LAVENDER Trial Enrollment Complete

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by Marisa Wexler MS |

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Acadia Pharmaceuticals has completed enrollment in the Phase 3 clinical trial LAVENDER, which is testing the investigational medication trofinetide as a treatment for Rett syndrome.

Top-line results from the study are expected later this year, according to a press release.

LAVENDER (NCT04181723) enrolled approximately 180 female Rett syndrome patients ages 5 to 20. Participants are assigned randomly to either trofinetide or a placebo, given twice daily for 12 weeks.

As primary goals, the trial will evaluate the therapy’s safety, as well as its effectiveness at normalizing behaviors, as assessed both by caregivers (using the Rett Syndrome Behaviour Questionnaire) and by clinicians, with the Clinical Global Impression Scale-Improvement.

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After the 12-week treatment period, participants are allowed to join an open-label extension study called LILAC, which is testing the long-term safety, tolerability, and effectiveness of trofinetide over 40 weeks (about nine months). Being open-label means that all patients receive trofinetide. After LILAC, patients may join the LILAC-2 study to further evaluate trofinetide’s safety.


Trofinetide, previously known as NNZ-2566, is a man-made version of glypromate, a protein fragment that has been shown to protect neurons (nerve cells) from damage in the mature brain, and help respond to disease and cellular stress.

Trofinetide is a liquid solution treatment that can be swallowed or administered via a gastrostomy tube. The U.S. Food and Drug Administration has given the investigational medication fast-track, orphan drug, and rare pediatric disease designations.

In a previous Phase 2 clinical trial (NCT01894958) conducted in males with fragile X syndrome, treatment with a high dose of trofinetide was well-tolerated and eased symptoms including social avoidance, anxiety, and hyperactivity.

The LAVENDER clinical trial is sponsored by Acadia, which holds the exclusive rights to develop and commercialize trofinetide in North America through an agreement with Neuren Pharmaceuticals, which retains all rights to the medication elsewhere in the world.

“This successful completion of enrolment is a very important milestone for Neuren and we are now excited to await results from the Phase 3 trial before the end of 2021,” said Jon Pilcher, CEO of Neuren.

“Given the many challenges presented by the pandemic over the last 18 months, this is a great achievement by Acadia and reflects the remarkable commitment and determination of the Rett syndrome community in the United States,” Pilcher added.