~29 spots leftby Apr 2026

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

Recruiting at20 trial locations
Age: < 65
Sex: Female
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Waitlist Available
Sponsor: ACADIA Pharmaceuticals Inc.
Pivotal Trial (Near Approval)
Prior Safety Data

Trial Summary

What is the purpose of this trial?

To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome

Research Team

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
See 6 more

Treatment Details

Interventions

  • Placebo (Other)
  • Trofinetide (Other)
Participant Groups
2Treatment groups
Experimental Treatment
Placebo Group
Group I: Drug - TrofinetideExperimental Treatment1 Intervention
Trofinetide solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
Group II: PlaceboPlacebo Group1 Intervention
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)

Find a Clinic Near You

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)