The Safety and Efficacy of ANAVEX2-73 in Patients with Rett Syndrome
Research type
Research Study
Full title
A Double-Blind, Randomized, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients with Rett Syndrome
IRAS ID
282560
Contact name
Paramala Santosh
Contact email
Sponsor organisation
Anavex Germany GmbH
Eudract number
2019-004321-25
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 8 months, 13 days
Research summary
This is a multi-national Phase 2 clinical study, involving approx. 84 patients with Rett syndrome (RTT) aged 5 years and above but less than 18 years of age, in which neither the participants nor the researchers know who is receiving a
particular treatment. Rett syndrome (RTT) is a rare inherited disorder, which affects the development of the nervous system. It has a dynamic and ultimately progressive clinical course.
Considering the severity and progressive course of neurobehavioral impairments in RTT, there is a critical unmet medical need for this population that requires an urgent and immediate attention.
Anavex Life Sciences Corp. is developing ANAVEX2-73 for the treatment of RTT. ANAVEX2-73 is able to restore nervous system function, thus providing potential relief to patients with RTT.
The study will take place at investigational sites in Australia, Canada and the UK. Study participants are allocated by chance to receive one of two treatments, namely, an oral solution of the study drug (ANAVEX2-73) or an inactive solution which looks identical to the study drug solution, but has no pharmacological effects i.e. “placebo. One third of participants will receive placebo and two thirds will receive the study drug. In this 12 week study dosages are adjusted until the desired clinical effect is achieved. The aim of the study is to investigate the safety of the study drug, how well it is tolerated and its effectiveness in relieving the symptoms of RTT. Following this 12 week period, participants may roll over into a voluntary 48 week study in which both the researchers and participants will know which treatment is
being allocated. This voluntary extension study will be extended further based on a favourable safety review by the Data and Safety Monitoring Board.REC name
London - City & East Research Ethics Committee
REC reference
21/LO/0501
Date of REC Opinion
1 Sep 2021
REC opinion
Further Information Favourable Opinion