NCT06849973

To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome

A Randomized, Double-Blind, Placebo-Controlled, Phase 2/ Phase 3 Study of NA-921 (Bionetide) for the Treatment of Girls and Women with Rett Syndrome

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 187 (actual)
• Sponsor: Biomed Industries, Inc. · Industry
• Sex: Female
• Age: 5 Years – 20 Years
• Healthy volunteers: Not accepted

Summary

To study the efficacy \& safety of oral Bionetide versus placebo in girls and women with Rett syndrome

Detailed description

A Randomized, Double-Blind, Placebo-Controlled, Phase 2/3 Study of NA-921 (Bionetide) for the Treatment of Girls and Women with Rett Syndrome Primary Objective • To investigate the efficacy and safety of treatment with oral NA-921 (Bionetide) versus placebo in girls and women with Rett syndrome Key Secondary Objective • To investigate the efficacy of treatment with oral NA-921 (Bionetide) versus placebo on ability to communicate in girls and women with Rett syndrome 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	Drug: Bionetide	NA-921 (Bionetide) solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
DRUG	Placebo	NA-921 (Bionetide) placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks

Timeline

• Start date: 2024-03-15
• Primary completion: 2024-11-22
• Completion: 2025-01-28
• First posted: 2025-02-27
• Last updated: 2025-02-27

Locations

16 sites across 2 countries: United States, Australia

Regulatory

• FDA-regulated drug study