Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
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: No Intervention.
- Кому может быть актуально
- Состояния в реестре: Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn. Базовые параметры: 7 мес. — 15 мес. · 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
- Великобритания
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Обзор
This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudinal natural history in enrolled but not dosed participants who also participated in a parent protocol.
Подробное описание
This is a 14.5 year LTFU protocol to assess long term safety and clinical response to IP as well as to capture natural disease history of participants not dosed with IP in a parent protocol. No IP is provided as part of this protocol. Participants will remain on standard of care medication as indicated and prescribed by their physicians.
Вмешательства
- Другое No Intervention
No Intervention
Первичные конечные точки
- AE and SAE (incidence, severity, seriousness, and relatedness) [Срок оценки: Over 14.5 years post dosing with ECUR-506]
- Change from baseline over 14.5 years post infusion in length [Срок оценки: Over 14.5 years post dosing with ECUR-506]
- Change from baseline over 14.5 years post infusion in weight [Срок оценки: Over 14.5 years post dosing with ECUR-506]
- Urinalysis (Dip Stick) Evaluations [Срок оценки: Over 14.5 years post dosing with ECUR-506]
Вторичные конечные точки (12)
- qPCR measurement to evaluate the clearance of both vectors in blood over time. [Срок оценки: Over 14.5 years post dosing with ECUR-506]
- qPCR measurement to evaluate the clearance of both vectors in saliva over time. [Срок оценки: Over 14.5 years post dosing with ECUR-506]
- qPCR measurement to evaluate the clearance of both vectors in urine over time. [Срок оценки: Over 14.5 years post dosing with ECUR-506]
- qPCR measurement to evaluate the clearance of both vectors in feces over time. [Срок оценки: Over 14.5 years post dosing with ECUR-506]
- Percent Liver Transduction [Срок оценки: Over 14.5 years post dosing with ECUR-506]
- Number of hyperammonemic crises (HAC) [Срок оценки: Over 14.5 years post dosing with ECUR-506]
- Among participants who experience HAC with associated neurological status change and are hospitalized: Asses daily ammonia levels for duration of hospitalization for each event [Срок оценки: Over 14.5 years post dosing with ECUR-506]
- Among participants who experience HAC with associated neurological status change and are hospitalized: Assess duration of hospitalization for each event. [Срок оценки: Over 14.5 years post dosing with ECUR-506]
- Among participants who experience HAC with associated neurological status change and are hospitalized: Assess requirement for ICU care. [Срок оценки: Over 14.5 years post dosing with ECUR-506]
- Number of HAC with the following severities: a. Mild: adjustment of dietary protein intake and oral scavenger medication b. Moderate: cessation of dietary protein intake and initiation of IV scavenger therapy c. Severe: requirement for hemodialysis [Срок оценки: Over 14.5 years post dosing with ECUR-506]
- Scavenger drug dose per body surface area (BSA) [Срок оценки: Over 14.5 years post dosing with ECUR-506]
- Protein allowance g/kg [Срок оценки: Over 14.5 years post dosing with ECUR-506]
Критерии участия
Критерии включения
- Enrolled in an iECURE parent protocol and have either completed or discontinued that protocol
- Participant parent(s)/LAR is willing and able to adhere to the protocol requirements.
- Consent was obtained by the participants parent(s)/LAR (and participant assent, where applicable), prior to any study-related data being collected.
Критерии исключения
1\. Participants who enroll into an interventional drug or gene therapy clinical trial utilizing an IP other than the IP provided in the parent protocol will be excluded from this protocol.
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Модель наблюдения
- Другое
Центры проведения
Великобритания · 1 центр
- Great Ormond Street Hospital — London
Идентификаторы
NCT: NCT06805695 · ECUR-LTFU