In an online letter to the Rett community, Acadia announced these recent additions: Seattle Children’s Hospital; the University of North Carolina at Chapel Hill; the Kennedy Krieger Institute/ Johns Hopkins School of Medicine, in Baltimore, Maryland; and the Montefiore Medical Center, in New York.
The trial (NCT04181723) intends to enroll about 184 girls and young women, ages 5 to 20. More information on trial locations and contacts is available here. The trial’s expected completion is October.
Trofinetide, previously known as NNZ-2566, is a man-made version of glypromate, a protein fragment produced during the breakdown of insulin-like growth factor 1 in the brain.
Both proteins are required for a healthy development of the brain, with studies having shown that they help ease inflammation in the mature brain, have neuroprotective effects, and help respond to disease and stress.
In the LAVENDER study, participants are assigned randomly to either trofinetide or a placebo, given twice daily for 12 weeks. Trofinetide, a liquid solution treatment, can be swallowed or taken via a gastrostomy tube.
The study will assess the therapy’s safety and effectiveness at easing neurobehavioral symptoms, as assessed by caregivers using the Rett Syndrome Behaviour Questionnaire and by clinicians using the Clinical Global Impression Scale-Improvement.
After the 12-week treatment period, patients will have the opportunity to join the LILAC extension study. LILAC, which is open-label (meaning that all patients receive trofinetide), will assess the long-term safety, tolerability, and effectiveness of trofinetide over 40 weeks (about nine months).
In a Phase 2 trial (NCT01894958) in males with fragile X syndrome, trofinetide treatment with higher dose yielded encouraging results as it was well-tolerated and eased social avoidance, anxiety, hyperactivity, and other symptoms.
Acadia holds the exclusive rights to develop and commercialize trofinetide in North America, after signing an exclusive license from Neuren Pharmaceuticals, which retains all rights elsewhere.
The U.S. Food and Drug Administration (FDA) has granted trofinetide rare pediatric disease designation. This means that if the FDA ultimately approves trofinetide, Acadia may quality for a voucher redeemable for priority review of a regulatory application for a different product. Trofinetide also has received orphan drug designation from both the FDA and the European Medicines Agency, and fast track designation from the FDA as a potential treatment for Rett syndrome.
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