A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Phase 3 Study of Bionetide for the Treatment of Girls and Women With Rett Syndrome
Biomed Industries, Inc.
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
Description
Co-Primary Endpoints * Rett Syndrome Behavior Questionnaire (RSBQ) total score - Change from Baseline to Week 12 * Clinical Global Impression-Improvement (CGI-I) Score at Week 12 Key Secondary Endpoint Change from Baseline to Week 12 in: • Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist- Social Composite Score (CSBS-DP-IT Social)
Eligibility
- Age range
- 5–20 years
- Sex
- Female
- 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 con…
Interventions
- DrugBionetide
Bionetide solution of 20-40 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
- DrugPlacebo
Placebo • Bionetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Locations (18)
- Biomed Testing Facility # BIO-01-85012Phoenix, Arizona
- Biomed Testing Facility # BIO-05-92093La Jolla, California
- Biomed Testing Facility # BIO-04-90095Los Angeles, California
- Biomed Testing Facility # BIO-03-95817Sacramento, California
- Biomed Testing Facility # BIO-02-94104San Francisco, California
- Biomed Testing Facility #BIO-06-80042Aurora, Colorado