Phase 2
N=23
A Study to Evaluate Ketamine for the Treatment of Rett Syndrome
Rett Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT03633058 ↗Enrolled (actual)
23
Serious AEs
0.0%
Results posted
Mar 2024
Primary outcome: Primary: Dose-Limiting Adverse Events — 2; 4; 6; 3 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Ketamine (Drug)
- Age
- Pediatric · 6+ yrs
- Sex
- Female
- Sponsor
- Rett Syndrome Research Trust
- Primary completion
- Nov 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Dose-Limiting Adverse Events |
2; 4; 6; 3; 0; 0 | — |
Summary
This 2 cohort, sequential, ascending dose study will assess the safety, tolerability and efficacy of oral ketamine dosed in a single 5-day BID regimen in addition to placebo, in a 4-week cross-over design in patients with Rett Syndrome. Approximately 12 patients per cohort are anticipated to participate for approximately 8-10 weeks at approximately 7 US study centers.
Eligibility Criteria
Inclusion Criteria
- Female Rett Syndrome patients diagnosed with Rett Syndrome with a confirmed MECP2 mutation
- between the ages of 6 and 12, inclusive, who have not achieved menarche
- ability to take oral medications
- are generally healthy.
Exclusion Criteria
- Patients not on stable medication regimens/other types of behavioral, educational, or dietary interventions for at least 4 weeks,
- are taking medications that may interact with ketamine,
- have a condition where increased blood pressure, spinal fluid pressure, or ocular pressure may put the patient at increased risk.
Data sourced from ClinicalTrials.gov (NCT03633058). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.