To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome
- Conditions
- Rett Syndrome
- Interventions
- Drug: BionetideDrug: Placebo
- Registration Number
- NCT06840496
- Lead Sponsor
- Biomed Industries, Inc.
- 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)
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Female
- Target Recruitment
- 210
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Bionetide 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 Placebo Placebo: Bionetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
- Primary Outcome Measures
Name Time Method Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12 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 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.
- Secondary Outcome Measures
Name Time Method Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental 12 Weeks Treatment Duration 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.
Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score 12 Weeks Treatment Duration The Impact of Childhood Neurologic Disability (ICND) scale evaluates the effect of four conditions or health problems on 11 aspects of a child's or family's life as "A lot", "Some", "A little", "Not at all", or "Does not apply". The four conditions or health problems are 1) inattentiveness, impulsivity, or mood, 2) ability to think and remember, 3) neurologic or physical limitations, and 4) epilepsy. The ICND score the range of 0 to 132 and a higher score represents a worse outcome.
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF) 12 Weeks Treatment Duration Clinical assessment of the subject's ability to use their hands for functional purposes (such as reaching for and grasping objects, self-feeding, or drawing). The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment.
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB) 12 Weeks Treatment Duration Clinical assessment of the subject's ability to sit, stand, and ambulate (e.g., walking, running, and climbing stairs). The assessment was made on an 8-point Likert scale (0 to 7), with 0 denoting normal functioning and 7 as the most severe impairment.
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC) 12 Weeks Treatment Duration 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.
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM) 12 Weeks Treatment Duration Clinical assessment of the subject's ability to communicate verbally (e.g. words and phrases). The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment.
Change From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S) 12 Weeks Treatment Duration A 7 point scale that rates the severity of the subject's illness at the time of assessment, relative to the clinician's experience with subjects who have the same diagnosis. A subject is assessed on severity of illness at the time of rating: 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill.
Change From Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24) 12 Weeks Treatment Duration The Rett Syndrome Caregiver Burden Inventory (RTT-CBI) scale is intended to directly address caregiver burden and indirectly assess the significance of treatment effects on function in the context of activities of daily living. Ratings are on a 5-point Likert scale including: 0-never; 1-rarely; 2-sometimes; 3-frequently and 4-nearly always. As in the original Caregiver Burden Inventory, the RTT-CBI has 24 negatively worded items (items 1 through 24) yielding a total score up to 96. The RTT-CBI the range is 0 to 96 and a higher score represents a worse outcome.
Change From Baseline to Week 12 in Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND) 12 Weeks Treatment Duration The overall quality of life of the subject is also rated by responding to the following: "Please rate your child's overall 'Quality of Life' on the scale below. Choose the number which you feel is best and circle it." The choices range from 1 ("Poor") to 6 ("Excellent").
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Trial Locations
- Locations (24)
Biomed Testing Facility # BIO-01-85012
🇺🇸Phoenix, Arizona, United States
Biomed Testing Facility # BIO-05-92093
🇺🇸La Jolla, California, United States
Biomed Testing Facility # BIO-04-90095
🇺🇸Los Angeles, California, United States
Biomed Testing Facility # BIO-03-95817
🇺🇸Sacramento, California, United States
Biomed Testing Facility # BIO-02-94104
🇺🇸San Francisco, California, United States
Biomed Testing Facility #BIO-06-80042
🇺🇸Aurora, Colorado, United States
Biomed Testing Facility #BIO-07-33606
🇺🇸Tampa, Florida, United States
Biomed Testing Facility #BIO-08-60612
🇺🇸Chicago, Illinois, United States
Maryland Locations Biomed Testing Facility #BIO-9-21205
🇺🇸Baltimore, Maryland, United States
Biomed Testing Facility #BIO-10-02115
🇺🇸Boston, Massachusetts, United States
Minnesota Locations Biomed Testing Facility #BIO-11-55101
🇺🇸Saint Paul, Minnesota, United States
Missouri Locations Biomed Testing Facility #BIO-12-63110
🇺🇸Saint Louis, Missouri, United States
Biomed Testing Facility #BIO-13-10467
🇺🇸Bronx, New York, United States
Biomed Testing Facility #BIO-14-27599
🇺🇸Chapel Hill, North Carolina, United States
Biomed Testing Facility #BIO-15-45229
🇺🇸Cincinnati, Ohio, United States
Biomed Testing Facility #BIO-16-44195
🇺🇸Cleveland, Ohio, United States
Biomed Testing Facility #BIO-17-19104
🇺🇸Philadelphia, Pennsylvania, United States
Biomed Testing Facility #BIO-18-37232
🇺🇸Nashville, Tennessee, United States
Biomed Testing Facility #BIO-19-77030
🇺🇸Houston, Texas, United States
Biomed Testing Facility #BIO-20-98105
🇺🇸Seattle, Washington, United States
Biomed Research Unit-BIO-23-NSW 2050
🇦🇺Camperdown, New South Wales, Australia
Biomed Research Unit-BIO-24-NSW-4101
🇦🇺South Brisbane, Queensland, Australia
Biomed Research Unit-BIO-21-VIC-3084
🇦🇺Heidelberg West, Victoria, Australia
Biomed Research Unit- BIO-22-VIC 3010
🇦🇺Parkville, Victoria, Australia