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

A Randomized, Double-Blind, Placebo-Controlled, Phase 2/ Phase 3 Study of NA-921 (Bionetide) for the Treatment of Girls and Women with Rett Syndrome

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

Study Overview

• Status: Completed
• Conditions: Rett Syndrome
• Intervention / Treatment: Drug: Drug: Bionetide; Drug: Placebo

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 Type: Interventional
• Enrollment (Actual): 187
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Camperdown, New South Wales, Australia, 2050
      • Biomed Research Unit-BIO-16-NSW
    • Sydney, New South Wales, Australia, 2060
      • Biomed Research Unit-BIO-15
  • Victoria
    • Heidelberg West, Victoria, Australia, 3084
      • Biomed Research Unit-BIO-13-VIC-3084 Heidelberg West, Victoria, Australia, 3084
    • Parkville, Victoria, Australia, 3010
      • Biomed Research Unit- BIO-14-VIC 3010
• United States
  • California
    • La Jolla, California, United States, 92093
      • Biomed Testing Facility # BIO-04-92093
    • Los Angeles, California, United States, 90095
      • Biomed Testing Facility # BIO-03-90095
    • Sacramento, California, United States, 95817
      • Biomed Testing Facility # BIO-02-95817
    • San Francisco, California, United States, 94104
      • Biomed Testing Facility # BIO-01-94104
  • Colorado
    • Aurora, Colorado, United States, 80042
      • Biomed Testing Facility #BIO-05-80042
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Biomed Testing Facility #BIO-06-60612
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Maryland Locations Biomed Testing Facility #BIO-7-21205
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Biomed Testing Facility #BIO-8-02115
  • New York
    • Bronx, New York, United States, 10467
      • Biomed Testing Facility #BIO-9-10467
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Biomed Testing Facility #BIO-10-44195
  • Texas
    • Houston, Texas, United States, 77030
      • Biomed Testing Facility #BIO-11-77030
  • Washington
    • Seattle, Washington, United States, 98105
      • Biomed Testing Facility #BIO-12-98105

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

INCLUSION CRITERIA:

• Female subjects 5 to 20 years of age, inclusive, at Screening
• Body weight ≥12 kg at Screening
• Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
• Has classic/typical Rett syndrome (RTT)
• Has a documented disease-causing mutation in the MECP2 gene
• Has a stable pattern of seizures, or has had no seizures, within 8 weeks of Screening
• 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.
• The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
• 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 :

• Has been treated with insulin within 12 weeks of Baseline
• 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
• Has a history of, or current, cerebrovascular disease or brain trauma
• Has significant, uncorrected visual or uncorrected hearing impairment
• Has a history of, or current, malignancy
• 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).

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Experimental: Drug - NA-921 (Bionetide); NA-921 (Bionetide) solution of 20-40 mL based on the subject's weight at Basel	Drug: Drug: Bionetide; NA-921 (Bionetide) solution administered based on the subject's weight at Baseline, twice daily for 12 weeks; Other Names: NA-921
Placebo Comparator: Placebo Comparator: 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)	Drug: Placebo; NA-921 (Bionetide) placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks; Other Names: 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	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	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 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.	12 Weeks Treatment Duration
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.	12 Weeks Treatment Duration

Collaborators and Investigators

• Sponsor: Biomed Industries, Inc.
• Investigators: Study Director: David Nguyen, PhD, Biomed Industries, Inc.; Study Director: Lloyd L. Tran, PhD, Biomed Industries, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): March 15, 2024
• Primary Completion (Actual): November 22, 2024
• Study Completion (Actual): January 28, 2025

Study Registration Dates

• First Submitted: February 24, 2025
• First Submitted That Met QC Criteria: February 26, 2025
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: February 26, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Keywords: Rett syndrome; Genetic Diseases, X-Linked; Mental Retardation, X-Linked
• Additional Relevant MeSH Terms: Neurologic Manifestations; Nervous System Diseases; Pathologic Processes; Genetic Diseases, Inborn; Disease; Neurobehavioral Manifestations; Heredodegenerative Disorders, Nervous System; Mental Retardation, X-Linked; Intellectual Disability; Genetic Diseases, X-Linked; Syndrome; Rett Syndrome
• Other Study ID Numbers: BIO-2024-R01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED
• IPD Plan Description: We will decide whether to share IPD at a later date

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No