To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
Start Date
9/30/2026
Completion Date
12/30/2027
Summary
Primary Objective • To investigate the efficacy of treatment with oral Bionetide versus placebo in girls and women with Rett syndrome Key Secondary Objective • To investigate the efficacy of treatment with oral Bionetide versus placebo on ability to communicate in girls and women with Rett syndrome
Detailed Description
Co-Primary Endpoints * Rett Syndrome Behavior Questionnaire (RSBQ) total score - Change from Baseline to Week 12 * Clinical Global Impression-Improvement (CGI-I) Score at Week 12 Key Secondary Endpoint Change from Baseline to Week 12 in: • Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist- Social Composite Score (CSBS-DP-IT Social)
Eligibility Criteria
Age Range: 5 years to 20 years
Interventions
Bionetide
Placebo
Conditions
Locations
Biomed Testing Facility # BIO-01-85012
Phoenix, Arizona 85012
United States
Biomed Testing Facility # BIO-05-92093
La Jolla, California 92093
United States
Biomed Testing Facility # BIO-04-90095
Los Angeles, California 90045
United States
Biomed Testing Facility # BIO-03-95817
Sacramento, California 95817
United States
Biomed Testing Facility # BIO-02-94104
San Francisco, California 94104
United States
Biomed Testing Facility #BIO-06-80042
Aurora, Colorado 80042
United States
Biomed Testing Facility #BIO-07-33606
Tampa, Florida 33606
United States
Biomed Testing Facility #BIO-08-60612
Chicago, Illinois 60612
United States
Maryland Locations Biomed Testing Facility #BIO-9-21205
Baltimore, Maryland 21205
United States
Biomed Testing Facility #BIO-10-02115
Boston, Massachusetts 02115
United States
Biomed Testing Facility #BIO-13-10467
The Bronx, New York 10467
United States
Biomed Testing Facility #BIO-17-19104
Philadelphia, Pennsylvania 19104
United States
Biomed Testing Facility #BIO-19-77030
Houston, Texas 77030
United States
Biomed Testing Facility #BIO-20-98105
Seattle, Washington 98105
United States
Biomed Research Unit-BIO-23-NSW 2050
Camperdown, New South Wales 2050
Australia
Biomed Research Unit-BIO-24-NSW-4101
South Brisbane, Queensland 4101
Australia
Biomed Research Unit-BIO-21-VIC-3084
Heidelberg West, Victoria 3084
Australia
Biomed Research Unit- BIO-22-VIC 3010
Parkville, Victoria 3010
Australia