Идёт набор NCT07353099

A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma

— Наблюдательное Thrombotic Thrombocytopenic Purpura (TTP)

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: Thrombotic Thrombocytopenic Purpura (TTP). Базовые параметры: Без ограничений · 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: Австрия, Германия
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 Post-Authorization Safety Study (PASS) to Further Evaluate Real-World Safety in Patients With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma

Обзор

Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by a problem in a gene passed down from parents to children, which affects the body's ability to produce a enzyme called ADAMTS13. This enzyme helps to cut down a larger form of protein called von Willebrand Factor (VWF). People with cTTP have low levels of ADAMTS13. Without ADAMTS13, large forms of vWF build up and cause blood clots in small blood vessels. These clots can block blood flow to vital organs, causing serious health problems. Adzynma is a human ADAMTS13 protein made in the laboratory. It works the same way as natural ADAMTS13 does and may provide higher levels of ADAMTS13. The main aim of this study is to learn more about the risk of children and adults with cTTP treated with Adzynma developing antibodies that prevent Adzynma from working properly (called neutralizing antibodies) within 6 months after the first treatment and to understand the risk of allergic reactions within 7 days of the first treatment with Adzynma. Other aims are to better understand how safe treatment with Adzynma is over a longer period of time (called long-term safety) in children and adults with cTTP and to gather information about pregnancies and babies of women who have received Adzynma while pregnant. Only data already available in the medical records of the people who received Adzynma for the treatment of cTTP in normal clinical practice will be reviewed and collected during this study.

Вмешательства

Другое No Intervention
This is a non-interventional study.

Первичные конечные точки

Number of Participants With Hypersensitivity Reactions Following the Index Adzynma Infusions [Срок оценки: Up to 6 months following the index Adzynma infusion]
Number of Participants With Neutralizing Antibodies to ADAMTS13 Following the Index Adzynma Infusions [Срок оценки: Up to 6 months following the index Adzynma infusion]

Вторичные конечные точки (12)

Number of Participants With Long Term Safety Risk of Hypersensitivity Reactions Following the Index Adzynma Infusion [Срок оценки: Up to 6 months of follow-up time from the index Adzynma infusion]
Number of Participants With Risk of Hypersensitivity Reactions After Each Administration of Adzynma Following the Index Infusion [Срок оценки: Up to 7 days after each administration of Adzynma]
Number of Participants With Treatment-emergent Adverse Events (TEAEs) [Срок оценки: From start of the study drug administration up to 5.5 years]
Gestational age at the Time of Infant Birth [Срок оценки: At birth]
Number of Participants With Spontaneous Abortion [Срок оценки: Up to 5.5 years]
Number of Participants With Stillbirth [Срок оценки: Up to 5.5 years]
Number of Participants With Induced Abortion [Срок оценки: Up to 5.5 years]
Number of Participants With Live Birth [Срок оценки: Up to 5.5 years]
Number of Participants With any Pregnancy Related Complications [Срок оценки: Up to 5.5 years]
Number of Participants Categorized According to Gestational age of Greater Than or Equal to (>=) 37 Gestational Weeks and Less Than (<) 37 Gestational Weeks at Birth [Срок оценки: At birth]
Number of Infants Categorized According to Year of Birth [Срок оценки: Up to 5.5 years]
Number of Infants With Normal Birth Weight [Срок оценки: Up to 5.5 years]

Критерии участия

Критерии включения

Is diagnosed with cTTP.
Had received commercially available Adzynma treatment for cTTP within the eligibility period of which there must be a recorded date and dose of at least one Adzynma administration in their medical chart.
Provides a signed informed consent form (informed consent form \[ICF\]; or assent and consent forms, if applicable), in accordance with local ethical and institutional requirements.

Критерии исключения

Has a history or presence of a functional ADAMTS13 inhibitor (that is, neutralizing antibodies to ADAMTS13/rADAMTS13).
Has concurrent use of an investigational drug or is enrolled in another clinical trial at the time of index Adzynma infusion.

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

Здоровые добровольцы: Нет