To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls with Rett syndrome
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
15
Trofinetide solution of 10-30 mL based on subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
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
Safety and Tolerability of Treatment With Oral Trofinetide
Percentage of patients with treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), withdrawals due to AEs, potentially clinically important (PCI) changes in other safety assessments (laboratory values, vital signs, or ECGs, following protocol-defined PCI criteria)
Time frame: Mean study drug exposure 434 days, corresponding to 1.2 years
AUC0-12,ss (Area Under the Concentration-time Curve From Time 0 to 12 h at Steady State)
Area under the concentration-time curve from time 0 to 12 h at steady state as obtained from population pharmacokinetic (PK) modelling
Time frame: PK samples were taken predose and at Weeks 2, 4, 8, and 12
Cmax,ss (Maximum Observed Drug Concentration at Steady State)
Maximum observed drug concentration at steady state as obtained from population pharmacokinetic (PK) modelling
Time frame: PK samples were taken predose and at Weeks 2, 4, 8, and 12
Cmin,ss (Minimum Observed Drug Concentration at Steady State of Oral Trofinetide)
Minimum observed drug concentration at steady state of oral trofinetide as obtained from population pharmacokinetic (PK) modeling
Time frame: PK samples were taken predose and at Weeks 2, 4, 8, and 12
Tmax (Time of the Maximum Observed Drug Concentration at Steady State)
Time of the maximum observed drug concentration at steady state as obtained from population pharmacokinetic (PK) modelling
Time frame: PK samples were taken predose and at Weeks 2, 4, 8, and 12
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Gillette Children's Hospital
Saint Paul, Minnesota, United States
Washington University
St Louis, Missouri, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States