CClinicalTrials.gg
Sign In
CompletedNCT04181723Updated Apr 8, 2024Results posted

Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)

A Phase 3 interventional study of Trofinetide and Placebo in Rett Syndrome, sponsored by ACADIA Pharmaceuticals Inc.. Completed at 21 sites in United States. Open to female participants aged 5 Years to 20 Years. Per ClinicalTrials.gov, last updated 2024-04-08.

Sponsored by ACADIA Pharmaceuticals Inc. · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
187
Allocation
Randomized
Ages
5 Years to 20 Years
Sex
Female
01

Study summary

To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome

02

Conditions studied

  • Rett Syndrome
03

In context

Rett Syndrome

98 studies on the registry are indexed under Rett Syndrome; 20 are open to participants now.

This study's enrollment of 187 is above the median of 30 across 64 interventional studies indexed under Rett Syndrome.

Browse Rett Syndrome studies →

Lead sponsor

ACADIA Pharmaceuticals Inc. is the lead sponsor of 41 studies on the registry; 5 are open to participants now.

Of its 25 completed or terminated interventional studies of FDA-regulated products, 23 (92%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
5 Years to 20 Years
Sexes eligible
Female
Accepts healthy volunteers
No

Inclusion criteria

  1. Female subjects 5 to 20 years of age, inclusive, at Screening
  2. Body weight ≥12 kg at Screening
  3. Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
  4. Has classic/typical Rett syndrome (RTT)
  5. Has a documented disease-causing mutation in the MECP2 gene
  6. Has a stable pattern of seizures, or has had no seizures, within 8 weeks of Screening
  7. Subjects of childbearing potential must abstain from sexual activity for the duration of the study and for at least 30 days thereafter or must agree to use acceptable methods of contraception. Subject must not be pregnant or breastfeeding.
  8. The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
  9. Subject and caregiver(s) must reside at a location to which study drug can be delivered and have been at their present residence for at least 3 months prior to Screening

Exclusion criteria

Exclusion Criteria:

  1. Has been treated with insulin within 12 weeks of Baseline
  2. Has current clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, Type 1 diabetes mellitus, or uncontrolled Type 2 diabetes mellitus), renal, hepatic, respiratory or gastrointestinal disease (such as celiac disease or inflammatory bowel disease) or has major surgery planned during the study
  3. Has a history of, or current, cerebrovascular disease or brain trauma
  4. Has significant, uncorrected visual or uncorrected hearing impairment
  5. Has a history of, or current, malignancy
  6. Has a known history or symptoms of long QT syndrome

Additional inclusion/exclusion criteria apply. Patients will be evaluated at screening to ensure that all criteria for study participation are met. Patients may be excluded from the study based on these assessments (and specifically, if it is determined that their baseline health and condition do not meet all pre-specified entry criteria).

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
187 participants (actual)

Study arms

  • Experimental
    Drug - Trofinetide

    Trofinetide solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)

    Drug: Trofinetide

  • Placebo comparator
    Placebo

    Trofinetide placebo solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)

    Other: Placebo

Interventions

  • DrugTrofinetide

    Trofinetide solution administered based on the subject's weight at Baseline, twice daily for 12 weeks

  • OtherPlacebo

    Trofinetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks

06

What researchers measure

Primary outcomes

  1. Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to 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.

    Time frame: Baseline and Week 12

  2. Clinical Global Impression-Improvement (CGI-I) Score at Week 12

    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.

    Time frame: 12 Weeks Treatment Duration

Secondary outcomes

  1. Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social)

    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.

    Time frame: 12 Weeks Treatment Duration

  2. Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score

    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.

    Time frame: 12 Weeks Treatment Duration

  3. Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF)

    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.

    Time frame: 12 Weeks Treatment Duration

  4. Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB)

    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.

    Time frame: 12 Weeks Treatment Duration

  5. Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC)

    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.

    Time frame: 12 Weeks Treatment Duration

  6. Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM)

    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.

    Time frame: 12 Weeks Treatment Duration

  7. Change From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S)

    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.

    Time frame: 12 Weeks Treatment Duration

  8. Change From Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24)

    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.

    Time frame: 12 Weeks Treatment Duration

  9. Change From Baseline to Week 12 in Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND)

    The overall quality of life of the subject is also rated by responding to the following: "Please rate your child's overall 'Quality of Life' on the scale below. Choose the number which you feel is best and circle it." The choices range from 1 ("Poor") to 6 ("Excellent").

    Time frame: 12 Weeks Treatment Duration

07

Results

Posted Apr 8, 2024

Participant flow

Participant flow — Overall Study
MilestoneDrug - TrofinetidePlacebo
Started9394
Completed7085
Not completed239

Outcome measures

PrimaryRett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to 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.

