Phase 2 safety, tolerability and efficacy study is designed as a double-blind, randomized, placebo-controlled study. 7-week placebo-controlled study of ANAVEX2-73 oral solution for the treatment of patients with RTT 18 years or older. A voluntary option will be offered for all patients who meet the exposure criteria for ANAVEX2-73 to continue a 12-week open label extension.
This Phase 2 safety, tolerability and efficacy study is designed as a double-blind, randomized, placebo-controlled study. This is a 7-week placebo-controlled study of ANAVEX2-73 oral solution for the treatment of patients with RTT 18 years or older. A voluntary option will be offered for all patients who meet the exposure criteria for ANAVEX2-73 to continue a 12-week open label extension.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
31
Liquid oral solution
Liquid oral solution
UAB | The University of Alabama at Birmingham
Birmingham, Alabama, United States
UC Davis University of California - Davis MIND Institute
Sacramento, California, United States
Rush University Medical Center
Chicago, Illinois, United States
Incidence of Adverse Events
Incidence of Adverse Events
Time frame: 7 weeks
Maximum Plasma Concentration [Cmax] of ANAVEX2-73
PK of ANAVEX2-73 and metabolite
Time frame: 7 weeks
Area Under the Curve [AUC] of ANAVEX2-73
PK of ANAVEX2-73 and metabolite
Time frame: 7 weeks
Lipid panel
Significant laboratory findings
Time frame: 7 weeks
RSBQ
Change from baseline to End of Treatment (EOT) in the Rett Syndrome Behaviour Questionnaire (RSBQ). Total score and a pre-specified subset of the RSBQ
Time frame: 7 weeks
CGI-I
Change from baseline to End of Treatment (EOT) in the Clinical Global Impression Improvement Scale (CGI-I) score. Total score and a pre-specified subset of the CGI-I
Time frame: 7 weeks
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