A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP)
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: garetosmab.
- Кому может быть актуально
- Состояния в реестре: Fibrodysplasia Ossificans Progressiva (FOP). Базовые параметры: 2 лет — 18 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
- Список центров уточняется — проверьте первичный протокол.
- 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
Phase 3 Evaluation of the Safety, Pharmacokinetics, and Efficacy of Garetosmab (Anti-Activin A Monoclonal Antibody) in Children and Adolescents With Fibrodysplasia Ossificans Progressiva
Обзор
This study is researching an experimental drug called garetosmab, referred to as "study drug". The study is focused on children and adolescent participants with FOP. The aim of the study is to see how safe, tolerable, and effective the study drug is. The study is looking at several other research questions, including: * What side effects may happen from taking the study drug * How much study drug is in the blood at different times * Whether the body makes antibodies against the study drug (which could make the study drug less effective or could lead to side effects)
Вмешательства
- Препарат garetosmab
Administered per the protocol
Первичные конечные точки
- Occurrence of Treatment-Emergent Adverse Event (TEAEs) [Срок оценки: Baseline to week 28]
- Occurrence of TEAEs [Срок оценки: Baseline to week 56]
- Severity of TEAEs [Срок оценки: Baseline to week 28]
- Severity of TEAEs [Срок оценки: Baseline to week 56]
- Concentrations of functional garetosmab in serum [Срок оценки: Through week 56]
Вторичные конечные точки (12)
- Total volume of new Heterotopic Ossification (HO) lesion [Срок оценки: At week 28 and week 56]
- Number of new HO lesions [Срок оценки: At week 28 and week 56]
- Occurrence of new HO lesions [Срок оценки: At week 28 and week 56]
- Number of clinician-assessed flare-ups [Срок оценки: Through week 28 and week 56]
- Occurrence of clinician-assessed flare-ups [Срок оценки: Through week 28 and week 56]
- Number of patient/caregiver-reported flare-ups [Срок оценки: Through Week 28 and week 56]
- Occurrence of patient/caregiver-reported flare-ups [Срок оценки: Through week 28 and week 56]
- Change from baseline in Tanner puberty scale [Срок оценки: At week 28 and week 56]
- Characteristics of menstrual cycles for female participants who reached menarche [Срок оценки: Over 28 weeks and 56 weeks]
- Height-for-age Z-Scores according to the World Health Organization (WHO) Growth Reference Data for Children [Срок оценки: Through week 56]
- Concentrations of total activin A in serum [Срок оценки: Through week 56]
- Occurence of Anti-Drug Antibody (ADA) to garetosmab [Срок оценки: Through week 56]
Критерии участия
Критерии включения
- For USA participants, age criteria are 4 to < 18 years old, at the time of the administration of the first dose of study intervention. Non-USA participants age criteria are 2 to < 18 years old
- Must have a confirmation of FOP diagnosis, as described in the protocol
- At the time of enrollment, participants must weight:
- Cohort 1 > 30 kg
- Cohort 2 > 30 kg
- Cohort 3 ≤ 30 kg
Критерии исключения
- Cumulative Analog Joint Involvement Scale (CAJIS) score > 19 at the time of screening
- Participant has significant concomitant illness or history of significant illness, as described in the protocol
- Previous history or diagnosis of cancer
- Ongoing significant viral or bacterial illness, within 2 weeks of the first study drug administration
- History of severe respiratory compromise requiring oxygen, respiratory support
- Known history of cerebral vascular malformation
- Participants with a history of severe, non-traumatic bleeding requiring transfusion or hospitalization for hemodynamic compromise
- Participants with a known pre-existing medical history of a bleeding diathesis, as described in the protocol
NOTE: Other Protocol-defined Inclusion/Exclusion Criteria Apply
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Распределение
- Нерандомизированное
- Модель
- Одна группа
- Маскирование
- Открытое
- Основная цель
- Лечение
Центры проведения
Список центров уточняется — проверьте первичный протокол.
Идентификаторы
NCT: NCT07559513 · R2477-FOP-2413 · 2024-518415-19-01