Rett SyndromePhase 2/3NCT06849973
To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome
A Phase 2/3 study testing Drug: Bionetide in people with Rett Syndrome, run by Biomed Industries, Inc. at 16 sites in 2 countries.
Trial summary
To study the efficacy \& safety of oral Bionetide versus placebo in girls and women with Rett syndrome Read more about this study
- Who is it for?
- Ages 5 to 20 years, women only
- Conditions the study is for
- Rett Syndrome
- What is being tested?
- Drug: Bionetide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 187 participants
- Where
- 16 sitesin 2 countries
- Study dates
- 15 Mar 2024to 28 Jan 2025
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 20 years
- Women only
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Show all 9Show fewer
- 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
Who cannot join
- 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
- Additional inclusion/exclusion criteria apply. Patients will be evaluated at screening to ensure that all criteria for study participation are met. Patients may be excluded from the study based on these assessments (and specifically, if it is determined that their baseline health and condition do not meet all pre-specified entry criteria).
Trial locations and contacts
16 sites in 2 countries.
16 sites in 2 countries
United States12 sites
Biomed Testing Facility #BIO-05-80042
Aurora, Colorado, 80042
Maryland Locations Biomed Testing Facility #BIO-7-21205
Baltimore, Maryland, 21205
Biomed Testing Facility #BIO-8-02115
Boston, Massachusetts, 02115
Biomed Testing Facility #BIO-06-60612
Chicago, Illinois, 60612
Biomed Testing Facility #BIO-10-44195
Cleveland, Ohio, 44195
Biomed Testing Facility #BIO-11-77030
Houston, Texas, 77030
Biomed Testing Facility # BIO-04-92093
La Jolla, California, 92093
Biomed Testing Facility # BIO-03-90095
Los Angeles, California, 90095
Biomed Testing Facility # BIO-02-95817
Sacramento, California, 95817
Biomed Testing Facility # BIO-01-94104
San Francisco, California, 94104
Biomed Testing Facility #BIO-12-98105
Seattle, Washington, 98105
Biomed Testing Facility #BIO-9-10467
The Bronx, New York, 10467
Australia4 sites
Biomed Research Unit-BIO-16-NSW
Camperdown, New South Wales, 2050
Biomed Research Unit-BIO-13-VIC-3084 Heidelberg West, Victoria, Australia, 3084
Heidelberg West, Victoria, 3084
Biomed Research Unit- BIO-14-VIC 3010
Parkville, Victoria, 3010
Biomed Research Unit-BIO-15
Sydney, New South Wales, 2060
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Phase 2/ Phase 3 Study of NA-921 (Bionetide) for the Treatment of Girls and Women with Rett Syndrome
To study the efficacy \& safety of oral Bionetide versus placebo in girls and women with Rett syndrome
Read the detailed description
A Randomized, Double-Blind, Placebo-Controlled, Phase 2/3 Study of NA-921 (Bionetide) for the Treatment of Girls and Women with Rett Syndrome
Primary Objective • To investigate the efficacy and safety of treatment with oral NA-921 (Bionetide) versus placebo in girls and women with Rett syndrome
Key Secondary Objective • To investigate the efficacy of treatment with oral NA-921 (Bionetide) versus placebo on ability to communicate in girls and women with Rett syndrome
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)
- Conditions
- Rett Syndrome
- Being tested
- Drug: Bionetide, Placebo
- Keywords
- Rett syndrome, Mental Retardation, X-Linked, Genetic Diseases, X-Linked
- Medical subject headings
- Rett Syndrome, X-Linked Intellectual Disability, Genetic Diseases, X-Linked
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Drug: BionetideDrug
Also called NA-921
NA-921 (Bionetide) solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
PlaceboDrug
Also called NA-921 placebo
NA-921 (Bionetide) placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
| Study group | Role | What they receive |
|---|---|---|
| Experimental: Drug - NA-921 (Bionetide) | Experimental | NA-921 (Bionetide) solution of 20-40 mL based on the subject's weight at Basel Receives: Drug: Drug: Bionetide |
| Placebo Comparator: Placebo | Placebo comparator | Bionetide placebo solution of 20-40 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube) Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 12 Weeks Treatment Duration
Secondary outcomes (2)
Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social)
Standardized screening scale for assessing communication and pre-linguistic skills in young children 12-24 months and can be used with older children with developmental delay. The CSBS-DP includes a suite of three separate measures: The Infant-Toddler Checklist, a follow-up Caregiver Questionnaire and a Behavior Sample. In this study only the Infant-Toddler (CSBS-DP-IT) Checklist was used. The CSBS-DP-IT Checklist is a 24-item rating scale and each item is scored using a three-level rating of frequency: "not yet", "sometimes" and "often". The CSBS-DP-IT Social Composite score the range was 0 to 26 and a higher score represented a worse outcome. Three composite scores can be calculated: 1) Social Composite; 2) Speech Composite; 3) Symbolic Composite.
