The Safety and Efficacy of ANAVEX2-73 in Patients with Rett Syndrome
Research type
Research Study
Full title
A Double-Blind, Randomised, Placebo-Controlled, Safety and Efficacy Study of ANAVEX2-73 in Patients with Rett Syndrome
IRAS ID
270499
Contact name
Christopher Missling
Contact email
Sponsor organisation
Anavex Germany GmbH
Eudract number
2019-004345-32
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 6 months, 0 days
Research summary
A double-blind, placebo-controlled study to assess the safety and efficacy of ANAVEX2-73 in patients with Rett (RTT) Syndrome. RTT is a progressive neurodevelopmental disorder occurring predominantly in females. RTT patients develop normally until 6 to 18 months and then regress in physical, cognitive, and neurological functions. Patients lose expressive language skills, motor skills and develop stereotypic hand movements. Multiple issues develop, including microcephaly, seizures, anxiety, ataxia, hyperventilation and gastrointestinal dysfunction.
ANAVEX2-73 is an oral sigma-1 receptor agonist that restores cellular homeostasis by targeting protein misfolding, oxidative stress, mitochondrial dysfunction, inflammation and cellular stress. It is an effective neuroprotective, anticonvulsive and anti-depressive agent. Assessments of cognition, behaviour, motor function, seizures, respiratory and other autonomic dysfunction will be used to measure the safety, tolerability and efficacy of ANAVEX2-73 over a 7 week period (followed by a 48 week voluntary extension) in female RTT patients aged 18 or over. Therapeutic benefit will be measured as improvement in behavioral and physiological activity. Study sites include a Neuroscience site and a Genomic Medicine site in the UK and a Hospital Neurology Department in Ireland. Anavex Life Sciences Corp will fund the research and exploratory efficacy endpoints will be measured as:
RSBQ Emotional Factor-Adult
Anxiety, Depression, and Mood Scale (ADAMS)
Children’s Sleep Habits Questionnaire (CSHQ)
Visual Analog Scale (VAS) of the top three concerns a family has for their daughter
Rett syndrome Caregiver Inventory Assessment (RTT CIA)
Child Health Questionnaire-Parent Form 50 (CHQ-PF50)
Seizure Frequency via seizure diary.
A Clinical Global Impression Scale will be used to assess severity improvement of the Rett condition.
This current study, which measures the safety and efficacy of ANAVEX2-73, has already been started at 2 clinical sites in Australia.
In the future, Anavex Life Sciences would also like to assess the possible therapeutic benefit of ANAVEX2-73 in paediatric RTT Patients.REC name
London - City & East Research Ethics Committee
REC reference
20/LO/0297
Date of REC Opinion
26 May 2020
REC opinion
Further Information Favourable Opinion