Trial · NCT04181723
Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
NCT04181723, Phase 3, Completed, ACADIA Pharmaceuticals Inc.; results first posted 2024-04-08
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™). Trials, record NCT04181723. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04181723
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
- NCT number
- NCT04181723
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 187 participants, actual
- Lead sponsor
- ACADIA Pharmaceuticals Inc.
- Conditions
- Rett Syndrome
- Interventions
- Trofinetide, Placebo
- First posted
- 2019-11-29
- Last updated
- 2024-04-08
- Results first posted
- 2024-04-08
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12 | Baseline and Week 12 | The RSBQ is a 45-item caregiver-completed rating scale that 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. |
| Primary | Clinical Global Impression-Improvement (CGI-I) Score at Week 12 | 12 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. |
| Secondary | Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social) | 12 Weeks Treatment Duration | Standardized screening scale for assessing communication and pre-linguistic skills in young children 12-24 months and can be used with older children with developmental delay. The CSBS-DP includes a suite of three separate measures: The Infant-Toddler Checklist, a follow-up Caregiver Questionnaire and a Behavior Sample. In this study only the Infant-Toddler (CSBS-DP-IT) Checklist was used. The CSBS-DP-IT Checklist is a 24-item rating scale and each item is scored using a three-level rating of frequency: "not yet", "sometimes" and "often". The CSBS-DP-IT Social Composite score the range was 0 to 26 and a higher score represented a worse outcome. Three composite scores can be calculated: 1) Social Composite; 2) Speech Composite; 3) Symbolic Composite. |
| Secondary | Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score | 12 Weeks Treatment Duration | The Impact of Childhood Neurologic Disability (ICND) scale evaluates the effect of four conditions or health problems on 11 aspects of a child's or family's life as "A lot", "Some", "A little", "Not at all", or "Does not apply". The four conditions or health problems are 1) inattentiveness, impulsivity, or mood, 2) ability to think and remember, 3) neurologic or physical limitations, and 4) epilepsy. The ICND score the range of 0 to 132 and a higher score represents a worse outcome. |
| Secondary | Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF) | 12 Weeks Treatment Duration | Clinical assessment of the subject's ability to use their hands for functional purposes (such as reaching for and grasping objects, self-feeding, or drawing). The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment. |
| Secondary | Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB) | 12 Weeks Treatment Duration | Clinical assessment of the subject's ability to sit, stand, and ambulate (e.g., walking, running, and climbing stairs). The assessment was made on an 8-point Likert scale (0 to 7), with 0 denoting normal functioning and 7 as the most severe impairment. |
| Secondary | Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC) | 12 Weeks Treatment Duration | Clinical assessment of the subject's ability to communicate their choices or preferences, which can include the use of nonverbal means such as eye contact or gestures. The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment. |
| Secondary | Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM) | 12 Weeks Treatment Duration | Clinical assessment of the subject's ability to communicate verbally (e.g. words and phrases). The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment. |
| Secondary | Change From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S) | 12 Weeks Treatment Duration | A 7 point scale that rates the severity of the subject's illness at the time of assessment, relative to the clinician's experience with subjects who have the same diagnosis. A subject is assessed on severity of illness at the time of rating: 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill. |
| Secondary | Change From Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24) | 12 Weeks Treatment Duration | The Rett Syndrome Caregiver Burden Inventory (RTT-CBI) scale is intended to directly address caregiver burden and indirectly assess the significance of treatment effects on function in the context of activities of daily living. Ratings are on a 5-point Likert scale including: 0-never; 1-rarely; 2-sometimes; 3-frequently and 4-nearly always. As in the original Caregiver Burden Inventory, the RTT-CBI has 24 negatively worded items (items 1 through 24) yielding a total score up to 96. The RTT-CBI the range is 0 to 96 and a higher score represents a worse outcome. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12 | Baseline and Week 12 | score on a scale | LEAST_SQUARES_MEAN | |
