A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Phase 3 Study of Bionetide for the Treatment of Girls and Women With Rett Syndrome
Status: Recruiting
Location: See all (24) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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
Participation Requirements
Sex: Female
Minimum Age: 5
Maximum Age: 20
Healthy Volunteers: f
View:
• 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
• 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
Experimental: 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_comparator: Placebo
Placebo: Bionetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks