Идёт набор NCT06840496

To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome

Фаза III С лечением Rett Syndrome

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: В протоколе указаны: Bionetide, Placebo.
Кому может быть актуально: Состояния в реестре: Rett Syndrome. Базовые параметры: 5 лет — 20 лет · Женщины.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: США, Австралия
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Phase 3 Study of Bionetide for the Treatment of Girls and Women With Rett Syndrome

Обзор

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

Подробное описание

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)

Вмешательства

Препарат 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)
Препарат Placebo
Placebo • Bionetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks

Первичные конечные точки

Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12 [Срок оценки: Baseline and Week 12]
Clinical Global Impression-Improvement (CGI-I) Score at Week 12 [Срок оценки: 12 Weeks Treatment Duration]

Вторичные конечные точки (9)

Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental [Срок оценки: 12 Weeks Treatment Duration]
Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score [Срок оценки: 12 Weeks Treatment Duration]
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF) [Срок оценки: 12 Weeks Treatment Duration]
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB) [Срок оценки: 12 Weeks Treatment Duration]
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC) [Срок оценки: 12 Weeks Treatment Duration]
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM) [Срок оценки: 12 Weeks Treatment Duration]
Change From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S) [Срок оценки: 12 Weeks Treatment Duration]
Change From Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24) [Срок оценки: 12 Weeks Treatment Duration]
Change From Baseline to Week 12 in Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND) [Срок оценки: 12 Weeks Treatment Duration]

Критерии участия

Критерии включения

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

Критерии исключения

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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет