To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome

March 24, 2026 updated by: Biomed Industries, Inc.

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Phase 3 Study of Bionetide for the Treatment of Girls and Women With Rett Syndrome

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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)

Study Type

Interventional

Enrollment (Estimated)

210

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Australia
    • New South Wales
      • Camperdown, New South Wales, Australia, 2050
        • Recruiting
        • Biomed Research Unit-BIO-23-NSW 2050
        • Contact:
        • Contact:
    • Queensland
      • South Brisbane, Queensland, Australia, 4101
        • Recruiting
        • Biomed Research Unit-BIO-24-NSW-4101
        • Contact:
        • Contact:
    • Victoria
      • Heidelberg West, Victoria, Australia, 3084
        • Recruiting
        • Biomed Research Unit-BIO-21-VIC-3084
        • Contact:
        • Contact:
      • Parkville, Victoria, Australia, 3010
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85012
        • Recruiting
        • Biomed Testing Facility # BIO-01-85012
        • Contact:
        • Contact:
    • California
      • La Jolla, California, United States, 92093
      • Los Angeles, California, United States, 90045
      • Sacramento, California, United States, 95817
      • San Francisco, California, United States, 94104
        • Recruiting
        • Biomed Testing Facility # BIO-02-94104
        • Contact:
        • Contact:
    • Colorado
      • Aurora, Colorado, United States, 80042
    • Florida
      • Tampa, Florida, United States, 33606
    • Illinois
      • Chicago, Illinois, United States, 60612
    • Maryland
      • Baltimore, Maryland, United States, 21205
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
    • New York
      • The Bronx, New York, United States, 10467
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
    • Texas
      • Houston, Texas, United States, 77030
    • Washington
      • Seattle, Washington, United States, 98105

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

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 at a location to which study drug can be delivered 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Bionetide
Experimental: Bionetide Bionetide solution of 20-40 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
Drug: Bionetide
Bionetide solution of 20-40 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
Other Names:
  • NA-921
Placebo Comparator: Placebo
Placebo: Bionetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Drug: Placebo

Placebo

• Bionetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks

Other Names:
  • Placebo of Bionedtide
  • NA-921 placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
12 Weeks Treatment Duration
Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12
Time Frame: 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.
Baseline and Week 12

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score
Time Frame: 12 Weeks Treatment Duration
The Impact of Childhood Neurologic Disability (ICND) 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 ICND score the range of 0 to 132 and a higher score represents a worse outcome.
12 Weeks Treatment Duration
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.
12 Weeks Treatment Duration
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 sit, stand, and ambulate (e.g., walking, running, and climbing stairs). The assessment was made on an 8-point Likert scale (0 to 7), with 0 denoting normal functioning and 7 as the most severe impairment.
12 Weeks Treatment Duration
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.
12 Weeks Treatment Duration
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.
12 Weeks Treatment Duration
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.
12 Weeks Treatment Duration
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 Rett Syndrome Caregiver Burden Inventory (RTT-CBI) 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. The RTT-CBI the range is 0 to 96 and a higher score represents a worse outcome.
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)
Time Frame: 12 Weeks Treatment Duration
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").
12 Weeks Treatment Duration
Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental
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 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.
12 Weeks Treatment Duration

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Biomed Industries, Inc.

Collaborators

Bioneurals Ltd

Investigators

  • Study Director: Lloyd L Tran, PhD, Biomed Industries, Inc.
  • Study Director: David Nguyen, PhD, Biomed Industries, Inc.
  • Study Director: Zung V Tran, PhD, Biomed Industries, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

September 30, 2026

Primary Completion (Estimated)

September 30, 2027

Study Completion (Estimated)

December 30, 2027

Study Registration Dates

First Submitted

February 17, 2025

First Submitted That Met QC Criteria

February 18, 2025

First Posted (Actual)

February 21, 2025

Study Record Updates

Last Update Posted (Actual)

March 27, 2026

Last Update Submitted That Met QC Criteria

March 24, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BIO-2025R02

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

We do not plan to make individual participant data (IPD) available to other researchers

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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