 Dec 2025 (actual)
- Study completion
- 30 Dec 2025 (actual)
- First posted
- 10 Jan 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Esketamine treatment | Experimental | Drug: Esketamine hydrochlorideEsketamine, diluted with 20ml Saline at a dose of 0.25mg/kg, intravenously for 40 minutes, once a week; 5 weeks in total. |
Who may qualify
- Ages
- 5 to 10 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Classic/typical RTT
- Causing mutation in MECP2 gene
- Stable pattern of seizures, or has had no seizures at least 8 weeks
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Esketamine hydrochlorideIntravenous infusion of Ketamine | Drug | Esketamine treatment |
Primary outcome
Efficacy of treatment with Esketamine in girls with RTT
Time frame: 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
Time frame: baseline, week 5
Safety of treatment with Esketamine in girls with RTT
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; Minimum value 0; Maximum values 7; higher scores mean a worse outcome.
8 secondary outcomes
Autism Diagnostic Observation Schedule, Second Edition (ADOS-2)
Time frame: baseline, week 5, and month 6
Griffiths Scales of Child Development
Time frame: baseline, week 5, and month 6
Revised-Motor Behavior Assessment scale (R-MBA)
Time frame: baseline, week 5, and month 6
Rett Syndrome Severity Scale (RSSS)
Time frame: baseline, week 5, and month 6
Sleep improvement by Esketamine treatment
Time frame: baseline, week 5, and month 6
Behavior observation
Time frame: baseline, week 5, and month 6
Brain image change
Time frame: baseline, week 5, and month 6
Brain function change
Time frame: baseline, week 5, and month 6
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 May 2026.
- Individual participant dataSharing planned: Yes. all collected IPD, all IPD that underlie results in a publication
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Huiping Li principal investigator
Children's Hospital of Fudan University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Esketamine hydrochloride in people with Rett Syndrome, run by Children's Hospital of Fudan University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy of treatment with Esketamine in girls with RTT (time frame: baseline, week 5, and month 6). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 May 2026.