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Recruiting NCT06840496

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

Phase III Interventional Rett Syndrome

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Bionetide, Placebo.
Who it may be relevant to
Registry conditions: Rett Syndrome. Basic parameters: 5 years — 20 years · Female.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Australia
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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

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)

Interventions

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

Primary outcome measures

  • Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12 [Time frame: Baseline and Week 12]
  • Clinical Global Impression-Improvement (CGI-I) Score at Week 12 [Time frame: 12 Weeks Treatment Duration]
Secondary outcome measures (9)
  • Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental [Time frame: 12 Weeks Treatment Duration]
  • Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score [Time frame: 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]
  • 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]
  • Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC) [Time frame: 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]
  • Change From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S) [Time frame: 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]
  • 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]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

United States · 14 centers
  • Biomed Testing Facility # BIO-01-85012 — Phoenix
  • Biomed Testing Facility # BIO-05-92093 — La Jolla
  • Biomed Testing Facility # BIO-04-90095 — Los Angeles
  • Biomed Testing Facility # BIO-03-95817 — Sacramento
  • Biomed Testing Facility # BIO-02-94104 — San Francisco
  • Biomed Testing Facility #BIO-06-80042 — Aurora
  • Biomed Testing Facility #BIO-07-33606 — Tampa
  • Biomed Testing Facility #BIO-08-60612 — Chicago
  • … and 6 more centers
Australia · 4 centers
  • Biomed Research Unit-BIO-23-NSW 2050 — Camperdown
  • Biomed Research Unit-BIO-24-NSW-4101 — South Brisbane
  • Biomed Research Unit-BIO-21-VIC-3084 — Heidelberg West
  • Biomed Research Unit- BIO-22-VIC 3010 — Parkville

Identifiers

NCT: NCT06840496 · BIO-2025R02

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