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Efficacy of Bionetide for treating Rett syndrome in females

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

Phase 3 Interventional Biomed Industries, Inc. · NCT06840496

This study is testing if an oral medication called Bionetide can help improve behavior and communication in girls and young women aged 5 to 20 with Rett syndrome.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment210 (estimated)
Ages5 Years to 20 Years
SexFemale
SponsorBiomed Industries, Inc. Industry-sponsored
Locations18 sites (Phoenix, Arizona and 17 other locations)
Trial IDNCT06840496 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the effectiveness of oral Bionetide compared to a placebo in females aged 5 to 20 with Rett syndrome. The primary focus is on measuring changes in behavior and communication abilities over a 12-week period. Key assessments include the Rett Syndrome Behavior Questionnaire and the Clinical Global Impression-Improvement score. The study aims to provide insights into the potential benefits of Bionetide for improving the quality of life in affected individuals.

Who should consider this trial

Good fit: Ideal candidates are females aged 5 to 20 with a documented MECP2 gene mutation and a stable seizure pattern.

Not a fit: Patients who are male or do not have a confirmed diagnosis of classic Rett syndrome may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve communication and behavioral outcomes for females with Rett syndrome.

How similar studies have performed: While this approach is novel in the context of Rett syndrome, similar studies targeting genetic conditions have shown promising results.

Eligibility criteria

Show full inclusion / exclusion criteria 
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

Where this trial is running

Phoenix, Arizona and 17 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Rett SyndromeRett syndrome,Genetic Diseases, X-Linked
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.