CompletedPhase 3

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

United States154 participantsStarted 2020-01-29

Plain-language summary

To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls and women with Rett syndrome

Who can participate

Age range5 Years – 21 Years

SexFEMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Has completed the Week 12/End-of-treatment visit of the antecedent study, Study ACP-2566-003
✓. Met all entry criteria for the antecedent study
✓. May benefit from long-term treatment with open-label trofinetide in the judgment of the Investigator
✓. Can still swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
✓. The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
✓. Subject and caregiver(s) must reside at a location to which study drug can be delivered and have been at their present residence for at least 3 months prior to Baseline

Exclusion criteria

✕. Began treatment with growth hormone during the antecedent study
✕. Began treatment with IGF-1 during the antecedent study
✕. Began treatment with insulin during the antecedent study
✕. Has developed a 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
✕. Subject is judged by the Investigator or the Medical Monitor to be inappropriate for the study due to AEs, medical condition, or noncompliance with investigational product or study procedures in the antecedent study
✕. Has a clinically significant abnormality in vital signs at Baseline
✕. Has a QTcF interval of \>450 ms on the Baseline ECG performed before the first dose of trofinetide is given in the present study
✕. Has developed a clinically significant ECG finding during the antecedent study

What they're measuring

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

Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in ECG

Timeframe: 40 Weeks Treatment Duration

Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs

Timeframe: 40 Weeks Treatment Duration

Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Body Weight

Timeframe: 40 Weeks Treatment Duration

Subjects (N, %) With Post-baseline Potentially Clinically Important Changes

Timeframe: 40 Weeks Treatment Duration

Trial details

NCT IDNCT04279314

SponsorACADIA Pharmaceuticals Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-08-19

Results submitted2024-03-14

View on ClinicalTrials.gov More Rett Syndrome trials

Who can participate

Age range5 Years – 21 Years

SexFEMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Has completed the Week 12/End-of-treatment visit of the antecedent study, Study ACP-2566-003
✓. Met all entry criteria for the antecedent study
✓. May benefit from long-term treatment with open-label trofinetide in the judgment of the Investigator
✓. Can still swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
✓. The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
✓. Subject and caregiver(s) must reside at a location to which study drug can be delivered and have been at their present residence for at least 3 months prior to Baseline

Exclusion criteria

✕. Began treatment with growth hormone during the antecedent study
✕. Began treatment with IGF-1 during the antecedent study
✕. Began treatment with insulin during the antecedent study
✕. Has developed a 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
✕. Subject is judged by the Investigator or the Medical Monitor to be inappropriate for the study due to AEs, medical condition, or noncompliance with investigational product or study procedures in the antecedent study
✕. Has a clinically significant abnormality in vital signs at Baseline
✕. Has a QTcF interval of \>450 ms on the Baseline ECG performed before the first dose of trofinetide is given in the present study
✕. Has developed a clinically significant ECG finding during the antecedent study

What they're measuring

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

Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in ECG

Timeframe: 40 Weeks Treatment Duration

Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs

Timeframe: 40 Weeks Treatment Duration

Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Body Weight

Timeframe: 40 Weeks Treatment Duration

Subjects (N, %) With Post-baseline Potentially Clinically Important Changes

Timeframe: 40 Weeks Treatment Duration