To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome
Female subjects 5 to 20 years of age, inclusive, at Screening
Body weight ≥12 kg at Screening
Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
Has classic/typical Rett syndrome (RTT)
Has a documented disease-causing mutation in the MECP2 gene
Has a stable pattern of seizures, or has had no seizures, within 8 weeks of Screening
Subjects of childbearing potential must abstain from sexual activity for the duration of the study and for at least 30 days thereafter or must agree to use acceptable methods of contraception. Subject must not be pregnant or breastfeeding.
The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
Subject and caregiver(s) must reside at a location to which study drug can be delivered and have been at their present residence for at least 3 months prior to Screening
Has been treated with insulin within 12 weeks of Baseline
Has current clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, Type 1 diabetes mellitus, or uncontrolled Type 2 diabetes mellitus), renal, hepatic, respiratory or gastrointestinal disease (such as celiac disease or inflammatory bowel disease) or has major surgery planned during the study
Has a history of, or current, cerebrovascular disease or brain trauma
Has significant, uncorrected visual or uncorrected hearing impairment
Has a history of, or current, malignancy
Has a known history or symptoms of long QT syndrome
Additional inclusion/exclusion criteria apply. Patients will be evaluated at screening to ensure that all criteria for study participation are met. Patients may be excluded from the study based on these assessments (and specifically, if it is determined that their baseline health and condition do not meet all pre-specified entry criteria).
November 2, 2022
Primary Contact Information
For more information on this trial, visit clinicaltrials.gov.
For more information and to contact the study team: