There's a lot that's being measured. We'll see how much they include into the TLR...
Outcome Measure Measure Description Time Frame
RSBQ Change from baseline to End of Treatment (EOT) in the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score 12 weeks
Incidents of Adverse Events Change from baseline to End of Treatment (EOT) 12 weeks
Secondary Outcome Measures
Outcome Measure Measure Description Time Frame
CGI-I Change from baseline to End of Treatment (EOT) in the Clinical Global Impression Improvement Scale (CGI-I) score 12 weeks
Anxiety, Depression, and Mood Scale (ADAMS) Anxiety, Depression, and Mood Scale (ADAMS) 12 weeks
Motor Behavioral Assessment-7 dynamic pediatric items (MBA-Ped7) Motor Behavioral Assessment-7 dynamic pediatric items (MBA-Ped7) 12 weeks
Children's Sleep Habits Questionnaire (CSHQ) Children's Sleep Habits Questionnaire (CSHQ) 12 weeks
Seizure Frequency via seizure diary Seizure Frequency via seizure diary 12 weeks
Incidence of Adverse Events Incidence of Adverse Events 12 weeks
RSBQ Emotional Factor-Pediatric (subset of the RSBQ) RSBQ Emotional Factor-Pediatric (subset of the RSBQ) 12 weeks
Rett Syndrome Caregiver Inventory Assessment (RTT CIA) Rett Syndrome Caregiver Inventory Assessment (RTT CIA) 12 weeks
Child Health Questionnaire-Parent Form 50 (CHQ-PF50) Child Health Questionnaire-Parent Form 50 (CHQ-PF50) 12 weeks
Other Outcome Measures
Outcome Measure Measure Description Time Frame
Glutamate Plasma Concentration Glutamate Plasma Concentration 12 weeks
GABA Plasma Concentration GABA Plasma Concentration 12 weeks
Genetic variant SIGMAR1, COMT Genetic variant SIGMAR1, COMT 12 weeks
Maximum Plasma Concentration [Cmax] Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 12 weeks
Maximum Plasma Concentration [Cmax] relationship with RSBQ Number of participants with positive Maximum Plasma Concentration [Cmax] relationship with RSBQ 12 weeks
Other Amino Acid Plasma concentrations Other Amino Acid Plasma concentrations 12 weeks
Measure of gene DNA variants and gene RNA expressions Number of participants with active dose compared gene DNA variants and gene RNA expressions 12 weeks
