Rett Syndrome
Conditions
Keywords
Rett syndrome, Mental Retardation, X-Linked, Genetic Diseases, X-Linked
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)
Interventions
DRUGDrug: Bionetide
NA-921 (Bionetide) solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
DRUGPlacebo
NA-921 (Bionetide) placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Sponsors
Biomed Industries, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
FEMALE
Age
5 Years to 20 Years
Healthy volunteers
No
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/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social) | 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 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. |
Countries
Australia, United States
Outcome results
None listed