Pharmacogenomic Contributions to Trihexyphenidyl Biotransformation and Response in Children With Dystonic Cerebral Palsy
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: Trihexyphenidyl.
- Кому может быть актуально
- Состояния в реестре: Pediatric Disorder, Genetic Predisposition, Dystonia, Secondary, Dystonia. Базовые параметры: 5 лет — 17 лет · Все.
- 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
- 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
Pharmacogenomic Contribution to the Biotransformation of Trihexyphenidyl and Development of a Precision Dosing Model for Children With Dystonia and Cerebral Palsy
Обзор
This study looks at how a medicine called trihexyphenidyl works in children with dystonic cerebral palsy. The study aims to understand how trihexyphenidyl is broken down and used in the body of pediatric patients and whether this is impacted by a person's genetics. Information from this study will also be used to design future clinical trials.
Подробное описание
This is a 16-week single-arm nonrandomized pilot study of trihexyphenidyl in children with dystonic cerebral palsy (DCP) to 1) evaluate the pharmacokinetics (PK) of trihexyphenidyl (THP) and variation in PK parameters between CYP2D6 and CYP2C19 genotypes and 2) evaluate the feasibility of a future exposure-controlled clinical trial of THP.
Вмешательства
- Препарат Trihexyphenidyl
6-week dose escalation up to 0.25mg/kg TID, followed by a 9-week maintenance period at this dose
Первичные конечные точки
- Difference in Cmax between CYP2D6 and CYP2C19 phenotype groups [Срок оценки: Baseline]
- Difference in AUC0-n between CYP2D6 and CYP2C19 phenotype groups [Срок оценки: Baseline]
- Difference in AUC0-∞ between CYP2D6 and CYP2C19 phenotype groups [Срок оценки: Baseline]
- Recruitment percentage [Срок оценки: Through study completion, an average of 2 years]
- Retention percentage [Срок оценки: Through study completion, an average of 2 years]
- Dystonia Efficacy Measures Outcome Completion [Срок оценки: Through study completion, an average of 2 years]
Вторичные конечные точки (12)
- Number of participants with at least one adverse event as measured by the Safety Monitoring Uniform Report Form (SMURF) [Срок оценки: Through study completion, an average of 2 years]
- Change from baseline in dystonia duration as measured by the Dyskinesia Impairment Scale, Version 2 (DIS-2) (exploratory) [Срок оценки: Baseline, 16 weeks]
- Change from baseline in dystonia amplitude as measured by the Dyskinesia Impairment Scale (exploratory) [Срок оценки: Baseline, 16 weeks]
- Change from baseline in dystonia as measured by the Quality of Upper Extremity Skills Test (QUEST) (exploratory) [Срок оценки: Baseline, 16 weeks]
- Change in functional impact from baseline as measured by the Dyskinetic Cerebral Palsy Functional Impact Scale (D-FIS) (exploratory) [Срок оценки: Baseline, 16 weeks]
- Change in priority scores in functional impact from baseline as measured by the Dyskinetic Cerebral Palsy Functional Impact Scale (D-FIS) (exploratory) [Срок оценки: Baseline, 16 weeks]
- Change in patient-driven performance from baseline as measured by the Canadian Occupational Performance Measure (exploratory) [Срок оценки: Baseline, 16 weeks]
- Change in patient-driven goal satisfaction from baseline as measured by the Canadian Occupational Performance Measure (exploratory) [Срок оценки: Baseline, 16 weeks]
- Change in caregiver's perspective about their child in 4 domains: health status, comfort, wellbeing, functional abilities, and ease of caregiving from baseline as measured by Caregiver Priorities and Child Health Index of Life with Disabilities [Срок оценки: Baseline, 16 weeks]
- Measure the acceptability of outcome measures at 16 weeks as measured by the Acceptability of Intervention Measure [Срок оценки: 16 weeks]
- Change in disease severity from baseline as measured by the Patient Global Impression of Severity (PGI-S) (exploratory) [Срок оценки: Baseline and 16 weeks]
- Change in overall status as measured by the Patient Global Impression of Change (PGI-C) [Срок оценки: Week 16]
Критерии участия
Критерии включения
- Ages 5-17 years of age
- Diagnosis of cerebral palsy and dystonia causing interference
- Parent/legal guardian of a child with a diagnosis of cerebral palsy and dystonia
- Parent/legal guardian is willing and able to provide informed permission/assent for the study
Критерии исключения
- Previously or currently taking trihexyphenidyl
- Patients turning 18 years of age within the study period (16 weeks from Study Day 1)
- A language barrier for the patient that precludes communication and/or the ability to complete study-related requirements
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Распределение
- Не применимо
- Модель
- Одна группа
- Маскирование
- Открытое
- Основная цель
- Лечение
Центры проведения
США · 1 центр
- Children's Mercy Hospital Kansas City — Kansas City
Идентификаторы
NCT: NCT06554288 · STUDY00003228