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

NCT06840496 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: BIO-2025R02
• Study Type: interventional
• Target: 210
• Locations: 2 countries
• Sites: 18

Brief 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

Conditions

Rett Syndrome

Keywords

Rett syndrome,; Genetic Diseases, X-Linked

Outcome Measures

Primary Outcomes (2)

• Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to 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.

  Baseline and Week 12

• Clinical Global Impression-Improvement (CGI-I) Score at Week 12

  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.

  12 Weeks Treatment Duration

Secondary Outcomes (9)

• Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental

  12 Weeks Treatment Duration

• Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score

  12 Weeks Treatment Duration

• Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF)

  12 Weeks Treatment Duration

• Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB)

  12 Weeks Treatment Duration

• Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC)

  12 Weeks Treatment Duration

Study Arms (2)

Bionetide

EXPERIMENTAL

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)

Drug: Bionetide

Placebo

PLACEBO COMPARATOR

Placebo: Bionetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks

Drug: Placebo

Interventions

Bionetide DRUG

Bionetide solution of 20-40 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)

Also known as: NA-921

Bionetide

Placebo DRUG

Placebo • Bionetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks

Also known as: Placebo of Bionedtide, NA-921 placebo

Placebo

Eligibility Criteria

• Age: 5 Years - 20 Years
• Gender Eligibility Details: 5 Years to 20 Years Female (Child, Adult )
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• Has been treated with insulin within 12 weeks of Baseline
• 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
• Has a history of, or current, cerebrovascular disease or brain trauma
• Has significant, uncorrected visual or uncorrected hearing impairment
• Has a history of, or current, malignancy
• Has a known history or symptoms of long QT syndrome

Sponsors & Collaborators

• Bioneurals Ltd: collaborator
• Biomed Industries, Inc.: lead

Study Sites (18)

Biomed Testing Facility # BIO-01-85012

Phoenix, Arizona, 85012, United States

RECRUITING

Biomed Testing Facility # BIO-05-92093

La Jolla, California, 92093, United States

RECRUITING

Biomed Testing Facility # BIO-04-90095

Los Angeles, California, 90045, United States

RECRUITING

Biomed Testing Facility # BIO-03-95817

Sacramento, California, 95817, United States

RECRUITING

Biomed Testing Facility # BIO-02-94104

San Francisco, California, 94104, United States

RECRUITING

Biomed Testing Facility #BIO-06-80042

Aurora, Colorado, 80042, United States

RECRUITING

Biomed Testing Facility #BIO-07-33606

Tampa, Florida, 33606, United States

RECRUITING

Biomed Testing Facility #BIO-08-60612

Chicago, Illinois, 60612, United States

RECRUITING

Maryland Locations Biomed Testing Facility #BIO-9-21205

Baltimore, Maryland, 21205, United States

RECRUITING

Biomed Testing Facility #BIO-10-02115

Boston, Massachusetts, 02115, United States

RECRUITING

Biomed Testing Facility #BIO-13-10467

The Bronx, New York, 10467, United States

RECRUITING

Biomed Testing Facility #BIO-17-19104

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Biomed Testing Facility #BIO-19-77030

Houston, Texas, 77030, United States

RECRUITING

Biomed Testing Facility #BIO-20-98105

Seattle, Washington, 98105, United States

RECRUITING

Biomed Research Unit-BIO-23-NSW 2050

Camperdown, New South Wales, 2050, Australia

RECRUITING

Biomed Research Unit-BIO-24-NSW-4101

South Brisbane, Queensland, 4101, Australia

RECRUITING

Biomed Research Unit-BIO-21-VIC-3084

Heidelberg West, Victoria, 3084, Australia

RECRUITING

Biomed Research Unit- BIO-22-VIC 3010

Parkville, Victoria, 3010, Australia

RECRUITING

MeSH Terms

Conditions

Rett Syndrome; Genetic Diseases, X-Linked

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System

Study Officials

• Lloyd L Tran, PhD

  Biomed Industries, Inc.

  STUDY DIRECTOR

• David Nguyen, PhD

  Biomed Industries, Inc.

  STUDY DIRECTOR

• Zung V Tran, PhD

  Biomed Industries, Inc.

  STUDY DIRECTOR

Central Study Contacts

David L Nguyen, PhD

CONTACT

1-800-824-5135; research@biomedind.com

Jennifer Thomposn, MS

CONTACT

1-800-824-5135; research@biomedind.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Quadruple Participant/Care Provider/ Investigator/ Outcome Assessor
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Parallel Assignment

Study Record Dates

• First Submitted: February 17, 2025
• First Posted: February 21, 2025
• Study Start (Estimated): September 30, 2026
• Primary Completion (Estimated): September 30, 2027
• Study Completion (Estimated): December 30, 2027
• Last Updated: March 27, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

AU (4); US (14)