Summary

for females ages 5-20 (full criteria)
at UCSD
study started
estimated completion

Description

Summary

To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome

Official Title

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome

Keywords

Rett Syndrome Syndrome Pharmaceutical Solutions Trofinetide Drug - Trofinetide

Eligibility

You can join if…

Open to females ages 5-20

  1. Female subjects 5 to 20 years of age, inclusive, at Screening
  2. Body weight ≥12 kg at Screening
  3. Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
  4. Has classic/typical Rett syndrome (RTT)
  5. Has a documented disease-causing mutation in the MECP2 gene
  6. Has a stable pattern of seizures, or has had no seizures, within 8 weeks of Screening
  7. 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.

You CAN'T join if...

  1. Has been treated with insulin within 12 weeks of Baseline
  2. 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
  3. Has a history of, or current, cerebrovascular disease or brain trauma
  4. Has significant, uncorrected visual or uncorrected hearing impairment
  5. Has a history of, or current, malignancy
  6. 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).

Locations

  • University of California, San Diego accepting new patients
    La Jolla California 92093 United States
  • Translational Gemomics Research Institute (TGen) accepting new patients
    Phoenix Arizona 85012 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
ACADIA Pharmaceuticals Inc.
ID
NCT04181723
Phase
Phase 3
Study Type
Interventional
Last Updated