Trofinetide
- Generic Name
- Trofinetide
- Brand Names
- Daybue
- Drug Type
- Small Molecule
- Chemical Formula
- C13H21N3O6
- CAS Number
- 853400-76-7
- Unique Ingredient Identifier
- Z2ME8F52QL
- Background
Trofinetide is a novel synthetic analog of glypromate, also known as glycine–proline–glutamate (GPE), a naturally occurring protein in the brain and the N-terminal tripeptide of insulin-like growth factor 1 (IGF-1). Trofinetide was approved by the FDA on March 10, 2023, for the treatment of Rett syndrome, which is an X-linked neurodevelopmental disorder char...
- Indication
Trofinetide is indicated for the treatment of Rett syndrome in adults and pediatric patients two years of age and older.
- Associated Conditions
- Rett Syndrome
- Associated Therapies
- -
An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome
- First Posted Date
- 2021-08-04
- Last Posted Date
- 2023-06-22
- Lead Sponsor
- ACADIA Pharmaceuticals Inc.
- Target Recruit Count
- 15
- Registration Number
- NCT04988867
- Locations
- 🇺🇸
Gillette Children's Hospital, Saint Paul, Minnesota, United States
🇺🇸Children's Hospital Colorado, Aurora, Colorado, United States
🇺🇸Vanderbilt University Medical Center, Nashville, Tennessee, United States
Open-Label Extension Study of Trofinetide for Rett Syndrome
- First Posted Date
- 2021-03-02
- Last Posted Date
- 2023-08-01
- Lead Sponsor
- ACADIA Pharmaceuticals Inc.
- Target Recruit Count
- 78
- Registration Number
- NCT04776746
- Locations
- 🇺🇸
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
🇺🇸UC Davis MIND Institute, Sacramento, California, United States
🇺🇸Rush University Medical Center, Chicago, Illinois, United States
Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
- First Posted Date
- 2020-02-21
- Last Posted Date
- 2024-04-11
- Lead Sponsor
- ACADIA Pharmaceuticals Inc.
- Target Recruit Count
- 154
- Registration Number
- NCT04279314
- Locations
- 🇺🇸
Emory Genetics Clinical Trial Center, Atlanta, Georgia, United States
🇺🇸Gillette Children's Specialty Healthcare, Saint Paul, Minnesota, United States
🇺🇸Kennedy Krieger Institute - Clinical Trials Unit, Baltimore, Maryland, United States
Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
- First Posted Date
- 2019-11-29
- Last Posted Date
- 2024-04-08
- Lead Sponsor
- ACADIA Pharmaceuticals Inc.
- Target Recruit Count
- 187
- Registration Number
- NCT04181723
- Locations
- 🇺🇸
Kennedy Krieger Institute - Clinical Trials Unit, Baltimore, Maryland, United States
🇺🇸Rush University Children's Hospital, Chicago, Illinois, United States
🇺🇸Boston Children's Hospital Harvard Medical School, Boston, Massachusetts, United States
Drug Development Updates
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