Time frame:
Baseline and Week 12
Reported as:
Least squares mean · score on a scale
Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12
score on a scaleDrug - TrofinetidePlacebo
Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12-4.9 ± 0.94-1.7 ± 0.90
Statistical analysis
  • Drug - Trofinetide vs Placebo · Mixed-effects model for repeated measure · p = 0.0175 · Lsm difference: -3.1 · 95% CI -5.7 to -0.6
PrimaryClinical Global Impression-Improvement (CGI-I) Score at Week 12

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.

Time frame:
12 Weeks Treatment Duration
Reported as:
Least squares mean · score on a scale
Clinical Global Impression-Improvement (CGI-I) Score at Week 12
score on a scaleDrug - TrofinetidePlacebo
Clinical Global Impression-Improvement (CGI-I) Score at Week 123.5 ± 0.073.8 ± 0.07
Statistical analysis
  • Drug - Trofinetide vs Placebo · Mixed-effects model for repeated measure · p = 0.0030 · Lsm difference: -0.3 · 95% CI -0.5 to -0.1
SecondaryChange From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social)

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.

Time frame:
12 Weeks Treatment Duration
Reported as:
Least squares mean · 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)
score on a scaleDrug - TrofinetidePlacebo
Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social)-0.1 ± 0.26-1.1 ± 0.25
Statistical analysis
  • Drug - Trofinetide vs Placebo · Mixed-effects model for repeated measure · p = 0.0064 · Lsm difference: 1.0 · 95% CI 0.3 to 1.7
SecondaryChange From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score

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.

Time frame:
12 Weeks Treatment Duration
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score
score on a scaleDrug - TrofinetidePlacebo
Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score-6.5 ± 3.44-1.9 ± 3.14
Statistical analysis
  • Drug - Trofinetide vs Placebo · ANCOVA · p = 0.3376 · Lsm difference: -4.5 · 95% CI -13.8 to 4.8
SecondaryChange From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF)

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.

Time frame:
12 Weeks Treatment Duration
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF)
score on a scaleDrug - TrofinetidePlacebo
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF)-0.1 ± 0.080.0 ± 0.07
Statistical analysis
  • Drug - Trofinetide vs Placebo · Mixed-effects model for repeated measure · p = 0.3649 · Lsm difference: -0.1 · 95% CI -0.3 to 0.1
SecondaryChange From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB)

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.

Time frame:
12 Weeks Treatment Duration
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB)
score on a scaleDrug - TrofinetidePlacebo
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB)-0.2 ± 0.070.0 ± 0.07
Statistical analysis
  • Drug - Trofinetide vs Placebo · Mixed-effects model for repeated measure · p = 0.2114 · Lsm difference: -0.1 · 95% CI -0.3 to 0.1
SecondaryChange From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC)

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.

Time frame:
12 Weeks Treatment Duration
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC)
score on a scaleDrug - TrofinetidePlacebo
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC)-0.4 ± 0.110.0 ± 0.10
Statistical analysis
  • Drug - Trofinetide vs Placebo · Mixed-effects model for repeated measure · p = 0.0257 · Lsm difference: -0.3 · 95% CI -0.6 to 0.0
SecondaryChange From Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM)

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.

Time frame:
12 Weeks Treatment Duration
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM)
score on a scaleDrug - TrofinetidePlacebo
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM)0.0 ± 0.060.0 ± 0.06
Statistical analysis
  • Drug - Trofinetide vs Placebo · Mixed-effects model for repeated measure · p = 0.9799 · Lsm difference: 0.0 · 95% CI -0.2 to 0.2
SecondaryChange From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S)

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.

Time frame:
12 Weeks Treatment Duration
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S)
score on a scaleDrug - TrofinetidePlacebo
Change From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S)0.0 ± 0.030.0 ± 0.03
Statistical analysis
  • Drug - Trofinetide vs Placebo · Mixed-effects model for repeated measure · p = 0.5304 · Lsm difference: 0.0 · 95% CI -0.1 to 0.1
SecondaryChange From Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24)

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.

Time frame:
12 Weeks Treatment Duration
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24)
score on a scaleDrug - TrofinetidePlacebo
Change From Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24)-1.1 ± 1.01-0.4 ± 0.96
Statistical analysis
  • Drug - Trofinetide vs Placebo · ANCOVA · p = 0.5855 · Lsm difference: -0.8 · 95% CI -3.5 to 2.0
SecondaryChange From Baseline to Week 12 in Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND)

The overall quality of life of the subject is also rated by responding to the following: "Please rate your child's overall 'Quality of Life' on the scale below. Choose the number which you feel is best and circle it." The choices range from 1 ("Poor") to 6 ("Excellent").

