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Plain language summary
This is a summary of an article about the LAVENDER study, which was published in Nature Medicine in June 2023. The study involved girls and young women with a rare genetic condition called Rett syndrome, which affects the way the brain develops. Researchers wanted to find out if a drug called trofinetide could improve the symptoms of Rett syndrome.
What happened during the LAVENDER study?
A total of 187 girls and young women took trofinetide (brand name DAYBUE™) liquid or a placebo (something that looks the same as the trofinetide liquid but does not contain medicine) 2 times a day either by drinking it or through a tube into the stomach called a gastrostomy tube (g-tube). Changes in Rett syndrome symptoms were looked for by study doctors for 12 weeks by using a rating scale called the Clinical Global Impression–Improvement (CGI–I) and by caregivers (usually a parent) who completed a questionnaire called the Rett Syndrome Behaviour Questionnaire (RSBQ). Caregivers were also asked to rate communication and social interaction skills, which resulted in the ‘Social Composite’ score on a questionnaire called the Communication and Symbolic Behavior Scales Developmental Profile™ Infant–Toddler (CSBS-DP-IT) checklist.
What were the results?
After 12 weeks of treatment, girls and young women who received trofinetide had greater improvements in their symptoms than those who took the placebo, as rated by the RSBQ and CGI–I. Participants who took trofinetide could communicate better than participants who took the placebo as rated on the CSBS–DP–IT Social Composite scale. The differences between trofinetide and the placebo were statistically significant, which means that it was unlikely that the benefit seen with trofinetide was caused by chance. The most common side effects in the trofinetide group were diarrhea (frequent watery bowel movements) and vomiting, and in almost all cases these were mild or moderate.
What do the results of the study mean?
In the LAVENDER study, trofinetide helped girls and young women with Rett syndrome by improving several important symptoms. This is the first study to show that a medicine, trofinetide, improves the symptoms of Rett syndrome.
Clinical Trial Registration: NCT04181723 (LAVENDER) (ClinicalTrials.gov)
Acknowledgments
The authors would like to thank the participants of the LAVENDER study and their family members and caregivers. The authors would also like to thank the study site staff who cared for and supported the study participants.
Financial & competing interests disclosure
JL Neul has received research funding from the International Rett Syndrome Foundation, the National Institutes of Health and Rett Syndrome Research Trust; has received personal consultancy fees from Acadia Pharmaceuticals Inc., Analysis Group, AveXis, GW Pharmaceuticals, Hoffmann-La Roche, Myrtelle, Neurogene Inc., Newron Pharmaceuticals, Signant Health and Taysha Gene Therapies, and for the preparation of CME activities for Medscape and PeerView Institute; serves on the scientific advisory board of Alcyone Lifesciences; is a scientific cofounder of LizarBio Therapeutics; and was a member of a data and safety monitoring board for clinical trials conducted by Ovid Therapeutics. AK Percy is coeditor of Translational Science of Rare Diseases, received research funding from the National Institutes of Health, and is a consultant for Acadia Pharmaceuticals Inc., AveXis, GW Pharmaceuticals and Anavex Life Sciences Corp., as well as adviser to the International Rett Syndrome Foundation. TA Benke reports receiving research funding from GRIN2B Foundation, the International Foundation for CDKL5 Research, Loulou Foundation, the National Institutes of Health and Simons Foundation; consultancy fees from Alcyone, AveXis, GRIN Therapeutics, GW Pharmaceuticals, the International Rett Syndrome Foundation, Marinus Pharmaceuticals, Neurogene, Ovid Therapeutics and Takeda Pharmaceutical Company Limited; clinical studies with Acadia Pharmaceuticals Inc., GW Pharmaceuticals, Marinus Pharmaceuticals, Ovid Therapeutics and Rett Syndrome Research Trust; all remuneration has been made to his department. EM Berry-Kravis reports receiving funding from Acadia Pharmaceuticals Inc., Alcobra Pharmaceuticals, AMO Pharma, Asuragen, AveXis, Biogen, BioMarin, Cydan Development, EryDel, Fulcrum Therapeutics, GeneTx, GW Pharmaceuticals, Ionis Pharmaceuticals, Jaguar Health, Lumos Pharma, Marinus Pharmaceuticals, Neuren Pharmaceuticals, Neurogene, Neurotrope, Novartis, Orphazyme, Ovid Therapeutics, Retrophin, Roche, Seaside Therapeutics, Taysha Gene Therapies, Tetra Bio-Pharma, Ultragenyx, Yamo Pharmaceuticals, Zynerba Pharmaceuticals and Vtesse/Sucampo/Mallinckrodt Pharmaceuticals to consult on study design or run clinical or lab validation studies in genetic neurodevelopmental or neurodegenerative disorders, all of which is directed to Rush University Medical Center in support of rare disease programs; EM Berry-Kravis receives no personal funds, and Rush University Medical Center has no relevant financial interest in any of the commercial entities listed. DG Glaze has received personal compensation and research support from Acadia Pharmaceuticals Inc., Neuren Pharmaceuticals and Newron Pharmaceuticals. V Abler, T Lin, KM Bishop and JM Youakim are employees of Acadia Pharmaceuticals Inc. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
Medical writing and editorial assistance in the development of this article were provided by Stuart Murray, MSc, CMPP, of Evidence Scientific Solutions, Inc., and funded by Acadia Pharmaceuticals Inc.
