To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females W… (NCT06840496) | Clinical Trial Compass
RecruitingPhase 3
To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
United States210 participantsStarted 2026-09-30
Plain-language 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
Who can participate
Age range5 Years – 20 Years
SexFEMALE
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Inclusion criteria
✓. 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
Exclusion criteria
✕. 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
What they're measuring
1
Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12
Timeframe: Baseline and Week 12
2
Clinical Global Impression-Improvement (CGI-I) Score at Week 12