- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04181723
Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
April 5, 2024 updated by: ACADIA Pharmaceuticals Inc.
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome
Study Overview
Study Type
Interventional
Enrollment (Actual)
187
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Alabama
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Birmingham, Alabama, United States, 35233
- University of Alabama at Birmingham
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Arizona
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Phoenix, Arizona, United States, 85012
- Translational Gemomics Research Institute (TGen)
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California
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La Jolla, California, United States, 92093
- University of California, San Diego
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Sacramento, California, United States, 95817
- UC Davis MIND Institute
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Colorado
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Aurora, Colorado, United States, 80042
- Children's Hospital Colorado
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Florida
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Tampa, Florida, United States, 33606
- Children Medical Services
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Georgia
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Atlanta, Georgia, United States, 30322
- Emory Genetics Clinical Trial Center
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Illinois
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Chicago, Illinois, United States, 60612
- Rush University Children's Hospital
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Maryland
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Baltimore, Maryland, United States, 21205
- Kennedy Krieger Institute - Clinical Trials Unit
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Massachusetts
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Boston, Massachusetts, United States, 02115
- Boston Children's Hospital Harvard Medical School
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Minnesota
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Saint Paul, Minnesota, United States, 55101
- Gillette Children's Specialty Healthcare
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Missouri
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Saint Louis, Missouri, United States, 63110
- Washington University School of Medicine
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New York
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Bronx, New York, United States, 10467
- Montefiore Medical Center, Children's Hospital at Montefiore
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North Carolina
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Chapel Hill, North Carolina, United States, 27599
- The University of North Carolina at Chapel Hill
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Ohio
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Cincinnati, Ohio, United States, 45229
- Cincinnati Children's Hospital Medical Center
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Cleveland, Ohio, United States, 44195
- Cleveland Clinic
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- Children's Hospital of Philadelphia
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South Carolina
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Greenwood, South Carolina, United States, 29646
- Greenwood Genetic Center
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Tennessee
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Nashville, Tennessee, United States, 37232
- Vanderbilt University Medical Center
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Texas
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Houston, Texas, United States, 77030
- Texas Children's Hospital
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Washington
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Seattle, Washington, United States, 98105
- Seattle Children's
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
5 years to 20 years (Child, Adult)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Female subjects 5 to 20 years of age, inclusive, at Screening
- Body weight ≥12 kg at Screening
- Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
- Has classic/typical Rett syndrome (RTT)
- Has a documented disease-causing mutation in the MECP2 gene
- Has a stable pattern of seizures, or has had no seizures, within 8 weeks of Screening
- 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.
- The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
- 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:
- Has been treated with insulin within 12 weeks of Baseline
- 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
- Has a history of, or current, cerebrovascular disease or brain trauma
- Has significant, uncorrected visual or uncorrected hearing impairment
- Has a history of, or current, malignancy
- 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).
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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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)
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Trofinetide solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
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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)
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Trofinetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Clinical Global Impression-Improvement (CGI-I) Score at Week 12
Time Frame: 12 Weeks Treatment Duration
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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.
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12 Weeks Treatment Duration
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Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12
Time Frame: Baseline and Week 12
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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.
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Baseline and Week 12
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social)
Time Frame: 12 Weeks Treatment Duration
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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.
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12 Weeks Treatment Duration
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Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score
Time Frame: 12 Weeks Treatment Duration
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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.
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12 Weeks Treatment Duration
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Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF)
Time Frame: 12 Weeks Treatment Duration
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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.
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12 Weeks Treatment Duration
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Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB)
Time Frame: 12 Weeks Treatment Duration
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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.
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12 Weeks Treatment Duration
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Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC)
Time Frame: 12 Weeks Treatment Duration
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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.
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12 Weeks Treatment Duration
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Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM)
Time Frame: 12 Weeks Treatment Duration
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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.
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12 Weeks Treatment Duration
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Change From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S)
Time Frame: 12 Weeks Treatment Duration
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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.
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12 Weeks Treatment Duration
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Change From Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24)
Time Frame: 12 Weeks Treatment Duration
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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.
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12 Weeks Treatment Duration
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Change From Baseline to Week 12 in Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND)
Time Frame: 12 Weeks Treatment Duration
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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").
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12 Weeks Treatment Duration
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
November 6, 2019
Primary Completion (Actual)
October 28, 2021
Study Completion (Actual)
October 28, 2021
Study Registration Dates
First Submitted
November 26, 2019
First Submitted That Met QC Criteria
November 26, 2019
First Posted (Actual)
November 29, 2019
Study Record Updates
Last Update Posted (Actual)
April 8, 2024
Last Update Submitted That Met QC Criteria
April 5, 2024
Last Verified
April 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- ACP-2566-003
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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