Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 2 of 3 for:    trofinetide | rett

Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)

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: NCT04181723
Recruitment Status : Recruiting
First Posted : November 29, 2019
Last Update Posted : February 25, 2020
Sponsor:
Information provided by (Responsible Party):
ACADIA Pharmaceuticals Inc.

Brief Summary:
To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome

Condition or disease Intervention/treatment Phase
Rett Syndrome Drug: Trofinetide Other: Placebo Phase 3

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 184 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
Actual Study Start Date : November 6, 2019
Estimated Primary Completion Date : September 2021
Estimated Study Completion Date : October 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rett Syndrome

Arm Intervention/treatment
Experimental: Drug - Trofinetide
Trofinetide solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
Drug: Trofinetide
Trofinetide solution administered based on the subject's weight at Baseline, twice daily for 12 weeks

Placebo Comparator: Placebo
Trofinetide placebo solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
Other: Placebo
Trofinetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks




Primary Outcome Measures :
  1. Rett Syndrome Behaviour Questionnaire (RSBQ) total score - Change from Baseline to Week 12 [ Time Frame: 12 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 12 [ Time Frame: 12 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.


Secondary Outcome Measures :
  1. Change from Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social) [ Time Frame: 12 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).

  2. Change from Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF) [ Time Frame: 12 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.

  3. Change from Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB) [ Time Frame: 12 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.

  4. Change from Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S) [ Time Frame: 12 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.

  5. Change from Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (items 1-24) [ Time Frame: 12 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.

  6. Change from Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score [ Time Frame: 12 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").

  7. Change from Baseline to Week 12 in Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND) [ Time Frame: 12 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").

  8. Change from Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC) [ Time Frame: 12 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.

  9. Change from Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM) [ Time Frame: 12 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.



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.


Layout table for eligibility information
Ages Eligible for Study:   5 Years to 20 Years   (Child, Adult)
Sexes Eligible for Study:   Female
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Female subjects 5 to 20 years of age, inclusive, at Screening
  2. Body weight ≥12 kg at Screening
  3. Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
  4. Has classic/typical Rett syndrome (RTT)
  5. Has a documented disease-causing mutation in the MECP2 gene
  6. Has a stable pattern of seizures, or has had no seizures, within 8 weeks of Screening
  7. Subjects of childbearing potential must abstain from sexual activity for the duration of the study and for at least 30 days thereafter. OR must agree to use acceptable methods of contraception. Subject must not be pregnant or breastfeeding.

Exclusion Criteria:

  1. Has been treated with insulin within 12 weeks of Baseline
  2. 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
  3. Has a history of, or current, cerebrovascular disease or brain trauma
  4. Has significant, uncorrected visual or uncorrected hearing impairment
  5. Has a history of, or current, malignancy
  6. Has a known history or symptoms of long QT syndrome

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): NCT04181723


Contacts
Layout table for location contacts
Contact: Christine LaRock 442-232-2001 ACADIA-ACP-2566-003@precisionformedicine.com

Locations
Layout table for location information
United States, Alabama
University of Alabama at Birmingham Recruiting
Birmingham, Alabama, United States, 35294
United States, Arizona
Translational Gemomics 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, 80042
United States, Illinois
Rush University Children's Hospital Recruiting
Chicago, Illinois, United States, 60612
United States, Massachusetts
Boston Children's Hospital Harvard Medical School 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 Recruiting
Saint Louis, Missouri, United States, 63110
United States, New York
Montefiore Medical Center, Children's Hospital at Montefiore Recruiting
Bronx, New York, United States, 10467
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Cleveland Clinic Recruiting
Cleveland, Ohio, United States, 44195
United States, South Carolina
Greenwood Genetic Center Recruiting
Greenwood, South Carolina, United States, 29646
United States, Tennessee
Vanderbilt University Medical Center Recruiting
Nashville, Tennessee, United States, 37232
United States, Texas
Texas Children's Hospital Recruiting
Houston, Texas, United States, 77030
Sponsors and Collaborators
ACADIA Pharmaceuticals Inc.
Layout table for additonal information
Responsible Party: ACADIA Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT04181723    
Other Study ID Numbers: ACP-2566-003
First Posted: November 29, 2019    Key Record Dates
Last Update Posted: February 25, 2020
Last Verified: February 2020

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
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