Phase 2/3 Trial of Anavex 2-73 Enrolling Girls in Canada, Australia
The Phase 2/3 study (NCT04304482), called EXCELLENCE, will run for 12 weeks with patients randomly assigned in a 2:3 ratio to either Anavex 2-73 or a placebo. Those eligible must have a confirmed Rett diagnosis, and be at least six months out from their last loss of language or fine or gross motor skills.
Anavex 2-73 is a liquid, oral medication that has been shown to activate a protein called the sigma-1 receptor, which is known to help proteins fold properly. By activating this receptor, Anavex 2-73 has the potential to reduce protein misfolding within cells and prevent some of the neuroinflammation and neurological (emotional, sensory, motor, and cognitive) problems seen in people with Rett.
The disease almost exclusively affects females.
Results of a Phase 2 trial of Anavex 2-73 in 31 women in the U.S. (NCT03758924) showed that about two-thirds of treated patients (ages 18 to 45) had a significant reduction in several characteristic Rett behaviors, including general mood, breathing, hand behavior, and repetitive face movements.
The EXCELLENCE trial’s main goals are changes in characteristic behaviors from the study’s start (baseline) through 12 weeks, as assessed using the Rett Syndrome Behavior Questionnaire, in addition to treatment safety.
Secondary trial goals will include changes over the study’s course in measures of anxiety, depression and mood; in sleep habits and seizure frequency; and in motor behavior. Caregivers’ views of treatment effectiveness will also be assessed.
The trial requires participants to complete four in-person visits, and four checkups done by phone. Travel expenses related to in-person visits will be reimbursed by the study, the release stated.
Study visits will include collection of blood and urine samples, electrocardiograms to measure the electrical activity of the heart, physical exams, and questionnaires to be completed by the patients’ caregivers.
Patients who complete the main trial will be offered the option of entering its 48-week open label extension (OLE), in which all will be given Anavex 2-73 (no placebo group).
The three Australian sites are at centers in Sydney, Brisbane, and Melbourne.
EXCELLENCE is due to conclude in June 2022.