NCT04988867Phase 2, phase 3Terminated

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

Study sponsor: ACADIA Pharmaceuticals Inc. · ACAD

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

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Primary completion2023-05-31Actual

Enrollment · participants15Actual

Source updated2024-09-24Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

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Study details

Conditions studied

Rett Syndrome

Treatments or interventions studied

TrofinetideDrug

Primary outcomes · main study measures

Read the study measures

measure: Safety and Tolerability of Treatment With Oral Trofinetide; description: 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); timeFrame: Mean study drug exposure 434 days, corresponding to 1.2 years; measure: AUC0-12,ss (Area Under the Concentration-time Curve From Time 0 to 12 h at Steady State); description: Area under the concentration-time curve from time 0 to 12 h at steady state as obtained from population pharmacokinetic (PK) modelling; timeFrame: PK samples were taken predose and at Weeks 2, 4, 8, and 12; measure: Cmax,ss (Maximum Observed Drug Concentration at Steady State); description: Maximum observed drug concentration at steady state as obtained from population pharmacokinetic (PK) modelling; timeFrame: PK samples were taken predose and at Weeks 2, 4, 8, and 12; measure: Cmin,ss (Minimum Observed Drug Concentration at Steady State of Oral Trofinetide); description: Minimum observed drug concentration at steady state of oral trofinetide as obtained from population pharmacokinetic (PK) modeling; timeFrame: PK samples were taken predose and at Weeks 2, 4, 8, and 12; measure: Tmax (Time of the Maximum Observed Drug Concentration at Steady State); description: Time of the maximum observed drug concentration at steady state as obtained from population pharmacokinetic (PK) modelling; timeFrame: PK samples were taken predose and at Weeks 2, 4, 8, and 12

History coverage

Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.

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