NCT06840496

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

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 210 (estimated)
• Sponsor: Biomed Industries, Inc. · Industry
• Sex: Female
• Age: 5 Years – 20 Years
• Healthy volunteers: Not accepted

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

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)

Conditions

• Rett Syndrome

Interventions

Type	Name	Description
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

Timeline

• Start date: 2026-09-30
• Primary completion: 2027-09-30
• Completion: 2027-12-30
• First posted: 2025-02-21
• Last updated: 2026-03-27

Locations

18 sites across 2 countries: United States, Australia

Regulatory

• FDA-regulated drug study