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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06840496




Registration number
NCT06840496
Ethics application status
Date submitted
17/02/2025
Date registered
25/03/2025

Titles & IDs
Public title
To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
Scientific title
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Phase 3 Study of Bionetide for the Treatment of Girls and Women With Rett Syndrome
Secondary ID [1] 0 0
BIO-2025R02
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Rett Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Bionetide
Treatment: Drugs - Placebo

Experimental: Bionetide - 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


Treatment: Drugs: 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)

Treatment: Drugs: Placebo
Placebo

• Bionetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12
Timepoint [1] 0 0
Baseline and Week 12
Primary outcome [2] 0 0
Clinical Global Impression-Improvement (CGI-I) Score at Week 12
Timepoint [2] 0 0
12 Weeks Treatment Duration
Secondary outcome [1] 0 0
Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental
Timepoint [1] 0 0
12 Weeks Treatment Duration
Secondary outcome [2] 0 0
Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score
Timepoint [2] 0 0
12 Weeks Treatment Duration
Secondary outcome [3] 0 0
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF)
Timepoint [3] 0 0
12 Weeks Treatment Duration
Secondary outcome [4] 0 0
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB)
Timepoint [4] 0 0
12 Weeks Treatment Duration
Secondary outcome [5] 0 0
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC)
Timepoint [5] 0 0
12 Weeks Treatment Duration
Secondary outcome [6] 0 0
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM)
Timepoint [6] 0 0
12 Weeks Treatment Duration
Secondary outcome [7] 0 0
Change From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S)
Timepoint [7] 0 0
12 Weeks Treatment Duration
Secondary outcome [8] 0 0
Change From Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24)
Timepoint [8] 0 0
12 Weeks Treatment Duration
Secondary outcome [9] 0 0
Change From Baseline to Week 12 in Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND)
Timepoint [9] 0 0
12 Weeks Treatment Duration

Eligibility
Key inclusion criteria
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
Minimum age
5 Years
Maximum age
20 Years
Sex
Females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
15/06/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
15/03/2026
Actual
Sample size
Target
210
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Biomed Research Unit-BIO-23-NSW 2050 - Camperdown
Recruitment hospital [2] 0 0
Biomed Research Unit-BIO-24-NSW-4101 - South Brisbane
Recruitment hospital [3] 0 0
Biomed Research Unit-BIO-21-VIC-3084 - Heidelberg West
Recruitment hospital [4] 0 0
Biomed Research Unit- BIO-22-VIC 3010 - Parkville
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
3084 - Heidelberg West
Recruitment postcode(s) [4] 0 0
3010 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Minnesota
Country [9] 0 0
United States of America
State/province [9] 0 0
Missouri
Country [10] 0 0
United States of America
State/province [10] 0 0
New York
Country [11] 0 0
United States of America
State/province [11] 0 0
North Carolina
Country [12] 0 0
United States of America
State/province [12] 0 0
Ohio
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
Tennessee
Country [15] 0 0
United States of America
State/province [15] 0 0
Texas
Country [16] 0 0
United States of America
State/province [16] 0 0
Washington

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Biomed Industries, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Lloyd L Tran, PhD
Address 0 0
Biomed Industries, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Lloyd L Tran, PhD
Address 0 0
Country 0 0
Phone 0 0
1-800-824-5135
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06840496