Measured: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC)
Clinical assessment of the subject's ability to communicate their choices or preferences, which can include the use of nonverbal means such as eye contact or gestures. The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment.
Measured: 12 Weeks Treatment Duration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2025
- Study start15 Mar 2024
- Primary completion22 Nov 2024
- Study completion28 Jan 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rett SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 187 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biomed Industries, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Lloyd L. Tran, PhD, Study director, Biomed Industries, Inc.David Nguyen, PhD, Study director, Biomed Industries, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06849973
- Sponsor's protocol ID
- BIO-2024-R01
- Status last verified
- Feb 2025
- First submitted
- 24 Feb 2025
- Last updated
- 27 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: We will decide whether to share IPD at a later date
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Phase 2/ Phase 3 Study of NA-921 (Bionetide) for the Treatment of Girls and Women with Rett Syndrome
- ClinicalTrials.gov ID
- NCT06849973
- Acronym / sponsor ID
- BIO-2024-R01
- Conditions
- Rett Syndrome
- Keywords
- Rett syndrome, Mental Retardation, X-Linked, Genetic Diseases, X-Linked
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 187 participants (actual)
- Lead sponsor
- Biomed Industries, Inc.Industry (pharma, biotech, devices)
- Study start
- 15 Mar 2024 (actual)
- Primary completion
- 22 Nov 2024 (actual)
- Study completion
- 28 Jan 2025 (actual)
- First posted
- 27 Feb 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To study the efficacy \& safety of oral Bionetide versus placebo in girls and women with Rett syndrome
Detailed description
A Randomized, Double-Blind, Placebo-Controlled, Phase 2/3 Study of NA-921 (Bionetide) for the Treatment of Girls and Women with Rett Syndrome
Primary Objective • To investigate the efficacy and safety of treatment with oral NA-921 (Bionetide) versus placebo in girls and women with Rett syndrome
Key Secondary Objective • To investigate the efficacy of treatment with oral NA-921 (Bionetide) versus placebo on ability to communicate in girls and women with Rett syndrome
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)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Experimental: Drug - NA-921 (Bionetide) | Experimental | Drug: Drug: BionetideNA-921 (Bionetide) solution of 20-40 mL based on the subject's weight at Basel |
| Placebo Comparator: Placebo | Placebo comparator | Drug: PlaceboBionetide placebo solution of 20-40 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube) |
Who may qualify
- Ages
- 5 to 20 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Drug: BionetideAlso: NA-921NA-921 (Bionetide) solution administered based on the subject's weight at Baseline, twice daily for 12 weeks | Drug | Experimental: Drug - NA-921 (Bionetide) |
| PlaceboAlso: NA-921 placeboNA-921 (Bionetide) placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks | Drug | Placebo Comparator: Placebo |
Primary outcome
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.
2 secondary outcomes
Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social)
Time frame: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC)
Time frame: 12 Weeks Treatment Duration
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2025.
- Individual participant dataSharing planned: Undecided. We will decide whether to share IPD at a later date
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lloyd L. Tran, PhD study director
Biomed Industries, Inc.
David Nguyen, PhD study director
Biomed Industries, Inc.
16 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Drug: Bionetide in people with Rett Syndrome, run by Biomed Industries, Inc. at 16 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12 (time frame: Baseline and Week 12). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Feb 2025.