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

(DAFFODIL™ Trial)

Recruiting at 6 trial locations

Overseen By Maile Krumpschmidt

Age: < 18

Sex: Female

Trial Phase: Phase 2 & 3

Sponsor: ACADIA Pharmaceuticals Inc.

Must not be taking: Insulin

Disqualifiers: Cardiovascular, Endocrine, Renal, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 2

Duration: 1.2 years

15 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a medicine called trofinetide, which is taken by mouth, to see if it is safe for extended use in girls with Rett syndrome. The medicine aims to help the brain work better and reduce the symptoms of the disorder.

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.

Eligibility Criteria

Inclusion Criteria

The person who will take care of you during the study speaks English well enough to complete the required assessments.

You have been diagnosed with classic/typical Rett syndrome or possible Rett syndrome based on specific diagnostic criteria.

You are a woman.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral trofinetide to assess safety and tolerability

1.2 years

PK samples at Weeks 2, 4, 8, and 12

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Trofinetide

Participant Groups

Group I: Drug - trofinetide Experimental Treatment 1 Intervention

Oral dose of trofinetide

Who Is Running the Clinical Trial?

ACADIA Pharmaceuticals Inc.

Lead Sponsor

Trials

49

Recruited

11,700+

Founded

1993

Headquarters

San Diego, USA

Known For

Neurological Disorders

Top Products

Nuplazid (pimavanserin), Daybue (trofinetide)