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Trial record 1 of 3 for:    trofinetide | rett
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Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LILAC™)

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04279314
Recruitment Status : Recruiting
First Posted : February 21, 2020
Last Update Posted : May 19, 2020
Sponsor:
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 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 : 180 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A 40-Week, Open-label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
Actual Study Start Date : January 29, 2020
Estimated Primary Completion Date : October 2022
Estimated Study Completion Date : October 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rett Syndrome

Arm Intervention/treatment
Experimental: Trofinetide Drug: Trofinetide
Trofinetide solution of 30-60 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 long-term treatment with oral trofinetide [ Time Frame: 40 Weeks Treatment Duration ]
    Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), withdrawals due to AEs, potentially clinically important changes in other safety assessments


Secondary Outcome Measures :
  1. Rett Syndrome Behaviour Questionnaire (RSBQ) total score - change from Baseline to Week 40 [ Time Frame: 40 Weeks Treatment Duration ]
    The scale includes 45 items, 39 of them grouped into 8 subscales, whose ratings reflect the severity and frequency of symptoms. Items are rated as "0" (not true), "1" (somewhat or sometimes true), or "2" (very true). The 8 subscales include general mood, breathing problems, hand behavior, face movements, body rocking/expressionless face, night-time behaviors, fear/anxiety, and walking/standing.

  2. Clinical Global Impression-Improvement (CGI-I) score at Week 40 [ Time Frame: 40 Weeks Treatment Duration ]
    To rate how much the subject's illness has improved or worsened relative to a baseline state, a 7-point scale is used from 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse, 7=very much worse.

  3. Change from Baseline to Week 40 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social) [ Time Frame: 40 Weeks Treatment Duration ]
    Standardized screening scale for assessing communication and pre-linguistic skills in young children 12-24 months and can be used with older children with developmental delay. The CSBS-DP includes a suite of three separate measures: The Infant-Toddler Checklist, a follow-up Caregiver Questionnaire and a Behavior Sample. In this study only the Infant-Toddler (CSBS-DP-IT) Checklist will be used. The CSBS-DP-IT Checklist is a 24-item rating scale and each item is scored using a three-level rating of frequency: "not yet", "sometimes" and "often". Three composite scores assessing 7 skill areas can be calculated: 1) Social Composite (including Emotion and Eye Gaze, Communication Rate and Function, and Gestures); 2) Speech Composite (including Sounds and Words); 3) Symbolic Composite (including Understanding and Object Use).

  4. Change from Baseline to Week 40 in Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND) [ Time Frame: 40 Weeks Treatment Duration ]
    The scale evaluates the effect of four conditions or health problems on 11 aspects of a child's or family's life as "A lot", "Some", "A little", "Not at all", or "Does not apply". The four conditions or health problems are 1) inattentiveness, impulsivity, or mood, 2) ability to think and remember, 3) neurologic or physical limitations, and 4) epilepsy. The overall quality of life of the subject is also rated by responding to the following: "Please rate your child's overall 'Quality of Life' on the scale below. Choose the number which you feel is best and circle it." The choices range from 1 ("Poor") to 6 ("Excellent").

  5. Change from Baseline to Week 40 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF) [ Time Frame: 40 Weeks Treatment Duration ]
    Clinical assessment of the subject's ability to use their hands for functional purposes (such as reaching for and grasping objects, self-feeding, or drawing). The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment.

  6. Change from Baseline to Week 40 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB) [ Time Frame: 40 Weeks Treatment Duration ]
    Clinical assessment of the subject's ability to sit, stand, and ambulate (e.g., walking, running, climbing stairs). The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment.

  7. Change from Baseline to Week 40 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC) [ Time Frame: 40 Weeks Treatment Duration ]
    Clinical assessment of the subject's ability to communicate their choices or preferences, which can include the use of nonverbal means such as eye contact or gestures. The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment.

  8. Change from Baseline to Week 40 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM) [ Time Frame: 40 Weeks Treatment Duration ]
    Clinical assessment of the subject's ability to communicate verbally (e.g., words and phrases). The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment.

  9. Change from Baseline to Week 40 in Clinical Global Impression-Severity (CGI-S) [ Time Frame: 40 Weeks Treatment Duration ]
    A 7 point scale that rates the severity of the subject's illness at the time of assessment, relative to the clinician's experience with subjects who have the same diagnosis. A subject is assessed on severity of illness at the time of rating: 1=normal, not at all ill; 2=borderline ill; 3=mildly ill; 4=moderately ill; 5=markedly ill; 6=severely ill; or 7= extremely ill.

  10. Change from Baseline to Week 40 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (items 1-24) [ Time Frame: 40 Weeks Treatment Duration ]
    The scale is intended to directly address caregiver burden and indirectly assess the significance of treatment effects on function in the context of activities of daily living. Ratings are on a 5-point Likert scale including: 0-never; 1-rarely; 2-sometimes; 3-frequently and 4-nearly always. As in the original Caregiver Burden Inventory, the RTT-CBI has 24 negatively worded items (items 1 through 24) yielding a total score up to 96.

  11. Change from Baseline to Week 40 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score [ Time Frame: 40 Weeks Treatment Duration ]
    The scale evaluates the effect of four conditions or health problems on 11 aspects of a child's or family's life as "A lot", "Some", "A little", "Not at all", or "Does not apply". The four conditions or health problems are 1) inattentiveness, impulsivity, or mood, 2) ability to think and remember, 3) neurologic or physical limitations, and 4) epilepsy. The overall quality of life of the subject is also rated by responding to the following: "Please rate your child's overall 'Quality of Life' on the scale below. Choose the number which you feel is best and circle it." The choices range from 1 ("Poor") to 6 ("Excellent").



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 21 Years   (Child, Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Has completed the Week 12/End-of-treatment visit of the antecedent study, Study ACP-2566-003
  2. Met all entry criteria for the antecedent study
  3. May benefit from long-term treatment with open-label trofinetide in the judgment of the Investigator
  4. Can still swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
  5. The subject's caregiver has sufficient language skills to complete the caregiver assessments in the language in which the study assessments are provided

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) or has major surgery planned during the study
  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 a QTcF interval of >450 ms on the Baseline ECG performed before the first dose of trofinetide is given in the present study
  8. Has developed a clinically significant ECG finding during the antecedent study

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): NCT04279314


Contacts
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Contact: Kelly O'Rourke-Kosko 442-232-2001 rettsyndromestudies@precisionformedicine.com

Locations
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United States, Alabama
University of Alabama at Birmingham Recruiting
Birmingham, Alabama, United States, 35223
United States, Arizona
Translational Genomics Research Institute (TGen) Recruiting
Phoenix, Arizona, United States, 85012
United States, California
University of California, San Diego Recruiting
La Jolla, California, United States, 92093
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
United States, Illinois
Rush University Medical Center Recruiting
Chicago, Illinois, United States, 60612
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
United States, Minnesota
Gillette Children's Specialty Healthcare Recruiting
Saint Paul, Minnesota, United States, 55101
United States, Missouri
Washington University School of Medicine, St. Louis Children's Hospital Recruiting
Saint Louis, Missouri, United States, 63110
United States, Texas
Texas Children's Hospital Recruiting
Houston, Texas, United States, 77030
Sponsors and Collaborators
ACADIA Pharmaceuticals Inc.
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Responsible Party: ACADIA Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT04279314    
Other Study ID Numbers: ACP-2566-004
First Posted: February 21, 2020    Key Record Dates
Last Update Posted: May 19, 2020
Last Verified: May 2020
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