LAVENDER phase 3 clinical trial launched for trofinetide in Rett syndrome by Acadia Pharmaceuticals

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US biopharma company Acadia Pharmaceuticals has launched the LAVENDER phase 3 clinical trial to evaluate the efficacy and safety of trofinetide for the treatment of Rett syndrome in girls and young women.

Rett syndrome is considered to be a serious and rare neurodevelopmental congenital disorder related to the central nervous system (CNS). Symptoms of Rett syndrome are usually seen between six to 18 months of age, and result in problems with cognitive, motor, sensory, and autonomic function.

Jeffrey L. Neul – the investigator of the LAVENDER phase 3 clinical trial 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.”

LAVENDER phase 3 clinical trial launched for trofinetide in Rett syndrome

Acadia Pharmaceuticals launches LAVENDER phase 3 clinical trial launched for trofinetide in Rett syndrome. Image courtesy of suphakit73 at

The LAVENDER phase 3 clinical trial is a double-blind, randomized, placebo-controlled study to be held for 12 weeks. The late-stage clinical study will have see the participation of nearly 180 girls and young women 5 to 20 years of age having Rett syndrome.

According to Acadia Pharmaceuticals, about 90 Rett syndrome patients will be subjected to trofinetide, while the remaining will receive placebo. The co-primary efficacy endpoints of the LAVENDER phase 3 clinical trial will measure symptom improvement using the Rett Syndrome Behavior Questionnaire (RSBQ), a caregiver assessment, and the Clinical Global Impression Scale-Improvement (CGI-I), which is a clinician assessment.

Commenting on the LAVENDER phase 3 clinical trial, Serge Stankovic – President of Acadia Pharmaceuticals 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 3 LAVENDER study.”

The LAVENDER phase 3 clinical trial will be followed by a nine-month extension study called LILAC in which all the participants, including those on placebo, will be eligible for trofinetide treatment. All the LILAC participants will be followed up to assessed long-term tolerability, safety, and effectiveness of trofinetide.

Furthermore, a second extension study called LILAC-2 will follow in which eligible patients who completed the LILAC trial will continue to be administered with trofinetide.

As per Acadia Pharmaceuticals, trofinetide is a synthetic analog of the amino‐terminal tripeptide of IGF-1, which has been designed to treat the main symptoms of Rett syndrome by potentially bringing down neuroinflammation and supporting synaptic function.

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