Rett syndrome is a serious and rare neurodevelopmental congenital CNS disorder with symptoms that typically present between six to 18 months of age, and lead to problems with cognitive, sensory, motor, and autonomic function.
“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,” said Jeffrey L. Neul, M.D., Ph.D., Annette Schaffer Eskind Chair and Director, Vanderbilt Kennedy Center; Professor of Pediatrics, Division of Neurology, Pharmacology, and Special Education, Vanderbilt Kennedy Center, Vanderbilt University Medical Center and LAVENDER study investigator. “I look forward to the outcomes of this clinical program evaluating trofinetide as a potentially new treatment for Rett syndrome.”
The LAVENDER Phase 3 study is a 12-week, double-blind, randomized, placebo-controlled study evaluating the efficacy and safety of trofinetide in approximately 180 girls and young women 5 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 Behavior Questionnaire (RSBQ), a caregiver assessment, and the Clinical Global Impression Scale-Improvement (CGI-I), a clinician assessment.
“For patients living with this debilitating disease, and the families whose dedication to their care inspires us, the LAVENDER study is an important next step in what we hope will result in the first FDA-approved treatment for Rett syndrome,” said Serge Stankovic, M.D., M.S.P.H., ACADIA’s President. “We are grateful to study participants and their families, investigators, Rettsyndrome.org, and Neuren Pharmaceuticals who have played instrumental roles in advancing trofinetide to this stage of clinical development and look forward to building upon this work to further evaluate trofinetide in the Phase 3 LAVENDER study.”
The LAVENDER study will be followed by LILAC, a nine-month extension study in which all participants, including those on placebo in the LAVENDER study, will be eligible to receive trofinetide. All LILAC participants will be followed to evaluate long-term tolerability, safety, and effectiveness of trofinetide. A second extension study, LILAC-2, will follow in which eligible patients who complete the LILAC trial will continue to receive trofinetide.
“The start of the trofinetide study has been highly anticipated by the Rett community, which currently has no approved treatment for Rett syndrome,” said Melissa Kennedy, Executive Director of RettSyndrome.org (RSO). “We are hopeful for what this study means for patients and their families as it potentially brings us closer to improving the lives of many living with Rett syndrome.”
In 2018, ACADIA entered into an exclusive North American license agreement with Neuren for the development and commercialization of trofinetide for Rett syndrome and other indications. In an end-of-Phase 2 meeting, the FDA confirmed that positive results from a pivotal Phase 3 study for trofinetide in Rett syndrome and the extension study could be the basis of a New Drug Application (NDA) submission.
Source: Company Press Release