Clinical trial NCT06849973: To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Rett Syndrome
Keywords: Rett syndrome
Mental Retardation, X-Linked
Genetic Diseases, X-Linked
Lead sponsor: Biomed Industries, Inc.
Sponsor class: INDUSTRY
First posted: 2025-02-27
Enrollment: 187
Sex: FEMALE
Minimum age (years): 5
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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)

Eligibility criteria: INCLUSION CRITERIA:

* 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

EXCLUSION CRITERIA :

* 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).

Primary outcomes:
measure: Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12
timeFrame: Baseline and Week 12
measure: Clinical Global Impression-Improvement (CGI-I) Score at Week 12
timeFrame: 12 Weeks Treatment Duration
Secondary outcomes:
measure: Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social)
timeFrame: 12 Weeks Treatment Duration
measure: Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC)
timeFrame: 12 Weeks Treatment Duration
Other outcomes:
Locations:
city: La Jolla
country: United States
facility: Biomed Testing Facility # BIO-04-92093
state: California
city: Los Angeles
country: United States
facility: Biomed Testing Facility # BIO-03-90095
state: California
city: Sacramento
country: United States
facility: Biomed Testing Facility # BIO-02-95817
state: California
city: San Francisco
country: United States
facility: Biomed Testing Facility # BIO-01-94104
state: California
city: Aurora
country: United States
facility: Biomed Testing Facility #BIO-05-80042
state: Colorado
city: Chicago
country: United States
facility: Biomed Testing Facility #BIO-06-60612
state: Illinois
city: Baltimore
country: United States
facility: Maryland Locations Biomed Testing Facility #BIO-7-21205
state: Maryland
city: Boston
country: United States
facility: Biomed Testing Facility #BIO-8-02115
state: Massachusetts
city: Bronx
country: United States
facility: Biomed Testing Facility #BIO-9-10467
state: New York
city: Cleveland
country: United States
facility: Biomed Testing Facility #BIO-10-44195
state: Ohio
city: Houston
country: United States
facility: Biomed Testing Facility #BIO-11-77030
state: Texas
city: Seattle
country: United States
facility: Biomed Testing Facility #BIO-12-98105
state: Washington
city: Camperdown
country: Australia
facility: Biomed Research Unit-BIO-16-NSW
state: New South Wales
city: Sydney
country: Australia
facility: Biomed Research Unit-BIO-15
state: New South Wales
city: Heidelberg West
country: Australia
facility: Biomed Research Unit-BIO-13-VIC-3084 Heidelberg West, Victoria, Australia, 3084
state: Victoria
city: Parkville
country: Australia
facility: Biomed Research Unit- BIO-14-VIC 3010
state: Victoria
Study officials:
affiliation: Biomed Industries, Inc.
name: Lloyd L. Tran, PhD
role: STUDY_DIRECTOR
affiliation: Biomed Industries, Inc.
name: David Nguyen, PhD
role: STUDY_DIRECTOR
References: