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

Sponsor

ACADIA Pharmaceuticals Inc. (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT04988867

Collaborator

(none)

Enrollment

Locations

Arm

21.3

Anticipated Duration (Months)

2.1

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Rett Syndrome	Drug: Trofinetide	Phase 2/Phase 3

Study Design

Study Type:

Interventional

Actual Enrollment :

15 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Sep 22, 2021

Anticipated Primary Completion Date :

Jul 1, 2023

Anticipated Study Completion Date :

Jul 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Drug - trofinetide Oral dose of trofinetide	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)

Arm

Intervention/Treatment

Experimental: Drug - trofinetide

Oral dose of trofinetide

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)

Outcome Measures

Primary Outcome Measures

Safety and tolerability of treatment with oral trofinetide [Approximately 24 Months Treatment Duration]
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
Whole blood concentration of oral trofinetide [Pre-dose and Weeks 2, 4, 8, and 12]
Area under the plasma concentration-time curve (AUC) [Pre-dose and Weeks 2, 4, 8, and 12]
Maximum (peak) observed drug concentration (Cmax) [Pre-dose and Weeks 2, 4, 8, and 12]
Apparent terminal elimination half-life (t½) [Pre-dose and Weeks 2, 4, 8, and 12]

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years to 5 Years

Sexes Eligible for Study:

Female

Accepts Healthy Volunteers:

Inclusion Criteria:

Female subject

2 to 4 years of age and body weight ≥9 kg and <20 kg at Screening OR
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:

QTcF interval of >450 ms at Screening or Baseline
History of a risk factor for torsades de pointes (e.g., heart failure or family history of long QT syndrome)
History of clinically significant QT prolongation that is deemed to put the subject at increased risk of clinically significant QT prolongation
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).

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Alabama at Birmingham	Birmingham	Alabama	United States	35233
2	Children's Hospital Colorado	Aurora	Colorado	United States	80045
3	Rush University Medical Center	Chicago	Illinois	United States	60612
4	Boston Children's Hospital/Harvard Medical School	Boston	Massachusetts	United States	02115
5	Gillette Children's Hospital	Saint Paul	Minnesota	United States	55101
6	Washington University	Saint Louis	Missouri	United States	63110
7	Vanderbilt University Medical Center	Nashville	Tennessee	United States	37232

Sponsors and Collaborators

ACADIA Pharmaceuticals Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

ACADIA Pharmaceuticals Inc.

ClinicalTrials.gov Identifier:

NCT04988867

Other Study ID Numbers:

ACP-2566-009

First Posted:

Aug 4, 2021

Last Update Posted:

May 6, 2022

Last Verified:

May 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Rett Syndrome

Syndrome

Study Results

No Results Posted as of May 6, 2022