To study the efficacy \& safety of oral Bionetide versus placebo in girls and women with Rett syndrome
A Randomized, Double-Blind, Placebo-Controlled, Phase 2/3 Study of NA-921 (Bionetide) for the Treatment of Girls and Women with Rett Syndrome Primary Objective • To investigate the efficacy and safety of treatment with oral NA-921 (Bionetide) versus placebo in girls and women with Rett syndrome Key Secondary Objective • To investigate the efficacy of treatment with oral NA-921 (Bionetide) versus placebo on ability to communicate in girls and women with Rett syndrome 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)
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
187
NA-921 (Bionetide) solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
NA-921 (Bionetide) placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Biomed Testing Facility # BIO-04-92093
La Jolla, California, United States
Biomed Testing Facility # BIO-03-90095
Los Angeles, California, United States
Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12
The RSBQ is a 45-item caregiver-completed rating scale that 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 are general mood, breathing problems, hand behavior, face movements, body rocking/expressionless face, night-time behaviors, fear/anxiety, and walking/standing. Scores for item 31 are reversed in the calculation of the total score. The total score ranges from 0 to 90 and is calculated as the sum of the item scores. Higher scores mean worse behavior.
Time frame: Baseline and Week 12
Clinical Global Impression-Improvement (CGI-I) Score at Week 12
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.
Time frame: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social)
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 was 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". The CSBS-DP-IT Social Composite score the range was 0 to 26 and a higher score represented a worse outcome. Three composite scores can be calculated: 1) Social Composite; 2) Speech Composite; 3) Symbolic Composite.
Time frame: 12 Weeks Treatment Duration
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Biomed Testing Facility # BIO-02-95817
Sacramento, California, United States
Biomed Testing Facility # BIO-01-94104
San Francisco, California, United States
Biomed Testing Facility #BIO-05-80042
Aurora, Colorado, United States
Biomed Testing Facility #BIO-06-60612
Chicago, Illinois, United States
Maryland Locations Biomed Testing Facility #BIO-7-21205
Baltimore, Maryland, United States
Biomed Testing Facility #BIO-8-02115
Boston, Massachusetts, United States
Biomed Testing Facility #BIO-9-10467
The Bronx, New York, United States
Biomed Testing Facility #BIO-10-44195
Cleveland, Ohio, United States
...and 6 more locations
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC)
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.
Time frame: 12 Weeks Treatment Duration