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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 : Completed
First Posted : March 2, 2021
Last Update Posted : August 1, 2023
Sponsor:
Information provided by (Responsible Party):
ACADIA Pharmaceuticals Inc.

Study Description
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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

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 78 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
Actual Primary Completion Date : June 30, 2023
Actual Study Completion Date : June 30, 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rett Syndrome

Arms and Interventions
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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).


Outcome Measures
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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 ]

Eligibility Criteria
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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).

Contacts and Locations
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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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Sponsors and Collaborators
ACADIA Pharmaceuticals Inc.
More Information
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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: August 1, 2023
Last Verified: July 2023
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