NCT06840496 · Biomed Industries, Inc.
To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
What this study is about
Primary Objective • To investigate the effectiveness of treatment with taken by mouth Bionetide versus placebo in girls and women with Rett syndrome Key Secondary Objective • To investigate the effectiveness of treatment with taken by mouth Bionetide versus placebo on ability to communicate in girls and women with Rett syndrome
View original scientific description
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
Interventions
DRUG
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)
DRUG
Placebo
Placebo • Bionetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Primary outcome measures
Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12
Time frame: 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
Time frame: 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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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
- Has been treated with in
Where
- Phoenix, Arizona
- La Jolla, California
- Los Angeles, California
- Sacramento, California
- San Francisco, California
- Aurora, Colorado
- Tampa, Florida
- Chicago, Illinois
- Baltimore, Maryland
- Boston, Massachusetts
- The Bronx, New York
- Philadelphia, Pennsylvania
And 2 more locations — see the full list below.
Collaborators
Bioneurals Ltd
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Mar 27, 2026 · Source of record for eligibility and locations