Phase 3 Study of Oral Bionetide Compared to Placebo for Treating Girls and Women with Rett Syndrome Aged 5 to 20 Years

What this Trial Is About

Researchers are evaluating the effects of oral Bionetide compared to placebo in girls and women with Rett syndrome, a genetic neurological disorder. This phase 3 randomized, double-blind study focuses on the treatments impact on behavior and communication abilities over a 12-week period. The study aims to provide insights into how Bionetide may influence symptoms and quality of life in this specific population. Participants receive either Bionetide solution, dosed between 20 to 40 mL based on body weight, or a placebo solution, both administered twice daily by mouth or gastrostomy tube. The treatment period lasts 12 weeks, during which participants are closely monitored. The study includes a placebo-controlled design to compare the effects of Bionetide against no active treatment under similar conditions. During the study, participants and their caregivers complete various assessments including the Rett Syndrome Behavior Questionnaire and Clinical Global Impression scales to measure behavioral changes and overall improvement. Additional evaluations focus on communication skills and motor functions. Safety and treatment adherence are monitored throughout, with the study concluding at week 12 to review all collected data on efficacy and impact.

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