ANAVEX2-73-RS-003 is a Phase 2/3, double-blind, randomized, placebo-controlled dose escalation safety, tolerability and efficacy study in patients 5-17 years of age with RTT using endpoints including multiple clinical and exploratory molecular and biochemical measures.
This Phase 2/3 efficacy study is designed as a double-blind, randomized, placebo-controlled study. This is a 12-week placebo-controlled study of ANAVEX2-73 oral solution for the treatment of patients with RTT 5-17 years of age. A voluntary option will be offered for all patients who meet the exposure criteria for ANAVEX2-73 to continue a 48-week open label extension.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
92
Liquid oral solution
Liquid oral solution
The Children's Hospital at Westmead
Sydney, New South Wales, Australia
Queensland Children's Hospital
Brisbane, Queensland, Australia
Austin Health
Melbourne, Victoria, Australia
RSBQ
Change from baseline to End of Treatment (EOT) in the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score
Time frame: 12 weeks
Incidents of Adverse Events
Change from baseline to End of Treatment (EOT)
Time frame: 12 weeks
CGI-I
Change from baseline to End of Treatment (EOT) in the Clinical Global Impression Improvement Scale (CGI-I) score
Time frame: 12 weeks
Anxiety, Depression, and Mood Scale (ADAMS)
Anxiety, Depression, and Mood Scale (ADAMS)
Time frame: 12 weeks
Motor Behavioral Assessment-7 dynamic pediatric items (MBA-Ped7)
Motor Behavioral Assessment-7 dynamic pediatric items (MBA-Ped7)
Time frame: 12 weeks
Children's Sleep Habits Questionnaire (CSHQ)
Children's Sleep Habits Questionnaire (CSHQ)
Time frame: 12 weeks
Seizure Frequency via seizure diary
Seizure Frequency via seizure diary
Time frame: 12 weeks
Incidence of Adverse Events
Incidence of Adverse Events
Time frame: 12 weeks
RSBQ Emotional Factor-Pediatric (subset of the RSBQ)
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Alberta Children's Hospital
Calgary, Alberta, Canada
British Columbia Children's Hospital
Vancouver, British Columbia, Canada
Children's Hospital LHSC
London, Ontario, Canada
Holland Bloorview Kids Hospital
Toronto, Ontario, Canada
Royal Hospital for Children
Edinburgh, United Kingdom
Evelina London Children's Hospital
London, United Kingdom
King's College of London
London, United Kingdom
...and 2 more locations
RSBQ Emotional Factor-Pediatric (subset of the RSBQ)
Time frame: 12 weeks
Rett Syndrome Caregiver Inventory Assessment (RTT CIA)
Rett Syndrome Caregiver Inventory Assessment (RTT CIA)
Time frame: 12 weeks
Child Health Questionnaire-Parent Form 50 (CHQ-PF50)
Child Health Questionnaire-Parent Form 50 (CHQ-PF50)
Time frame: 12 weeks