ANAVEX2-73-RS-002 is a Phase 3, double-blind, randomized, placebo-controlled dose escalation safety, tolerability and efficacy study in patients 18 years and older with RTT using endpoints including multiple clinical and exploratory molecular and biochemical measures.
This Phase 3 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 to continue a 48-week open label extension.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
33
Liquid oral solution
Liquid oral solution
HammondCare
Greenwich, New South Wales, Australia
Mater Misericordiae Ltd
South Brisbane, Queensland, Australia
Royal Melbourne Hospital (RMH)
Melbourne, Victoria, Australia
The Alfred Hospital
Melbourne, Victoria, Australia
RSBQ
Drug exposure-dependent response of the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score
Time frame: 7 weeks
Incidence of Adverse Events
Incidence of Adverse Events
Time frame: 7 weeks
CGI-I
Drug exposure-dependent response of the Clinical Global Impression of Improvement Scale (CGI-I) score
Time frame: 7 weeks
Anxiety, Depression, and Mood Scale (ADAMS)
Drug exposure-dependent response of the Anxiety, Depression, and Mood Scale (ADAMS)
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
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
The Keogh Institute for Medical Research
Nedlands, Western Australia, Australia
King's College of London
London, UK, United Kingdom
Manchester CGM, St. Mary's Hospital
Manchester, UK, United Kingdom