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Trofinetide for Rett Syndrome (DAFFODIL™ Trial)

Phase 2 & 3

Waitlist Available

Research Sponsored by ACADIA Pharmaceuticals Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up approximately 24 months treatment duration

Awards & highlights

DAFFODIL™ Trial Summary

This trial will study the safety and tolerability of trofinetide, given orally, in girls with Rett syndrome.

Eligible Conditions

Rett Syndrome

DAFFODIL™ Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ approximately 24 months treatment duration

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~approximately 24 months treatment duration

This trial's timeline: 3 weeks for screening, Varies for treatment, and approximately 24 months treatment duration for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Apparent terminal elimination half-life (t½)

Area under the plasma concentration-time curve (AUC)

Maximum (peak) observed drug concentration (Cmax)

+2 more

Side effects data

From 2021 Phase 3 trial • 187 Patients • NCT04181723

81%

Diarrhoea

27%

Vomiting

Pyrexia

Seizure

Irritability

Decreased appetite

Bronchiolitis

COVID-19 pneumonia

Bacteraemia

Urinary tract infection

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

Drug - Trofinetide

DAFFODIL™ Trial Design

1Treatment groups

Experimental Treatment

Group I: Drug - trofinetideExperimental Treatment1 Intervention

Oral dose of trofinetide

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Trofinetide

2020

Completed Phase 3

~360

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

ACADIA Pharmaceuticals Inc.Lead Sponsor

46 Previous Clinical Trials

10,925 Total Patients Enrolled

3 Trials studying Rett Syndrome

419 Patients Enrolled for Rett Syndrome

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Could you please explain the age limit for this clinical trial?

"This particular clinical trial is for children aged 2 to 5, but there are many other options available: 386 trials for patients younger than 18 and 1028 trials for those older than 65."

Answered by AI

Have there been other similar tests done before this one?

"There are 2 ongoing studies concerning Trofinetide in 21 cities and 1 nation. The research began in 2020 with a Phase 3 clinical trial involving 153 individuals, which was sponsored by ACADIA Pharmaceuticals Inc. 9 more trials have concluded since then."

Answered by AI

What is the precedent for Trofinetide's efficacy?

"At this current moment, there are two ongoing trials for Trofinetide. These studies are both in Phase 3 and 28 different locations across the world are conducting research on Trofinetide."

Answered by AI

How many test subjects are being administered the treatment?

"This study is not presently looking for new participants. The clinical trial was originally posted on 9/22/2021 and was last edited on 5/4/2022. However, there are other active trials that might be of interest. There are currently 1446 clinical trials actively enrolling participants with rett syndrome and 2 trials for Trofinetide that have open recruitment policies."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome

2021. "An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04988867.