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

Rett Syndrome Clinical Trial

— DAFFODIL™  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04988867
• Other study ID #: ACP-2566-009
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: September 22, 2021
• Est. completion date: May 31, 2023

Study information

• Verified date: June 2023
• Source: ACADIA Pharmaceuticals Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls with Rett syndrome

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: May 31, 2023
• Est. primary completion date: May 31, 2023
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 2 Years to 5 Years

Eligibility

Inclusion Criteria:

• Female subject

1. 2 to 4 years of age and body weight =9 kg and <20 kg at Screening OR

2. 5 years of age and body weight =9 kg and <12 kg at Screening

• Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube

• The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments

• Has classic/typical Rett syndrome (RTT) or possible RTT according to the Rett Syndrome Diagnostic Criteria

• Has a documented disease-causing mutation in the MECP2 gene

• Has a stable pattern of seizures, or has had no seizures, within 8 weeks prior to Screening

• 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 4 weeks 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 any of the following:

1. QTcF interval of >450 ms at Screening or Baseline

2. History of a risk factor for torsades de pointes (e.g., heart failure or family history of long QT syndrome)

3. History of clinically significant QT prolongation that is deemed to put the subject at increased risk of clinically significant QT prolongation

4. Other clinically significant finding on ECG at Screening or Baseline

Additional inclusion/exclusion criteria apply. Patients will be evaluated at baseline 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 prespecified entry criteria).

Related Conditions & MeSH terms

• Rett Syndrome
• Syndrome

Intervention

Drug:
• Trofinetide
Trofinetide solution of 10-30 mL based on subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)

Locations

Country	Name	City	State
United States	Children's Hospital Colorado	Aurora	Colorado
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	Boston Children's Hospital/Harvard Medical School	Boston	Massachusetts
United States	Rush University Medical Center	Chicago	Illinois
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	Washington University	Saint Louis	Missouri
United States	Gillette Children's Hospital	Saint Paul	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
ACADIA Pharmaceuticals Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and tolerability of treatment with oral trofinetide	Percentage of subjects with Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), withdrawals due to AEs, potentially clinically important changes in other safety assessments	Approximately 24 Months Treatment Duration
Primary	Whole blood concentration of oral trofinetide		Pre-dose and Weeks 2, 4, 8, and 12
Primary	Area under the plasma concentration-time curve (AUC)		Pre-dose and Weeks 2, 4, 8, and 12
Primary	Maximum (peak) observed drug concentration (Cmax)		Pre-dose and Weeks 2, 4, 8, and 12
Primary	Apparent terminal elimination half-life (t½)		Pre-dose and Weeks 2, 4, 8, and 12