| PRIMARY | Clinical Global Impression-Improvement (CGI-I) Score at Week 12 | 12 Weeks Treatment Duration | score on a scale | LEAST_SQUARES_MEAN | |
| SECONDARY | Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social) | 12 Weeks Treatment Duration | score on a scale | LEAST_SQUARES_MEAN | |
| SECONDARY | Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score | 12 Weeks Treatment Duration | score on a scale | LEAST_SQUARES_MEAN | |
| SECONDARY | Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF) | 12 Weeks Treatment Duration | score on a scale | LEAST_SQUARES_MEAN | |
| SECONDARY | Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB) | 12 Weeks Treatment Duration | score on a scale | LEAST_SQUARES_MEAN | |
| SECONDARY | Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC) | 12 Weeks Treatment Duration | score on a scale | LEAST_SQUARES_MEAN | |
| SECONDARY | Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM) | 12 Weeks Treatment Duration | score on a scale | LEAST_SQUARES_MEAN | |
| SECONDARY | Change From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S) | 12 Weeks Treatment Duration | score on a scale | LEAST_SQUARES_MEAN | |
| SECONDARY | Change From Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24) | 12 Weeks Treatment Duration | score on a scale | LEAST_SQUARES_MEAN |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group22
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12 | Drug - Trofinetide | -4.9 | Standard Error 0.94 | score on a scale | |
| Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12 | Placebo | -1.7 | Standard Error 0.90 | score on a scale | |
| Clinical Global Impression-Improvement (CGI-I) Score at Week 12 | Drug - Trofinetide | 3.5 | Standard Error 0.07 | score on a scale | |
| Clinical Global Impression-Improvement (CGI-I) Score at Week 12 | Placebo | 3.8 | Standard Error 0.07 | score on a scale | |
| Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social) | Drug - Trofinetide | -0.1 | Standard Error 0.26 | score on a scale | |
| Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social) | Placebo | -1.1 | Standard Error 0.25 | score on a scale | |
| Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score | Drug - Trofinetide | -6.5 | Standard Error 3.44 | score on a scale | |
| Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score | Placebo | -1.9 | Standard Error 3.14 | score on a scale | |
| Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF) | Drug - Trofinetide | -0.1 | Standard Error 0.08 | score on a scale | |
| Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF) | Placebo | 0.0 | Standard Error 0.07 | score on a scale |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Drug - Trofinetide | 93 | |
| Overall Study | STARTED | Placebo | 94 | |
| Overall Study | COMPLETED | Drug - Trofinetide | 70 | |
| Overall Study | COMPLETED | Placebo | 85 | |
| Overall Study | NOT COMPLETED | Drug - Trofinetide | 23 | |
| Overall Study | NOT COMPLETED | Placebo | 9 |
Source ClinicalTrials.gov
Adverse events28
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 | Drug - Trofinetide | 75 | 93 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 18 | 94 | |
| other | Vomiting | Gastrointestinal disorders | Drug - Trofinetide | 25 | 93 | |
| other | Vomiting | Gastrointestinal disorders | Placebo | 9 | 94 | |
| other | Pyrexia | General disorders | Drug - Trofinetide | 8 | 93 | |
| other | Pyrexia | General disorders | Placebo | 4 | 94 | |
| other | Decreased appetite | Metabolism and nutrition disorders | Drug - Trofinetide | 5 | 93 | |
| other | Decreased appetite | Metabolism and nutrition disorders | Placebo | 2 | 94 | |
| other | Seizure | Nervous system disorders | Drug - Trofinetide | 7 | 93 | |
| other | Seizure | Nervous system disorders | Placebo | 5 | 94 |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Drug - Trofinetide | 0 | 93 |
| Total, all-cause mortality | Placebo | 0 | 94 |
| Total, other adverse events | Drug - Trofinetide | 81 | 93 |
| Total, other adverse events | Placebo | 32 | 94 |
| Total, serious adverse events | Drug - Trofinetide | 3 | 93 |
| Total, serious adverse events | Placebo | 3 | 94 |
Source ClinicalTrials.gov
Papers4
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Drug - Trofinetide vs Placebo: deaths from any cause | Trial comparisons |
| Drug - Trofinetide vs Placebo: serious adverse events | Trial comparisons |
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/NCT04181723
GET https://conductscience.org/api/trials/v1/NCT04181723/results?page=2