Time frame:
12 Weeks Treatment Duration
Reported as:
Least squares mean · score on a scale
Change From Baseline to Week 12 in Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND)
score on a scaleDrug - TrofinetidePlacebo
Change From Baseline to Week 12 in Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND)0.2 ± 0.090.1 ± 0.09
Statistical analysis
  • Drug - Trofinetide vs Placebo · Mixed-effects model for repeated measure · p = 0.2507 · Lsm difference: 0.1 · 95% CI -0.1 to 0.4

Adverse events

Collected over Approximately 4 months. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Drug - Trofinetide0/93 (0%)3/93 (3.2%)81/93 (87.1%)
Placebo0/94 (0%)3/94 (3.2%)32/94 (34%)
Most frequent serious events
Most frequent serious events
EventDrug - TrofinetidePlacebo
BacteraemiaInfections and infestations1/930/94
BronchiolitisInfections and infestations1/930/94
COVID-19 pneumoniaInfections and infestations1/930/94
Urinary tract infectionInfections and infestations1/930/94
SeizureNervous system disorders1/930/94
ConstipationGastrointestinal disorders0/931/94
Pneumatosis intestinalisGastrointestinal disorders0/931/94
Respiratory distressRespiratory, thoracic and mediastinal disorders0/931/94
Most frequent other events
Most frequent other events
EventDrug - TrofinetidePlacebo
DiarrhoeaGastrointestinal disorders75/9318/94
VomitingGastrointestinal disorders25/939/94
PyrexiaGeneral disorders8/934/94
SeizureNervous system disorders7/935/94
IrritabilityPsychiatric disorders6/930/94
Decreased appetiteMetabolism and nutrition disorders5/932/94

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Drug - TrofinetidePlaceboTotal
<=18 years8589174
Between 18 and 65 years8513
>=65 years000
Age, Continuous
Age, Continuous(years)Drug - TrofinetidePlaceboTotal
Mean11 ± 4.6910.9 ± 4.5710.9 ± 4.62
Sex: Female, Male
Sex: Female, Male(Participants)Drug - TrofinetidePlaceboTotal
Female9394187
Male000
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Drug - TrofinetidePlaceboTotal
Hispanic or Latino71017
Not Hispanic or Latino8684170
Unknown or Not Reported000
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)Drug - TrofinetidePlaceboTotal
White8290172
Black or African American112
Asian516
American Indian or Alaska Native000
Native Hawaiian or Other Pacific Islander101
Other426
Region of Enrollment
Region of Enrollment(participants)Drug - TrofinetidePlaceboTotal
United States9394187
08

Study locations

21 sites
  • University of Alabama at Birmingham
    Birmingham, Alabama 35233, United States
  • Translational Gemomics Research Institute (TGen)
    Phoenix, Arizona 85012, United States
  • University of California, San Diego
    La Jolla, California 92093, United States
  • UC Davis MIND Institute
    Sacramento, California 95817, United States
  • Children's Hospital Colorado
    Aurora, Colorado 80042, United States
  • Children Medical Services
    Tampa, Florida 33606, United States
  • Emory Genetics Clinical Trial Center
    Atlanta, Georgia 30322, United States
  • Rush University Children's Hospital
    Chicago, Illinois 60612, United States
  • Kennedy Krieger Institute - Clinical Trials Unit
    Baltimore, Maryland 21205, United States
  • Boston Children's Hospital Harvard Medical School
    Boston, Massachusetts 02115, United States
  • Gillette Children's Specialty Healthcare
    Saint Paul, Minnesota 55101, United States
  • Washington University School of Medicine
    Saint Louis, Missouri 63110, United States
  • Montefiore Medical Center, Children's Hospital at Montefiore
    Bronx, New York 10467, United States
  • The University of North Carolina at Chapel Hill
    Chapel Hill, North Carolina 27599, United States
  • Cincinnati Children's Hospital Medical Center
    Cincinnati, Ohio 45229, United States
  • Cleveland Clinic
    Cleveland, Ohio 44195, United States
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania 19104, United States
  • Greenwood Genetic Center
    Greenwood, South Carolina 29646, United States
  • Vanderbilt University Medical Center
    Nashville, Tennessee 37232, United States
  • Texas Children's Hospital
    Houston, Texas 77030, United States
  • Seattle Children's
    Seattle, Washington 98105, United States
09

References and documents

Publications

  • Neul JL, Percy AK, Benke TA, Berry-Kravis EM, Glaze DG, Peters SU, Jones NE, Youakim JM. Design and outcome measures of LAVENDER, a phase 3 study of trofinetide for Rett syndrome. Contemp Clin Trials. 2022 Mar;114:106704. doi: 10.1016/j.cct.2022.106704. Epub 2022 Feb 8. PubMed 35149233 ↗

Study documents

  • Study protocol · Aug 7, 2020
  • Statistical analysis plan · Oct 20, 2021

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Apr 8, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT04181723
Lead sponsor
ACADIA Pharmaceuticals Inc.
Responsible party
Sponsor
First posted
Nov 29, 2019
Start date
Nov 6, 2019
Primary completion
Oct 28, 2021
Completion
Oct 28, 2021
Results posted
Apr 8, 2024
Last update
Apr 8, 2024

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Apr 2024. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Rett Syndrome→

Other studies from this sponsor

ACADIA Pharmaceuticals Inc.

Start the discussion