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

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome

Sponsors

Lead Sponsor: ACADIA Pharmaceuticals Inc.

Source ACADIA Pharmaceuticals Inc.
Brief Summary

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

Overall Status Recruiting
Start Date November 6, 2019
Completion Date October 2021
Primary Completion Date September 2021
Phase Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Rett Syndrome Behaviour Questionnaire (RSBQ) total score - Change from Baseline to Week 12 12 Weeks Treatment Duration
Clinical Global Impression-Improvement (CGI-I) Score at Week 12 12 Weeks Treatment Duration
Secondary Outcome
Measure Time Frame
Change from Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social) 12 Weeks Treatment Duration
Change from Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF) 12 Weeks Treatment Duration
Change from Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB) 12 Weeks Treatment Duration
Change from Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S) 12 Weeks Treatment Duration
Change from Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (items 1-24) 12 Weeks Treatment Duration
Change from Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score 12 Weeks Treatment Duration
Change from Baseline to Week 12 in Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND) 12 Weeks Treatment Duration
Change from Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC) 12 Weeks Treatment Duration
Change from Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM) 12 Weeks Treatment Duration
Enrollment 184
Condition
Intervention

Intervention Type: Drug

Intervention Name: Trofinetide

Description: Trofinetide solution administered based on the subject's weight at Baseline, twice daily for 12 weeks

Arm Group Label: Drug - Trofinetide

Intervention Type: Other

Intervention Name: Placebo

Description: Trofinetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks

Arm Group Label: Placebo

Eligibility

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.

8. The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments

9. Subject and caregiver(s) must reside in the 48 contiguous states and have been at their present residence for at least 3 months prior to Screening

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).

Gender: Female

Gender Based: Yes

Minimum Age: 5 Years

Maximum Age: 20 Years

Healthy Volunteers: No

Overall Contact

Last Name: Kelly O'Rourke-Kosko

Phone: 442-232-2001

Email: [email protected]

Location
Facility: Status:
University of Alabama at Birmingham | Birmingham, Alabama, 35294, United States Recruiting
Translational Gemomics Research Institute (TGen) | Phoenix, Arizona, 85012, United States Recruiting
University of California, San Diego | La Jolla, California, 92093, United States Recruiting
Children's Hospital Colorado | Aurora, Colorado, 80042, United States Recruiting
Rush University Children's Hospital | Chicago, Illinois, 60612, United States Recruiting
Boston Children's Hospital Harvard Medical School | Boston, Massachusetts, 02115, United States Recruiting
Gillette Children's Specialty Healthcare | Saint Paul, Minnesota, 55101, United States Recruiting
Washington University School of Medicine | Saint Louis, Missouri, 63110, United States Recruiting
Montefiore Medical Center, Children's Hospital at Montefiore | Bronx, New York, 10467, United States Recruiting
Cincinnati Children's Hospital Medical Center | Cincinnati, Ohio, 45229, United States Recruiting
Cleveland Clinic | Cleveland, Ohio, 44195, United States Recruiting
Greenwood Genetic Center | Greenwood, South Carolina, 29646, United States Recruiting
Vanderbilt University Medical Center | Nashville, Tennessee, 37232, United States Recruiting
Texas Children's Hospital | Houston, Texas, 77030, United States Recruiting
Location Countries

United States

Verification Date

July 2020

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Drug - Trofinetide

Type: Experimental

Description: Trofinetide solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)

Label: Placebo

Type: Placebo Comparator

Description: 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)

Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Source: ClinicalTrials.gov