Trial · NCT04279314
Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
NCT04279314, Phase 3, Completed, ACADIA Pharmaceuticals Inc.; results first posted 2024-04-11
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome. Trials, record NCT04279314. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04279314
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
- NCT number
- NCT04279314
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 154 participants, actual
- Lead sponsor
- ACADIA Pharmaceuticals Inc.
- Conditions
- Rett Syndrome
- Interventions
- Trofinetide
- First posted
- 2020-02-21
- Last updated
- 2024-04-11
- Results first posted
- 2024-04-11
Source ClinicalTrials.gov
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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 | 40 Weeks Treatment Duration | 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 |
| Primary | Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in ECG | 40 Weeks Treatment Duration | Potentially clinically important ECG changes were defined in the study protocol as absolute QTcF interval \>500 ms or QTcF interval change from the baseline value of previous study ACP-2566-003 of \>60 ms |
| Primary | Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs | 40 Weeks Treatment Duration | Potentially clinically important changes in vital signs were defined in the study protocol 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 ≥120 bpm and increased ≥15 bpm from baseline; Pulse ≤50 bpm and decreased ≥15 bpm from baseline |
| Primary | Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Body Weight | 40 Weeks Treatment Duration | Potentially clinically important changes in body weight were defined in the study protocol as: Weight increase ≥7% from baseline; Weight decrease ≥7% from baseline |
| Primary | Subjects (N, %) With Post-baseline Potentially Clinically Important Changes | 40 Weeks Treatment Duration | Potentially clinically important changes in laboratory parameters were defined in the study protocol 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 \>2.0 mmol/L; Blood urea nitrogen ≥10.71 mmol/L; Creatinine \>1.5 x upper limit of normal (ULN); Uric acid ≥505.75 μmol/L; Lactate dehydrogenase ≥3 x 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 aminotransferase ≥3 x ULN; Aspartate aminotransferase ≥3 x ULN; Gamma glutamyl transpeptidase ≥3 x ULN; Alkaline phosphatase ≥3 x ULN; Bilirubin ≥1.5 x ULN |
| Secondary | Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score Change From Baseline to Week 40 | 40 Weeks Treatment Duration | The RSBQ is a 45-item caregiver-completed rating scalescale includes 45 items, 39 of them grouped into 8 subscales, whose ratings reflect the severity and frequency of symptoms. Items are rated as 0 (not true), 1 (somewhat or sometimes true), or 2 (very true). The 8 subscales are general mood, breathing problems, hand behavior, face movements, body rocking/expressionless face, night-time behaviors, fear/anxiety, and walking/standing. Scores for item 31 are reversed in the calculation of the total score. The total score ranges from 0 to 90 and is calculated as the sum of the item scores. Higher scores mean worse behaviour. |
| Secondary | Clinical Global Impression-Improvement (CGI-I) Score at Week 40 | 40 Weeks Treatment Duration | To rate how much the subject's illness has improved or worsened relative to a baseline state, a 7-point scale is used from 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse, 7=very much worse. Higher CGI-I scores denote more severe illness and less improvement in the illness. |
| Secondary | Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social) Change From Baseline to Week 40 | 40 Weeks Treatment Duration | Scale to assess communication and pre-linguistic skills in children 12-24 months (or older children with developmental delay). The Checklist consists of 24 questions ranging from 0 to 4 points within each of 7 Clusters. 0 points are given for"Not Yet", 1 point for "Sometimes", or 2 points for "Often". For items describing a series of numbers or ranges, 0 points are given for "None" and 1 to 4 points for items containing numbered choices. The Social Composite score is one of 3 composite scores. It comprises 13 items in skill areas "Emotion and Eye Gaze" (items 1 to 4), "Communication" (items 5 to 8), and "Gestures" (items 9 to 13). The Social Composite raw score (items 1 to 13), ranging from 0 to 26, is calculated as the sum of the item scores. Higher Social Composite raw scores indicate better social communication development. |
| Secondary | Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND) Change From Baseline to Week 40 | 40 Weeks Treatment Duration | The overall quality of life score rating of the ICND ranges from 1 ("Poor") to 6 ("Excellent"); lower overall quality of life scores indicate lower quality of life. |
