To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Phase 3 Study of Bionetide for the Treatment of Girls and Women With Rett Syndrome
Biomed Industries, Inc.
210 participants
Sep 30, 2026
INTERVENTIONAL
Conditions
Summary
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
Eligibility
Inclusion Criteria9
- 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 Criteria6
- 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
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Interventions
Bionetide solution of 20-40 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
Placebo • Bionetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Locations(18)
View Full Details on ClinicalTrials.gov
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NCT06840496