ACADIA Pharmaceuticals, a biopharmaceutical company focused on the development and commercialisation of innovative medicines to address unmet medical needs in central nervous system disorders, has started the phase three LAVENDER placebo-controlled study to assess the efficacy and safety of Trofinetide intended for the treatment of girls and young women with Rett syndrome, it was reported yesterday.

The LAVENDER Phase three study is a 12-week, double-blind, randomised, placebo-controlled study assessing the efficacy and safety of trofinetide in around 180 girls and young women five to 20 years of age with Rett syndrome. Half of study participants will receive trofinetide and half will receive placebo. Co-primary efficacy endpoints of the study will measure symptom improvement using the Rett Syndrome Behaviour Questionnaire, a caregiver assessment, and the Clinical Global Impression Scale-Improvement, a clinician assessment.

Jeffrey L Neul, MD, PhD, Annette Schaffer Eskind chair and director, Vanderbilt Kennedy Center; professor of Paediatrics, Division of Neurology, Pharmacology, and Special Education, Vanderbilt Kennedy Center, Vanderbilt University Medical Center and LAVENDER study investigator, said, 'There is no approved treatment for Rett syndrome, which is a rare neurological disease that impacts nearly every aspect of a child's life, resulting in loss of speech, difficulty breathing, lack of motor control, loss of muscle tone and mobility, seizures, and more. I look forward to the outcomes of this clinical program evaluating trofinetide as a potentially new treatment for Rett syndrome.'