To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome
NCT ID: NCT06849973
Last Updated: 2025-02-27
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE2/PHASE3
187 participants
INTERVENTIONAL
2024-03-15
2025-01-28
Brief Summary
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Detailed Description
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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
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Experimental: Drug - NA-921 (Bionetide)
NA-921 (Bionetide) solution of 20-40 mL based on the subject's weight at Basel
Drug: Bionetide
NA-921 (Bionetide) solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Placebo Comparator: Placebo
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)
Placebo
NA-921 (Bionetide) placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Interventions
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Drug: Bionetide
NA-921 (Bionetide) solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Placebo
NA-921 (Bionetide) placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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
5 Years
20 Years
FEMALE
No
Sponsors
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Biomed Industries, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Lloyd L. Tran, PhD
Role: STUDY_DIRECTOR
Biomed Industries, Inc.
David Nguyen, PhD
Role: STUDY_DIRECTOR
Biomed Industries, Inc.
Locations
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Biomed Testing Facility # BIO-04-92093
La Jolla, California, United States
Biomed Testing Facility # BIO-03-90095
Los Angeles, California, United States
Biomed Testing Facility # BIO-02-95817
Sacramento, California, United States
Biomed Testing Facility # BIO-01-94104
San Francisco, California, United States
Biomed Testing Facility #BIO-05-80042
Aurora, Colorado, United States
Biomed Testing Facility #BIO-06-60612
Chicago, Illinois, United States
Maryland Locations Biomed Testing Facility #BIO-7-21205
Baltimore, Maryland, United States
Biomed Testing Facility #BIO-8-02115
Boston, Massachusetts, United States
Biomed Testing Facility #BIO-9-10467
The Bronx, New York, United States
Biomed Testing Facility #BIO-10-44195
Cleveland, Ohio, United States
Biomed Testing Facility #BIO-11-77030
Houston, Texas, United States
Biomed Testing Facility #BIO-12-98105
Seattle, Washington, United States
Biomed Research Unit-BIO-16-NSW
Camperdown, New South Wales, Australia
Biomed Research Unit-BIO-15
Sydney, New South Wales, Australia
Biomed Research Unit-BIO-13-VIC-3084 Heidelberg West, Victoria, Australia, 3084
Heidelberg West, Victoria, Australia
Biomed Research Unit- BIO-14-VIC 3010
Parkville, Victoria, Australia
Countries
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Other Identifiers
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BIO-2024-R01
Identifier Type: -
Identifier Source: org_study_id
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