Clinical trial NCT06840496: To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
Official title: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Phase 3 Study of Bionetide for the Treatment of Girls and Women With Rett Syndrome
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Rett Syndrome
Keywords: Rett syndrome,
Genetic Diseases, X-Linked
Lead sponsor: Biomed Industries, Inc.
Sponsor class: INDUSTRY
First posted: 2025-02-21
Enrollment: 210
Sex: FEMALE
Minimum age (years): 5
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Parallel Assignment
Primary purpose: TREATMENT

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

Detailed description: 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

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

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
timeFrame: 12 Weeks Treatment Duration
measure: Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score
timeFrame: 12 Weeks Treatment Duration
measure: Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF)
timeFrame: 12 Weeks Treatment Duration
measure: Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB)
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
measure: Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM)
timeFrame: 12 Weeks Treatment Duration
measure: Change From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S)
timeFrame: 12 Weeks Treatment Duration
measure: Change From Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24)
timeFrame: 12 Weeks Treatment Duration
measure: Change From Baseline to Week 12 in Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND)
timeFrame: 12 Weeks Treatment Duration
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Biomed Testing Facility # BIO-01-85012
state: Arizona
city: La Jolla
country: United States
facility: Biomed Testing Facility # BIO-05-92093
state: California
city: Los Angeles
country: United States
facility: Biomed Testing Facility # BIO-04-90095
state: California
city: Sacramento
country: United States
facility: Biomed Testing Facility # BIO-03-95817
state: California
city: San Francisco
country: United States
facility: Biomed Testing Facility # BIO-02-94104
state: California
city: Aurora
country: United States
facility: Biomed Testing Facility #BIO-06-80042
state: Colorado
city: Tampa
country: United States
facility: Biomed Testing Facility #BIO-07-33606
state: Florida
city: Chicago
country: United States
facility: Biomed Testing Facility #BIO-08-60612
state: Illinois
city: Baltimore
country: United States
facility: Maryland Locations Biomed Testing Facility #BIO-9-21205
state: Maryland
city: Boston
country: United States
facility: Biomed Testing Facility #BIO-10-02115
state: Massachusetts
city: Saint Paul
country: United States
facility: Minnesota Locations Biomed Testing Facility #BIO-11-55101
state: Minnesota
city: Saint Louis
country: United States
facility: Missouri Locations Biomed Testing Facility #BIO-12-63110
state: Missouri
city: Bronx
country: United States
facility: Biomed Testing Facility #BIO-13-10467
state: New York
city: Chapel Hill
country: United States
facility: Biomed Testing Facility #BIO-14-27599
state: North Carolina
city: Cincinnati
country: United States
facility: Biomed Testing Facility #BIO-15-45229
state: Ohio
city: Cleveland
country: United States
facility: Biomed Testing Facility #BIO-16-44195
state: Ohio
city: Philadelphia
country: United States
facility: Biomed Testing Facility #BIO-17-19104
state: Pennsylvania
city: Nashville
country: United States
facility: Biomed Testing Facility #BIO-18-37232
state: Tennessee
city: Houston
country: United States
facility: Biomed Testing Facility #BIO-19-77030
state: Texas
city: Seattle
country: United States
facility: Biomed Testing Facility #BIO-20-98105
state: Washington
city: Camperdown
country: Australia
facility: Biomed Research Unit-BIO-23-NSW 2050
state: New South Wales
city: South Brisbane
country: Australia
facility: Biomed Research Unit-BIO-24-NSW-4101
state: Queensland
city: Heidelberg West
country: Australia
facility: Biomed Research Unit-BIO-21-VIC-3084
state: Victoria
city: Parkville
country: Australia
facility: Biomed Research Unit- BIO-22-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
affiliation: Biomed Industries, Inc.
name: Zung V Tran, PhD
role: STUDY_DIRECTOR
References: