ClinicalTrials.gov record NCT06840496

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
Age5 years old to 20 years old
SexFemale
LocationAustralia, United States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 3 randomized, double-blind, placebo-controlled study compares oral Bionetide with placebo in girls and women with Rett syndrome. Treatment is given twice daily for 12 weeks and outcomes are measured at baseline and Week 12. Main assessments include a caregiver behavior questionnaire and a clinician-rated global improvement score, with additional communication and function measures.

What it is studying. The study tests oral Bionetide (NA-921) versus placebo to see how the two compare on caregiver-rated behavior (RSBQ), clinician-rated global improvement (CGI‑I), and communication measures after 12 weeks.

What the researchers hope to learn. To evaluate whether oral Bionetide changes caregiver-rated behavior, clinician-rated improvement, communication scores, motor and hand function, caregiver burden, and related quality-of-life measures compared with placebo over 12 weeks.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Bionetide
  • Placebo

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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)
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants are randomly assigned to receive either oral Bionetide or matching placebo twice daily for 12 weeks; the liquid medication may be taken by mouth or given via a gastrostomy tube. Assessments are done at baseline and at Week 12 and include caregiver questionnaires (for example the Rett Syndrome Behavior Questionnaire), clinician ratings (including Clinical Global Impression), and a communication checklist (CSBS‑DP‑IT). Study drug is provided based on the participant's weight and the record indicates drug delivery to the participant's residence; caregivers must complete study assessments in English. The registry does not list other specific procedures or visit schedules.

How the study is structured. Participants are randomly assigned to receive either Bionetide or a matching placebo twice daily for 12 weeks. The study is double-blind (neither families nor study staff know assignments). Caregivers complete behavior and communication questionnaires and clinicians perform standard ratings at baseline and Week 12 to compare the two groups.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
  • The listed sites are in Australia, United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Rett syndrome

Clinical trials20 trials listed for Rett syndromeEvery trial the registry lists for Rett syndrome, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Rett syndrome researchLatest monthly update: September 2026. Includes: Seizure add-on drug. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Sep 30, 2026 (estimated)
Primary completion
Sep 30, 2027 (estimated)
Study completion
Dec 30, 2027 (estimated)
Target enrollment
210 participants (estimated)
Last updated on registry
Mar 27, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Biomed Industries, Inc.
Collaborators
  • Bioneurals Ltd

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (18)

Terms used on this page · explanations by MedicalUpdates.ai

Primary Objective
The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
placebo
A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
Secondary Objective
An additional result the study measures alongside the primary outcome, for example quality of life or a laboratory value.
at Screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
study drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
Baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
INCLUSION CRITERIA
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
EXCLUSION CRITERIA
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Endpoints
Something the study measures to judge its results, such as a symptom score, a blood test or survival. Outcomes are grouped as primary, secondary and sometimes exploratory.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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