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

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ClinicalTrials.gov Identifier: NCT04181723
Recruitment Status : Completed
First Posted : November 29, 2019
Results First Posted : April 8, 2024
Last Update Posted : April 8, 2024
Sponsor:
Information provided by (Responsible Party):
ACADIA Pharmaceuticals Inc.

Study Description
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Brief Summary:
To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome

Condition or disease Intervention/treatment Phase
Rett Syndrome Drug: Trofinetide Other: Placebo Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 187 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
Actual Study Start Date : November 6, 2019
Actual Primary Completion Date : October 28, 2021
Actual Study Completion Date : October 28, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rett Syndrome

Arms and Interventions
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Arm Intervention/treatment
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
Trofinetide solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
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
Trofinetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks


Outcome Measures
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Primary Outcome Measures :
  1. Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12 [ Time Frame: 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.

  2. Clinical Global Impression-Improvement (CGI-I) Score at Week 12 [ Time Frame: 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 Outcome Measures :
  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) [ Time Frame: 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.

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

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

  4. 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 ]
    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.

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

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

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

  8. 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 ]
    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.

  9. 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 ]
    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").


Eligibility Criteria
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Ages Eligible for Study:   5 Years to 20 Years   (Child, Adult)
Sexes Eligible for Study:   Female
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

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:

  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).

Contacts and Locations
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Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04181723


Locations
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Sponsors and Collaborators
ACADIA Pharmaceuticals Inc.
  Study Documents (Full-Text)

Documents provided by ACADIA Pharmaceuticals Inc.:
Study Protocol  [PDF] August 7, 2020
Statistical Analysis Plan  [PDF] October 20, 2021

More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: ACADIA Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT04181723    
Other Study ID Numbers: ACP-2566-003
First Posted: November 29, 2019    Key Record Dates
Results First Posted: April 8, 2024
Last Update Posted: April 8, 2024
Last Verified: April 2024

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Rett Syndrome
Syndrome
Disease
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
 Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System