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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT04988867
Recruitment Status : Active, not recruiting
First Posted : August 4, 2021
Last Update Posted : May 6, 2022
Information provided by (Responsible Party):
ACADIA Pharmaceuticals Inc.

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

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Study Type : Interventional  (Clinical Trial)
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 : September 22, 2021
Estimated Primary Completion Date : July 2023
Estimated Study Completion Date : July 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rett Syndrome

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)

Primary Outcome Measures :
  1. Safety and tolerability of treatment with oral trofinetide [ Time Frame: 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

  2. Whole blood concentration of oral trofinetide [ Time Frame: Pre-dose and Weeks 2, 4, 8, and 12 ]
  3. Area under the plasma concentration-time curve (AUC) [ Time Frame: Pre-dose and Weeks 2, 4, 8, and 12 ]
  4. Maximum (peak) observed drug concentration (Cmax) [ Time Frame: Pre-dose and Weeks 2, 4, 8, and 12 ]
  5. Apparent terminal elimination half-life (t½) [ Time Frame: Pre-dose and Weeks 2, 4, 8, and 12 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 5 Years   (Child)
Sexes Eligible for Study:   Female
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No

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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04988867

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United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
United States, Illinois
Rush University Medical Center
Chicago, Illinois, United States, 60612
United States, Massachusetts
Boston Children's Hospital/Harvard Medical School
Boston, Massachusetts, United States, 02115
United States, Minnesota
Gillette Children's Hospital
Saint Paul, Minnesota, United States, 55101
United States, Missouri
Washington University
Saint Louis, Missouri, United States, 63110
United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
Sponsors and Collaborators
ACADIA Pharmaceuticals Inc.
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Responsible Party: ACADIA Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT04988867    
Other Study ID Numbers: ACP-2566-009
First Posted: August 4, 2021    Key Record Dates
Last Update Posted: May 6, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Rett Syndrome
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System