NCT04279314Phase 3Completed

Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With 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.

Open ClinicalTrials.gov record CSV export · Pro

Primary completion2022-08-19Actual

Enrollment · participants154Actual

Source updated2024-04-11Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

Pro unlocks the full available history and CSV export. View Pro features

No recorded changes are available for this view and time period. Our history may be incomplete; this does not establish that the trial stayed unchanged.

Study details

Conditions studied

Rett Syndrome

Treatments or interventions studied

TrofinetideDrug

Primary outcomes · main study measures

Read the study measures

measure: Percentage of Subjects With Treatment-emergent Adverse Events (TEAEs), Percentage of Subjects With Serious Adverse Events (SAEs), and Percentage of Subjects With Withdrawals Due to AEs; description: Percentage of subjects with treatment-emergent adverse events (TEAEs), percentage of subjects with serious adverse events (SAEs), and percentage of subjects with withdrawals due to AEs; timeFrame: 40 Weeks Treatment Duration; measure: Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in ECG; description: Potentially clinically important ECG changes were defined in the study protocol as absolute QTcF interval \>500 ms or QTcF interval change from the baseline value of previous study ACP-2566-003 of \>60 ms; timeFrame: 40 Weeks Treatment Duration; measure: Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs; description: Potentially clinically important changes in vital signs were defined in the study protocol as: systolic blood pressure (SBP) ≥180 mmHg and increased ≥20 mmHg from baseline; SBP ≤90 mmHg and decreased ≥20 mmHg from baseline; diastolic blood pressure (DBP) ≥ 105 mmHg and increased ≥15 mmHg from baseline; DBP ≤50 mmHg and decreased ≥15 mmHg from baseline; Pulse ≥120 bpm and increased ≥15 bpm from baseline; Pulse ≤50 bpm and decreased ≥15 bpm from baseline; timeFrame: 40 Weeks Treatment Duration; measure: Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Body Weight; description: Potentially clinically important changes in body weight were defined in the study protocol as: Weight increase ≥7% from baseline; Weight decrease ≥7% from baseline; timeFrame: 40 Weeks Treatment Duration; measure: Subjects (N, %) With Post-baseline Potentially Clinically Important Changes; description: Potentially clinically important changes in laboratory parameters were defined in the study protocol as: Sodium ≤125 mmol/L; Sodium ≥155 mmol/L; Potassium ≤3.0 mmol/L; Potassium ≥5.5 mmol/L; Chloride ≤85 mmol/L; Chloride ≥120 mmol/L; Calcium \<2.0 mmol/L; Calcium \>2.0 mmol/L; Blood urea nitrogen ≥10.71 mmol/L; Creatinine \>1.5 x upper limit of normal (ULN); Uric acid ≥505.75 μmol/L; Lactate dehydrogenase ≥3 x ULN; Glucose ≤2.48 mmol/L; Glucose ≥11 mmol/L; Albumin ≤26 g/L; Albumin ≥60 g/L; Protein ≤50 g/L; Protein ≥100 g/L; Alanine aminotransferase ≥3 x ULN; Aspartate aminotransferase ≥3 x ULN; Gamma glutamyl transpeptidase ≥3 x ULN; Alkaline phosphatase ≥3 x ULN; Bilirubin ≥1.5 x ULN; timeFrame: 40 Weeks Treatment Duration

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.

How to read this history