Acadia 003 & 004

These studies investigate trofinetide in girls and women age 5-20 years with Rett syndrome to determine safety tolerability, to identify any side-effects, and to understand how the body processes the medication. Trofinetide has not been approved by the United States FDA for any disease or symptom, but previous clinical studies on adult and adolescent Rett syndrome patients indicated that the study drug is safe and well-tolerated, and also provided some evidence that clinical improvement of Rett syndrome may occur following treatment.

Acadia 003 is a randomized, double-blind, placebo-controlled 19 week study that will include 5 in-person clinic visits that will last approximately 3 hours, and 1 follow-up phone call.

If a patient completes Acadia 003, they have the option of enrolling into the Acadia 004 study, an open-label study where they will know they are receiving trofinetide. Participation in this study will last approximately 44 weeks and includes 5 in-person clinic visits, and 1 follow-up phone call.

Contact Person

Irys Caristo, CCRC


ACADIA Pharmaceuticals Inc.