Study of Bionetide for Rett Syndrome
This study is looking into whether an oral medication called Bionetide can help girls and women with Rett syndrome. Researchers want to see if Bionetide is more effective than a placebo (an inactive substance) in improving behavior and communication. You might be able to join if you are a female between 5 and 20 years old, weigh at least 12 kg, can take liquid medication by mouth or G-tube (gastrostomy tube), and have classic Rett syndrome with a confirmed MECP2 gene mutation. The study will measure changes in behavior using a questionnaire and how much your condition improves overall after 12 weeks. The study aims to enroll 210 participants.
- Study design
- This is an interventional study comparing Bionetide to a placebo. It plans to include 210 participants.
- What's involved
- Participants will take either Bionetide or a placebo solution twice daily for 12 weeks. The amount of medication is based on your weight.
- Compensation
- Not stated in the trial record.
- Follow-up
- The study measures outcomes at 12 weeks of treatment.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
At a glance
Conditions
Where it's being run
18 sites across 14 statesStudy leadership
- Lloyd L Tran, PhD · STUDY_DIRECTOR · Biomed Industries, Inc.
- David Nguyen, PhD · STUDY_DIRECTOR · Biomed Industries, Inc.
- Zung V Tran, PhD · STUDY_DIRECTOR · Biomed Industries, Inc.
Who to contact
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What this trial measures
- Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12Baseline and Week 12
The RSBQ is a 45-item caregiver-completed rating scale that includes 45 items, 39 of them grouped into 8 subscales, whose ratings reflect the severity and frequency of symptoms. Items are rated as 0 (not true), 1 (somewhat or sometimes true), or 2 (very true). The 8 subscales are general mood, breathing problems, hand behavior, face movements, body rocking/expressionless face, night-time behaviors, fear/anxiety, and walking/standing. Scores for item 31 are reversed in the calculation of the total score. The total score ranges from 0 to 90 and is calculated as the sum of the item scores. Higher scores mean worse behavior.
- Clinical Global Impression-Improvement (CGI-I) Score at Week 1212 Weeks Treatment Duration
To rate how much the subject's illness has improved or worsened relative to a baseline state, a 7-point scale is used from 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse, 7=very much worse.