Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
Public ClinicalTrials.gov record NCT04279314. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A 40-Week, Open-label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls and women with Rett syndrome
Study identification
- NCT ID
- NCT04279314
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 154 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 5 Years to 21 Years
- Sex
- Female
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jan 29, 2020 (Actual)
- Primary completion
- Aug 19, 2022 (Actual)
- Completion
- Aug 19, 2022 (Actual)
- Last update posted
- Apr 11, 2024
2020 – 2022
United States locations
- U.S. sites
- 21
- U.S. states
- 19
- U.S. cities
- 21
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35223 | — |
| Translational Genomics Research Institute (TGen) | Phoenix | Arizona | 85012 | — |
| University of California, San Diego | La Jolla | California | 92093 | — |
| UC Davis MIND Institute | Sacramento | California | 95817 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Children Medical Services | Tampa | Florida | 33606 | — |
| Emory Genetics Clinical Trial Center | Atlanta | Georgia | 30322 | — |
| Rush University Medical Center | Chicago | Illinois | 60612 | — |
| Kennedy Krieger Institute - Clinical Trials Unit | Baltimore | Maryland | 21205 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Gillette Children's Specialty Healthcare | Saint Paul | Minnesota | 55101 | — |
| Washington University School of Medicine, St. Louis Children's Hospital | St Louis | Missouri | 63110 | — |
| Montefiore Medical Center, Children's Hospital at Montefiore | The Bronx | New York | 10467 | — |
| The University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| Cleveland Clinic | Cleveland | Ohio | 44195 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Greenwood Genetic Center | Greenwood | South Carolina | 29626 | — |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | — |
| Texas Children's Hospital | Houston | Texas | 77030 | — |
| Seattle Children's | Seattle | Washington | 98105 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04279314, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 11, 2024 · Synced Oct 22, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04279314 live on ClinicalTrials.gov.