Trofinetide for Rett Syndrome
(DAFFODIL™ Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests trofinetide, a medication, to determine its safety and tolerability for long-term use in girls with Rett syndrome. Rett syndrome is a rare genetic disorder that affects brain development, causing severe physical and intellectual challenges. Girls with classic or possible Rett syndrome, a documented mutation in the MECP2 gene, and stable seizure patterns or no seizures may be suitable candidates. The trial provides trofinetide as a liquid solution, which can be swallowed or administered through a feeding tube. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering a chance to contribute to important advancements in Rett syndrome care.
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. However, if you are taking insulin, you must have stopped it at least 12 weeks before the study starts.
Is there any evidence suggesting that trofinetide is likely to be safe for humans?
Research has shown that trofinetide is generally safe to use. In the LAVENDER study, some patients experienced side effects, occurring in about 5% of cases. These side effects were slightly more common than with a placebo, which contains no active drug.
The FDA has approved trofinetide for treating Rett syndrome in children aged 5 and older, indicating its safety for this age group. Additionally, safety data sheets classify trofinetide as non-hazardous, meaning it poses no significant health risks when used correctly.
Overall, while some side effects occur, trofinetide appears well-tolerated in children with Rett syndrome.12345Why do researchers think this study treatment might be promising?
Trofinetide is unique because it targets the core symptoms of Rett syndrome by aiming to restore normal nerve cell function. Unlike traditional treatments that mainly address symptoms like seizures or mood issues, trofinetide works on the underlying neurological problems by enhancing connections between neurons. This new approach could potentially improve overall brain function and quality of life for young girls with Rett syndrome. Researchers are excited about its potential to offer a more comprehensive treatment compared to the current symptom-focused therapies.
What evidence suggests that trofinetide might be an effective treatment for Rett syndrome?
Research has shown that trofinetide, the investigational treatment in this trial, might help treat Rett syndrome. Long-term studies have found that trofinetide improves symptoms of Rett syndrome without causing new safety concerns. One study noted a clear improvement in the RSBQ score, which measures symptom severity. Other research has shown that caregivers are satisfied with the results of trofinetide treatment. Additionally, studies in animals suggest that trofinetide can improve brain cell connections and communication, which might help with symptoms. These findings support the potential benefits of trofinetide for girls with Rett syndrome.678910
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive oral trofinetide to assess safety and tolerability
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Trofinetide
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Oral dose of trofinetide
Find a Clinic Near You
Who Is Running the Clinical Trial?
ACADIA Pharmaceuticals Inc.
Lead Sponsor
Citations
1.
acadia.com
acadia.com/en-us/media/news-releases/real-world-data-lotus-study-evaluating-long-term-efficacy-andReal World Data from LOTUS Study Evaluating Long-term ...
In animal studies, trofinetide has been shown to increase branching of dendrites and synaptic plasticity signals.
Trofinetide for the treatment of Rett syndrome: Long-term ...
Long-term treatment with trofinetide continued to improve RTT symptoms, without new safety concerns. Caregivers reported satisfaction with trofinetide.
Trofinetide for the treatment of Rett syndrome
Primary efficacy outcomes The mean (s.e.m.) change from baseline to week 12 in the RSBQ total score was −5.1 (0.99) and −1.7 (0.98) in the ...
Results from the phase 2/3 DAFFODIL study of trofinetide ...
Trofinetide was shown to have acceptable tolerability and provided long-term efficacy for up to 78 weeks, and weight-based dosing achieved ...
Real‐world benefits and tolerability of trofinetide for the ...
Results. Overall, 227 caregivers participated in this follow-up. The median trofinetide dose at week 1 was 36.0% of the target dose and ...
6.
file.medchemexpress.com
file.medchemexpress.com/batch_PDF/HY-16757/Trofinetide-SDS-MedChemExpress.pdfSafety Data Sheet
Trofinetide is not a hazardous substance. Avoid inhalation, eye/skin contact, and dust. Store in a cool, well-ventilated area, away from ...
Safety Data Sheet
Trofinetide is not classified as hazardous, with NFPA ratings of 0 for health, fire, and reactivity. No special handling or storage measures ...
Safety & Tolerability
Adverse reactions in at least 5% of patients treated with DAYBUE and at least 2% greater than placebo in the 12-week LAVENDER study were
DAYBUE™ (trofinetide) oral solution - accessdata.fda.gov
The safety and effectiveness of DAYBUE for the treatment of Rett syndrome in pediatric patients 5 years of age and older was established in a randomized, double ...
Safety Profiles of Trofinetide in Pediatric Rett Syndrome ...
This study provides critical real-world safety data on trofinetide use in pediatric Rett syndrome, identifying both expected and emerging AEs.
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