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Acadia Pharmaceuticals has initiated the Phase III LAVENDER placebo-controlled study of trofinetide to treat girls and young women with Rett syndrome.

The double-blind, randomised, placebo-controlled LAVENDER Phase III trial will evaluate the efficacy and safety of trofinetide in about 180 girls and young women aged five to 20 years.

Half of the subjects in the study will receive the investigational drug and half will receive placebo for 12 weeks.

As part of the study’s co-primary efficacy endpoints, symptom improvement will be measured with the help of the Rett Syndrome Behavior Questionnaire (RSBQ), a caregiver assessment, and the Clinical Global Impression Scale-Improvement (CGI-I).

Acadia Pharmaceuticals president Serge Stankovic said: “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.

“We are grateful to study participants and their families, investigators,, 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 III LAVENDER study.”

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The study will be followed by a nine-month extension trial called LILAC. All participants in this study will receive trofinetide.

They will be followed to evaluate the long-term tolerability, safety and effectiveness of the drug.

The second extension study, LILAC-2, will then be conducted, with eligible patients who complete the LILAC trial continuing to receive trofinetide.

The drug potentially reduces neuroinflammation and supports synaptic function to treat the core symptoms of Rett syndrome, a serious and rare neurodevelopmental congenital CNS disorder.