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Completed

NCT Number: NCT06849973

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

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

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Key information

Conditions

Rett Syndrome Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations X-Linked Intellectual Disability

Age range

5 year–20 year

Sex eligibility

Female

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

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

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About this study

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)

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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).

Treatment and study plan

Drug: Bionetide

Drug

NA-921 (Bionetide) solution administered based on the subject's weight at Baseline, twice daily for 12 weeks

Other names: NA-921

Placebo

Drug

NA-921 (Bionetide) placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks

Other names: NA-921 placebo

Primary outcomes

  1. 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.

  2. 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.

Secondary outcomes

  1. Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social)

    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.

  2. 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.

Sponsors and collaborators

Lead sponsor

Biomed Industries, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2024
Study completion
2025
First posted
Feb 27, 2025
Registry last updated
Feb 27, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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