NCT04776746Phase 3Terminated

Open-Label Extension Study of Trofinetide for 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 completion2023-06-30Actual

Enrollment · participants77Actual

Source updated2024-09-27Last 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: Percentage of Patients With Treatment-emergent Adverse Events (TEAEs), With Serious Adverse Events (SAEs), and With Withdrawals Due to AEs; description: Withdrawals due to AEs included both study drug discontinuation as well as study termination; timeFrame: Mean study drug exposure was 426 days, corresponding to 1.2 years; measure: Number (%) of Patients With Potentially Clinically Important Changes in ECG Post-baseline; description: Potentially clinically significant ECG changes were defined as QTcF \>500 ms or QTcF change from baseline (CFB) of \>60 ms; timeFrame: Mean study drug exposure was 426 days, corresponding to 1.2 years; measure: Number (%) of Patients With Potentially Clinically Important Changes in Vital Signs Post-baseline; description: Potentially clinically important changes from baseline in vital signs were defined 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 rate (PR) ≥120 bpm and increased ≥15 bpm from baseline; PR≤50 bpm and decreased ≥15 bpm from baseline. Baseline was the baseline value of previous study ACP-2566-003.; timeFrame: Mean study drug exposure was 426 days, corresponding to 1.2 years; measure: Number (%) of Patients With Potentially Clinically Important Changes in Body Weight Post-baseline; description: Potentially clinically important changes from baseline in body weight were defined as: Weight increase ≥7% from baseline Weight decrease ≥7% from baseline; timeFrame: Mean study drug exposure was 426 days, corresponding to 1.2 years; measure: Number (%) of Patients With Potentially Clinically Important Changes in Laboratory Parameters Post-baseline; description: Potentially clinically important changes in laboratory parameters were defined 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 \>2.75 mmol/L; blood urea nitrogen ≥10.71 mmol/L; creatinine \>1.5× upper limit of normal (ULN); uric acid ≥505.75 μmol/L; lactate dehydrogenase (LDH) ≥3×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 transaminase (ALT) ≥3×ULN; aspartate transaminase (AST) ≥3×ULN; gamma glutamyl transferase (GGT) ≥3×ULN; alkaline phosphatase (ALP) ≥3×ULN; bilirubin ≥1.5×ULN; timeFrame: Mean study drug exposure was 426 days, corresponding to 1.2 years

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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