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)
Placebo: Placebo
• Bionetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Study 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
Eligibility
Sex
FEMALE
Min age
5 Years
Max age
20 Years
Healthy volunteers
No
INCLUSION CRITERIA
1. Female subjects 5 to 20 years of age, inclusive, at Screening
2. Body weight ≥12 kg at Screening
3. Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
4. Has classic/typical Rett syndrome (RTT)
5. Has a documented disease-causing mutation in the MECP2 gene
6. Has a stable pattern of seizures, or has had no seizures, within 8 weeks of Screening
7. 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.
8. The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
9. Subject and caregiver(s) must reside at a location to which study drug can be delivered and have been at their present residence for at least 3 months prior to Screening
EXCLUSION CRITERIA
1. Has been treated with insulin within 12 weeks of Baseline
2. 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
3. Has a history of, or current, cerebrovascular disease or brain trauma
4. Has significant, uncorrected visual or uncorrected hearing impairment
5. Has a history of, or current, malignancy
6. Has a known history or symptoms of long QT syndrome
Primary outcome measure(s)
Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12 — Baseline 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 12 — 12 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.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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