An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome

Stopped early · Phase 2/Phase 3

Conditions studied: Rett Syndrome

In brief

To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls with Rett syndrome

Key facts

Study ID

NCT04988867

Run by

ACADIA Pharmaceuticals Inc.

People needed

15

Starts

2021-09-22

Expected to finish

2023-05-31

Last updated by the study team

2024-09-24

Who can join

Age: 2 and older, up to 5. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• Female subject
• 2 to 4 years of age and body weight ≥9 kg and <20 kg at Screening OR
• 5 years of age and body weight ≥9 kg and <12 kg at Screening
• Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
• The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
• Has classic/typical Rett syndrome (RTT) or possible RTT according to the Rett Syndrome Diagnostic Criteria
• Has a documented disease-causing mutation in the MECP2 gene
• Has a stable pattern of seizures, or has had no seizures, within 8 weeks prior to Screening
• 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 4 weeks prior to Screening

You may not qualify if…

• 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 any of the following:
• QTcF interval of >450 ms at Screening or Baseline
• History of a risk factor for torsades de pointes (e.g., heart failure or family history of long QT syndrome)
• History of clinically significant QT prolongation that is deemed to put the subject at increased risk of clinically significant QT prolongation
• Other clinically significant finding on ECG at Screening or Baseline
• Additional inclusion/exclusion criteria apply. Patients will be evaluated at baseline 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 prespecified entry criteria).

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• Rush University Medical Center — Chicago, Illinois, United States
• Boston Children's Hospital/Harvard Medical School — Boston, Massachusetts, United States
• Gillette Children's Hospital — Saint Paul, Minnesota, United States
• Washington University — St Louis, Missouri, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States

Full record on ClinicalTrials.gov

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