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Open-Label Extension Study of Trofinetide for Rett Syndrome

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: NCT04776746
Recruitment Status : Enrolling by invitation
First Posted : March 2, 2021
Last Update Posted : December 22, 2021
Sponsor:
Information provided by (Responsible Party):
ACADIA Pharmaceuticals Inc.

Brief Summary:
To investigate the safety and tolerability of continued long-term treatment with oral trofinetide in girls and women with Rett syndrome

Condition or disease Intervention/treatment Phase
Rett Syndrome Drug: trofinetide Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 153 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study of Continuing Treatment With Trofinetide for Rett Syndrome
Actual Study Start Date : November 8, 2020
Estimated Primary Completion Date : January 2023
Estimated Study Completion Date : January 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rett Syndrome

Arm Intervention/treatment
Experimental: Drug - trofinetide
trofinetide oral solution
Drug: trofinetide
Study drug is administered twice a day for up to approximately 32 months. Doses may be taken orally or administered by gastrostomy (G) tube. The subject's assigned dose for this study will be the final dose from the antecedent study (ACP-2566-004).




Primary Outcome Measures :
  1. Percentage of subjects with treatment-emergent adverse events (TAEs), percentage of subjects with serious adverse events (SAEs), and percentage of subjects withdrawals due to adverse events (AEs) [ Time Frame: 32 months ]
  2. Number of subjects with post-Baseline potentially clinically important changes in ECG [ Time Frame: 32 months ]
  3. Percentage of subjects with post-Baseline potentially clinically important changes in ECG [ Time Frame: 32 months ]
  4. Number of subjects with post-Baseline potentially clinically important changes in vital signs [ Time Frame: 32 months ]
  5. Percentage of subjects with post-Baseline potentially clinically important changes in vital signs [ Time Frame: 32 months ]
  6. Number of subjects with post-Baseline potentially clinically important changes in body weight [ Time Frame: 32 months ]
  7. Percentage of subjects with post-Baseline potentially clinically important changes in body weight [ Time Frame: 32 months ]
  8. Number of subjects with post-Baseline potentially clinically important changes in laboratory parameters [ Time Frame: 32 months ]
  9. Percentage of subjects with post-Baseline potentially clinically important changes in laboratory parameters [ Time Frame: 32 months ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   5 Years to 22 Years   (Child, Adult)
Sexes Eligible for Study:   Female
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Has completed the EOT visit of the antecedent trofinetide Study ACP-2566-004 (i.e., has completed 40 weeks)
  2. May benefit from continued treatment with open-label trofinetide in the judgment of the Investigator
  3. Can still swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
  4. The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments

    Childbearing Potential

  5. Subjects of childbearing potential must abstain from sexual activity for the duration of the study and for at least 30 days thereafter. If a subject is sexually active or becomes sexually active during the study, she must use 2 clinically acceptable methods of contraception (e.g., oral, intrauterine device [IUD], diaphragm plus spermicide, injectable, transdermal or implantable contraception) for the duration of the study and for at least 30 days thereafter. Subject must not be pregnant or breastfeeding.

Exclusion Criteria:

  1. Began treatment with growth hormone during the antecedent study
  2. Began treatment with IGF-1 during the antecedent study
  3. Began treatment with insulin during the antecedent study
  4. Has developed a 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)
  5. Subject is judged by the Investigator or the Medical Monitor to be inappropriate for the study due to AEs, medical condition, or noncompliance with investigational product or study procedures in the antecedent study
  6. Has a clinically significant abnormality in vital signs at Baseline
  7. Has an average QTcF interval of >450 ms on the Baseline ECG performed before the first dose of trofinetide is given in the present study (i.e., the ECG performed at the EOT visit of the antecedent study)
  8. Has developed a clinically significant ECG finding during the antecedent study

Additional inclusion/exclusion criteria apply. Patients will be evaluated at screening 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 pre-specified 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): NCT04776746


Locations
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United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
United States, Arizona
Translational Genomics Research Institute (TGen)
Phoenix, Arizona, United States, 85012
United States, California
University of California, San Diego
La Jolla, California, United States, 92093
United States, Colorado
Children's Hospital Colorado Aurora
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 Specialty Healthcare
Saint Paul, Minnesota, United States, 55101
United States, Missouri
Washington University School of Medicine
Saint Louis, Missouri, United States, 63110
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Cleveland Clinic
Cleveland, Ohio, United States, 44195
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
United States, Texas
Texas Children's Hospital
Houston, Texas, United States, 77030
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Sponsors and Collaborators
ACADIA Pharmaceuticals Inc.
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Responsible Party: ACADIA Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT04776746    
Other Study ID Numbers: ACP-2566-005
First Posted: March 2, 2021    Key Record Dates
Last Update Posted: December 22, 2021
Last Verified: November 2021
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
Syndrome
Disease
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