To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome

Phase 2

Completed

• Conditions: Rett Syndrome
• Interventions: Drug: Placebo

• Registration Number: NCT06849973
• Lead Sponsor: Biomed Industries, Inc.

Brief Summary

To study the efficacy \& safety of oral Bionetide versus placebo in girls and women with Rett syndrome

Detailed Description

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 Timeline

• Study Start: 2024-03-15
• Primary Completion: 2024-11-22
• Study Completion: 2025-01-28
• Status Verified: 2025-02
• Study First Submitted: 2025-02-24
• Study First Submitted QC: 2025-02-26
• Last Update Submitted: 2025-02-26
• Study First Posted: 2025-02-27
• Last Update Posted: 2025-02-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Female
• Target Recruitment: 187

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo Comparator: Placebo	Placebo	Bionetide placebo solution of 20-40 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)

Primary Outcome Measures

Name	Time	Method
Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12	Baseline and 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.
Clinical Global Impression-Improvement (CGI-I) Score at Week 12	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

Name	Time	Method
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	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.
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC)	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.

Trial Locations

Locations (16)

Biomed Testing Facility # BIO-02-95817; 🇺🇸 Sacramento, California, United States

Biomed Testing Facility #BIO-06-60612; 🇺🇸 Chicago, Illinois, United States

Biomed Testing Facility # BIO-04-92093; 🇺🇸 La Jolla, California, United States

Biomed Testing Facility # BIO-03-90095; 🇺🇸 Los Angeles, California, United States

Biomed Testing Facility # BIO-01-94104; 🇺🇸 San Francisco, California, United States

Biomed Testing Facility #BIO-05-80042; 🇺🇸 Aurora, Colorado, 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; 🇺🇸 Bronx, New York, United States

Biomed Testing Facility #BIO-10-44195; 🇺🇸 Cleveland, Ohio, United States

Biomed Testing Facility #BIO-11-77030; 🇺🇸 Houston, Texas, United States

Biomed Testing Facility #BIO-12-98105; 🇺🇸 Seattle, Washington, United States

Biomed Research Unit-BIO-16-NSW; 🇦🇺 Camperdown, New South Wales, Australia

Biomed Research Unit-BIO-15; 🇦🇺 Sydney, New South Wales, Australia

Biomed Research Unit-BIO-13-VIC-3084 Heidelberg West, Victoria, Australia, 3084; 🇦🇺 Heidelberg West, Victoria, Australia

Biomed Research Unit- BIO-14-VIC 3010; 🇦🇺 Parkville, Victoria, Australia