Phase 3
Completed N=187
Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
Source: ClinicalTrials.gov NCT04181723 ↗Enrolled (actual)
187
Serious AEs
3.2%
Results posted
Apr 2024
Primary outcomePrimary: Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12 — -4.9; -1.7 score on a scale — p=0.0175
◆ Published Evidence
Highly cited
190citations · ~63 / year
Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study.
Summary
To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome
Linked Publications (4)
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Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study.
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Design and outcome measures of LAVENDER, a phase 3 study of trofinetide for Rett syndrome.
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Trofinetide: a pioneering treatment for Rett syndrome.
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Exposure-Response Efficacy Modeling to Support Trofinetide Dosing in Individuals with Rett Syndrome.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12 |
-4.9; -1.7 | 0.0175 sig |
| PRIMARY Clinical Global Impression-Improvement (CGI-I) Score at Week 12 |
3.5; 3.8 | 0.0030 sig |
| 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) |
-0.1; -1.1 | 0.0064 sig |
| SECONDARY Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score |
-6.5; -1.9 | 0.3376 |
| SECONDARY Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF) |
-0.1; 0.0 | 0.3649 |
| SECONDARY Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB) |
-0.2; 0.0 | 0.2114 |
| SECONDARY Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC) |
-0.4; 0.0 | 0.0257 sig |
| SECONDARY Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM) |
0.0; 0.0 | 0.9799 |
| SECONDARY Change From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S) |
0.0; 0.0 | 0.5304 |
| SECONDARY Change From Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24) |
-1.1; -0.4 | 0.5855 |
| SECONDARY Change From Baseline to Week 12 in Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND) |
0.2; 0.1 | 0.2507 |
Eligibility Criteria
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).
Data sourced from ClinicalTrials.gov (NCT04181723) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.