Phase 3 Pivotal Study of Trofinetide for Rett Syndrome Initiated

The phase 3 LAVENDER placebo-controlled study to evaluate efficacy and safety of trofinetide for treatment of Rett syndrome has been initiated. Presenting primarily in girls and young women at age 6 to 18 months, individuals with Rett syndrome that causes cognitive, sensory, motor, and autonomic function. These children also have seizures, disorganized breathing patterns, sleep disturbances, and scoliosis. Clinical decline is rapid, and disability is high as children lose the ability to perform activities of daily living. 

Approximately 180 girls and women, age 5 to 20 years with Rett syndrome, will be enrolled and randomly assigned to receive trofinetide or placebo on a 1:1 basis for 12 weeks. Outcome measures will include symptom improvement using the Rett Syndrome Behavior Questionnaire (RSBQ), a caregiver assessment, and the Clinical Global Impression Scale-Improvement (CGI-I), a clinician assessment. All participants will be eligible to switch to or continue receiving trofinatide in open-label extension studies.

“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, MD, MSPH, 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 start of the trofinetide study has been highly anticipated by the Rett community, who 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.”

Trofinetide is a novel synthetic analog of the amino‐terminal tripeptide of insulin-like growth factor 1 (IGF-1) with potential to reduce inflammation and support neuronal survival.