TerminatedPhase 2/3

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

Stopped: As a result of marketing approval of trofinetide on 10 March 2023, the study was terminated by the Sponsor with the intent of switching patients to commercially available product.

United States15 participantsStarted 2021-09-22

Plain-language summary

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

Who can participate

Age range2 Years – 5 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

✓. 2 to 4 years of age and body weight ≥9 kg and \<20 kg at Screening OR
✓. 5 years of age and body weight ≥9 kg and \<12 kg at Screening

Exclusion criteria

✕. QTcF interval of \>450 ms at Screening or Baseline
✕. History of a risk factor for torsades de pointes (e.g., heart failure or family history of long QT syndrome)
✕. History of clinically significant QT prolongation that is deemed to put the subject at increased risk of clinically significant QT prolongation
✕. Other clinically significant finding on ECG at Screening or Baseline

What they're measuring

Safety and Tolerability of Treatment With Oral Trofinetide

Timeframe: 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)

Timeframe: PK samples were taken predose and at Weeks 2, 4, 8, and 12

Cmax,ss (Maximum Observed Drug Concentration at Steady State)

Timeframe: 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)

Timeframe: PK samples were taken predose and at Weeks 2, 4, 8, and 12

Tmax (Time of the Maximum Observed Drug Concentration at Steady State)

Timeframe: PK samples were taken predose and at Weeks 2, 4, 8, and 12

Trial details

NCT IDNCT04988867

SponsorACADIA Pharmaceuticals Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-05-31

Results submitted2024-08-19

View on ClinicalTrials.gov More Rett Syndrome trials

Who can participate

Age range2 Years – 5 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

✓. 2 to 4 years of age and body weight ≥9 kg and \<20 kg at Screening OR
✓. 5 years of age and body weight ≥9 kg and \<12 kg at Screening

Exclusion criteria

✕. QTcF interval of \>450 ms at Screening or Baseline
✕. History of a risk factor for torsades de pointes (e.g., heart failure or family history of long QT syndrome)
✕. History of clinically significant QT prolongation that is deemed to put the subject at increased risk of clinically significant QT prolongation
✕. Other clinically significant finding on ECG at Screening or Baseline