Trial · NCT04776746
Open-Label Extension Study of Trofinetide for Rett Syndrome
NCT04776746, Phase 3, Terminated, ACADIA Pharmaceuticals Inc.; results first posted 2024-09-27
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Open-Label Extension Study of Trofinetide for Rett Syndrome. Trials, record NCT04776746. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04776746
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Open-Label Extension Study of Trofinetide for Rett Syndrome
- NCT number
- NCT04776746
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 77 participants, actual
- Lead sponsor
- ACADIA Pharmaceuticals Inc.
- Conditions
- Rett Syndrome
- Interventions
- trofinetide
- First posted
- 2021-03-02
- Last updated
- 2024-09-27
- Results first posted
- 2024-09-27
- Limitations and caveats
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. Date of study termination was 30 June 2023.
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Patients With Treatment-emergent Adverse Events (TEAEs), With Serious Adverse Events (SAEs), and With Withdrawals Due to AEs | Mean study drug exposure was 426 days, corresponding to 1.2 years | Withdrawals due to AEs included both study drug discontinuation as well as study termination |
| Primary | Number (%) of Patients With Potentially Clinically Important Changes in ECG Post-baseline | Mean study drug exposure was 426 days, corresponding to 1.2 years | Potentially clinically significant ECG changes were defined as QTcF \>500 ms or QTcF change from baseline (CFB) of \>60 ms |
| Primary | Number (%) of Patients With Potentially Clinically Important Changes in Vital Signs Post-baseline | Mean study drug exposure was 426 days, corresponding to 1.2 years | 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. |
| Primary | Number (%) of Patients With Potentially Clinically Important Changes in Body Weight Post-baseline | Mean study drug exposure was 426 days, corresponding to 1.2 years | Potentially clinically important changes from baseline in body weight were defined as: Weight increase ≥7% from baseline Weight decrease ≥7% from baseline |
| Primary | Number (%) of Patients With Potentially Clinically Important Changes in Laboratory Parameters Post-baseline | Mean study drug exposure was 426 days, corresponding to 1.2 years | 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 |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Patients With Treatment-emergent Adverse Events (TEAEs), With Serious Adverse Events (SAEs), and With Withdrawals Due to AEs | Mean study drug exposure was 426 days, corresponding to 1.2 years | All patients enrolled and treated | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number (%) of Patients With Potentially Clinically Important Changes in ECG Post-baseline | Mean study drug exposure was 426 days, corresponding to 1.2 years | All patients enrolled and treated | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number (%) of Patients With Potentially Clinically Important Changes in Vital Signs Post-baseline | Mean study drug exposure was 426 days, corresponding to 1.2 years | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Number (%) of Patients With Potentially Clinically Important Changes in Body Weight Post-baseline | Mean study drug exposure was 426 days, corresponding to 1.2 years | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Number (%) of Patients With Potentially Clinically Important Changes in Laboratory Parameters Post-baseline | Mean study drug exposure was 426 days, corresponding to 1.2 years | All patients enrolled and treated and with at least one postbaseline value for the respective parameter (n=75; apart from deviating patient numbers of n=74 for uric acid, LDH, AST and bilirubin) | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group36
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Patients With Treatment-emergent Adverse Events (TEAEs), With Serious Adverse Events (SAEs), and With Withdrawals Due to AEs | Trofinetide | Patients with TEAEs | 68 | Participants | |
| Percentage of Patients With Treatment-emergent Adverse Events (TEAEs), With Serious Adverse Events (SAEs), and With Withdrawals Due to AEs | Trofinetide | Patients with SAEs | 23 | Participants | |
| Percentage of Patients With Treatment-emergent Adverse Events (TEAEs), With Serious Adverse Events (SAEs), and With Withdrawals Due to AEs | Trofinetide | Patients with AEs leading to discontinuation | 9 | Participants | |
| Number (%) of Patients With Potentially Clinically Important Changes in ECG Post-baseline | Trofinetide | QTcF >500 ms | 0 | Participants | |
| Number (%) of Patients With Potentially Clinically Important Changes in ECG Post-baseline | Trofinetide | QTcF CFB >60 ms | 1 | Participants | |
| Number (%) of Patients With Potentially Clinically Important Changes in Vital Signs Post-baseline | Trofinetide | SBP≥180 mmHg and increased ≥20 mmHg from BL | 0 | Participants | |
| Number (%) of Patients With Potentially Clinically Important Changes in Vital Signs Post-baseline | Trofinetide | SBP≤90 mmHg and decreased ≥20 mmHg from BL | 5 | Participants | |
| Number (%) of Patients With Potentially Clinically Important Changes in Vital Signs Post-baseline | Trofinetide | DBP≥105 mmHg and increased ≥15 mmHg from BL | 1 | Participants | |
| Number (%) of Patients With Potentially Clinically Important Changes in Vital Signs Post-baseline | Trofinetide | DBP≤50 mmHg and decreased ≥15 mmHg from BL | 4 | Participants | |
| Number (%) of Patients With Potentially Clinically Important Changes in Vital Signs Post-baseline | Trofinetide | PR≥120 bpm and increased ≥15 bpm from BL | 4 | Participants |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Trofinetide | 77 | |
| Overall Study | COMPLETED | Trofinetide | 0 | |
| Overall Study | NOT COMPLETED | Trofinetide | 77 |
Source ClinicalTrials.gov
Adverse events53
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Diarrhoea | Gastrointestinal disorders | Trofinetide | 13 | 77 | 18 |
| other | Vomiting | Gastrointestinal disorders | Trofinetide | 10 | 77 | 12 |
| other | Constipation | Gastrointestinal disorders | Trofinetide | 9 | 77 | 9 |
| other | Pyrexia | General disorders | Trofinetide | 12 | 77 | 20 |
| other | COVID-19 | Infections and infestations | Trofinetide | 20 | 77 | 22 |
| other | Urinary tract infection | Infections and infestations | Trofinetide | 12 | 77 | 21 |
| other | Upper respiratory tract infection | Infections and infestations | Trofinetide | 9 | 77 | 13 |
| other | Influenza | Infections and infestations | Trofinetide | 6 | 77 | 6 |
| other | Pharyngitis streptococcal | Infections and infestations | Trofinetide | 5 | 77 | 5 |
| other | Gastroenteritis viral | Infections and infestations | Trofinetide | 4 | 77 | 4 |
1 to 10 of 53Source ClinicalTrials.gov
See all 53 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Trofinetide | 4 | 77 |
| Total, other adverse events | Trofinetide | 57 | 77 |
| Total, serious adverse events | Trofinetide | 23 | 77 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04776746
GET https://conductscience.org/api/trials/v1/NCT04776746/results?page=2