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NCT06840496PHASE3Recruiting

To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome

Biomed Industries, Inc.

Start Date

9/30/2026

Completion Date

12/30/2027

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

Detailed 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 Criteria

Age Range: 5 years to 20 years

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

Interventions

DRUG

Bionetide

DRUG

Placebo

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Conditions

Rett Syndrome

Locations

Biomed Testing Facility # BIO-01-85012

Phoenix, Arizona 85012

United States

Biomed Testing Facility # BIO-05-92093

La Jolla, California 92093

United States

Biomed Testing Facility # BIO-04-90095

Los Angeles, California 90045

United States

Biomed Testing Facility # BIO-03-95817

Sacramento, California 95817

United States

Biomed Testing Facility # BIO-02-94104

San Francisco, California 94104

United States

Biomed Testing Facility #BIO-06-80042

Aurora, Colorado 80042

United States

Biomed Testing Facility #BIO-07-33606

Tampa, Florida 33606

United States

Biomed Testing Facility #BIO-08-60612

Chicago, Illinois 60612

United States

Maryland Locations Biomed Testing Facility #BIO-9-21205

Baltimore, Maryland 21205

United States

Biomed Testing Facility #BIO-10-02115

Boston, Massachusetts 02115

United States

Biomed Testing Facility #BIO-13-10467

The Bronx, New York 10467

United States

Biomed Testing Facility #BIO-17-19104

Philadelphia, Pennsylvania 19104

United States

Biomed Testing Facility #BIO-19-77030

Houston, Texas 77030

United States

Biomed Testing Facility #BIO-20-98105

Seattle, Washington 98105

United States

Biomed Research Unit-BIO-23-NSW 2050

Camperdown, New South Wales 2050

Australia

Biomed Research Unit-BIO-24-NSW-4101

South Brisbane, Queensland 4101

Australia

Biomed Research Unit-BIO-21-VIC-3084

Heidelberg West, Victoria 3084

Australia

Biomed Research Unit- BIO-22-VIC 3010

Parkville, Victoria 3010

Australia