| Secondary | Rett Syndrome Clinician Rating of Hand Function (RTT-HF) Change From Baseline to Week 40 | 40 Weeks Treatment Duration | The RTT-HF is a clinician completed clinical assessment of the subject's ability to use her hands for functional purposes. The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 registered outcomesOutcomes16
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | 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 | 40 Weeks Treatment Duration | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in ECG | 40 Weeks Treatment Duration | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs | 40 Weeks Treatment Duration | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Body Weight | 40 Weeks Treatment Duration | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Subjects (N, %) With Post-baseline Potentially Clinically Important Changes | 40 Weeks Treatment Duration | Subjects with at least 1 post-baseline value for the given parameter (n=151 for all parameters, except LDH (n=150) and AST (=150) | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score Change From Baseline to Week 40 | 40 Weeks Treatment Duration | score on a scale | MEAN | |
| SECONDARY | Clinical Global Impression-Improvement (CGI-I) Score at Week 40 | 40 Weeks Treatment Duration | score on a scale | MEAN | |
| SECONDARY | Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social) Change From Baseline to Week 40 | 40 Weeks Treatment Duration | score on a scale | MEAN | |
| SECONDARY | Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND) Change From Baseline to Week 40 | 40 Weeks Treatment Duration | score on a scale | MEAN | |
| SECONDARY | Rett Syndrome Clinician Rating of Hand Function (RTT-HF) Change From Baseline to Week 40 | 40 Weeks Treatment Duration | score on a scale | MEAN |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 outcomesResults by group47
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| 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 | Trofinetide | Percentage of subjects with TEAEs | 132 | Participants | |
| 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 | Trofinetide | percentage of subjects with SAEs | 19 | Participants | |
| 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 | Trofinetide | Percentage of subjects withdrawals due to AEs | 48 | Participants | |
| Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in ECG | Trofinetide | 2 | Participants | ||
| Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs | Trofinetide | SBP ≥180 mmHg and increased ≥20 mmHg from baseline | 0 | Participants | |
| Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs | Trofinetide | SBP ≤90 mmHg and decreased ≥20 mmHg from baseline | 11 | Participants | |
| Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs | Trofinetide | DBP ≥ 105 mmHg and increased ≥15 mmHg from baseline | 0 | Participants | |
| Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs | Trofinetide | DBP ≤50 mmHg and decreased ≥15 mmHg from baseline | 13 | Participants | |
| Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs | Trofinetide | Pulse ≥120 bpm and increased ≥15 bpm from baseline | 14 | Participants | |
| Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs | Trofinetide | Pulse ≤50 bpm and decreased ≥15 bpm from baseline | 0 | Participants |
1 to 10 of 47Source ClinicalTrials.gov
See all 47 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 | 154 | |
| Overall Study | COMPLETED | Trofinetide | 84 | |
| Overall Study | NOT COMPLETED | Trofinetide | 70 |
Source ClinicalTrials.gov
Adverse events39
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 | 91 | 154 | 129 |
| other | Vomiting | Gastrointestinal disorders | Trofinetide | 39 | 154 | 58 |
| other | Pyrexia | General disorders | Trofinetide | 11 | 154 | 14 |
| other | COVID-19 | Infections and infestations | Trofinetide | 16 | 154 | 16 |
| other | Upper respiratory tract infection | Infections and infestations | Trofinetide | 12 | 154 | 14 |
| other | Urinary tract infection | Infections and infestations | Trofinetide | 9 | 154 | 14 |
| other | Weight decreased | Investigations | Trofinetide | 9 | 154 | 9 |
| other | Decreased appetite | Metabolism and nutrition disorders | Trofinetide | 10 | 154 | 11 |
| other | Seizure | Nervous system disorders | Trofinetide | 10 | 154 | 14 |
| serious | Anaemia | Blood and lymphatic system disorders | Trofinetide | 1 | 154 | 1 |
1 to 10 of 39Source ClinicalTrials.gov
See all 39 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Trofinetide | 0 | 154 |
| Total, other adverse events | Trofinetide | 114 | 154 |
| Total, serious adverse events | Trofinetide | 19 | 154 |
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/NCT04279314
GET https://conductscience.org/api/trials/v1/NCT04279314/results?page=2