Un estudio en niños y adultos con púrpura trombocitopénica trombótica congénita (cTTP) tratados con Adzynma
Un Estudio de Seguridad Postautorización (PASS) para Evaluar Adicionalmente la Seguridad en el Mundo Real en Pacientes con Púrpura Trombocitopénica Trombótica Congénita (PTTc) Tratados con Adzynma
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.
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Tipo de estudio
Inscripción (Estimado)
Contactos y Ubicaciones
Estudio Contacto
- Nombre: Takeda Contact
- Número de teléfono: +1-877-825-3327
- Correo electrónico: medinfoUS@takeda.com
Ubicaciones de estudio
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Bonn, Alemania, 53127
- Reclutamiento
- Universitatsklinikum Bonn
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Contacto:
- Site Contact
- Correo electrónico: heiko.ruehl@ukbonn.de
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Investigador principal:
- Ruehl Heiko
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Cologne, Alemania, 50937
- Aún no reclutando
- Universitaetsklinikum Koeln
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Contacto:
- Site Contact
- Correo electrónico: lucas.kuehne@uk-koeln.de
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Investigador principal:
- Kuehne Lucas
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Jena, Alemania, 7743
- Aún no reclutando
- Universitaetsklinikum Jena
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Contacto:
- Site Contact
- Correo electrónico: karim.kentouche@med.uni-jena.de
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Investigador principal:
- Kentouche Karim
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Leipzig, Alemania, 4103
- Reclutamiento
- Universitaetsklinikum Leipzig
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Contacto:
- Site Contact
- Correo electrónico: maria.weise@medizin.uni-leipzig.de
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Investigador principal:
- Weise Maria
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München, Alemania
- Aún no reclutando
- LMU Klinikum
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Contacto:
- Site Contact
- Correo electrónico: ulf.schoenermarck@med.uni-muenchen.de
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Investigador principal:
- Schoenermarck Ulf
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München, Alemania, 81675
- Aún no reclutando
- Klinikum Rechts der Isar der Technischen Universitaet Muenchen
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Investigador principal:
- Braunisch Matthias
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Contacto:
- Site Contact
- Correo electrónico: matthiaschristoph.braunisch@mri.tum.de
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Vienna, Austria, 1090
- Reclutamiento
- AKH - Medizinische Universitaet Wien
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Contacto:
- Site Contact
- Correo electrónico: johannes.thaler@meduniwien.ac.at
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Investigador principal:
- Johannes Thaler
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
- Niño
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Método de muestreo
Población de estudio
Pediatric and adult participants who have received Adzynma for the treatment of cTTP, either prophylactically or as on-demand therapy for acute episodes-including those who became pregnant during Adzynma treatment.
Descripción
Inclusion criteria:
- 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.
Exclusion criteria:
- 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.
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Modelos observacionales: COHORT
- Perspectivas temporales: RETROSPECTIVE
Cohortes e Intervenciones
Grupo / Cohorte |
Intervención / Tratamiento |
|---|---|
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All Participants
Data of participants who have received Adzynma for the treatment of cTTP will be collected retrospectively from medical records for approximately 5 years. |
This is a non-interventional study. |
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Number of Participants With Hypersensitivity Reactions Following the Index Adzynma Infusions
Periodo de tiempo: Up to 6 months following the index Adzynma infusion
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Index infusion is defined as the first Adzynma infusion date recorded in the medical record using commercially available Adzynma (excluding doses received in clinical studies or early access program). |
Up to 6 months following the index Adzynma infusion
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Number of Participants With Neutralizing Antibodies to ADAMTS13 Following the Index Adzynma Infusions
Periodo de tiempo: Up to 6 months following the index Adzynma infusion
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Number of participants with neutralizing antibodies to ADAMTS13 following the index Adzynma infusions will be reported. |
Up to 6 months following the index Adzynma infusion
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Number of Participants With Long Term Safety Risk of Hypersensitivity Reactions Following the Index Adzynma Infusion
Periodo de tiempo: Up to 6 months of follow-up time from the index Adzynma infusion
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Number of participants with long term safety risk of hypersensitivity reactions following the index Adzynma infusion will be reported. |
Up to 6 months of follow-up time from the index Adzynma infusion
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Number of Participants With Risk of Hypersensitivity Reactions After Each Administration of Adzynma Following the Index Infusion
Periodo de tiempo: Up to 7 days after each administration of Adzynma
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Number of participants with risk of hypersensitivity reactions after each administration of Adzynma following the index infusion will be reported. |
Up to 7 days after each administration of Adzynma
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Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Periodo de tiempo: From start of the study drug administration up to 5.5 years
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TEAE is defined as any event emerging or manifesting at or after the initiation of treatment with a study intervention or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the study intervention or medicinal product. |
From start of the study drug administration up to 5.5 years
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Gestational age at the Time of Infant Birth
Periodo de tiempo: At birth
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Gestational age at the time of infant birth will be reported. |
At birth
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Number of Participants With Spontaneous Abortion
Periodo de tiempo: Up to 5.5 years
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Spontaneous abortion is defined as pregnancy loss before 20 weeks of gestation. |
Up to 5.5 years
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Number of Participants With Stillbirth
Periodo de tiempo: Up to 5.5 years
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Stillbirth is defined as a fetal death after 20 weeks of gestation. |
Up to 5.5 years
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Number of Participants With Induced Abortion
Periodo de tiempo: Up to 5.5 years
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Number of participants with induced abortion will be reported. |
Up to 5.5 years
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Number of Participants With Live Birth
Periodo de tiempo: Up to 5.5 years
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Number of participants with live birth will be reported. |
Up to 5.5 years
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Number of Participants With any Pregnancy Related Complications
Periodo de tiempo: Up to 5.5 years
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Number of participants with any pregnancy related complications will be reported. |
Up to 5.5 years
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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
Periodo de tiempo: At birth
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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 will be reported. |
At birth
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Number of Infants Categorized According to Year of Birth
Periodo de tiempo: Up to 5.5 years
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Number of infants categorized according to year of birth will be reported. |
Up to 5.5 years
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Number of Infants With Normal Birth Weight
Periodo de tiempo: Up to 5.5 years
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Number of infants with normal birth weight will be reported. |
Up to 5.5 years
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Number of Infants With Small for Gestational Age (SGA)
Periodo de tiempo: Up to 5.5 years
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Small for gestational age is defined as birth size (weight, length or head circumference) less than the 10th centile for sex and gestational age. |
Up to 5.5 years
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Number of Infants With Congenital Anomaly Detected at the Time of Birth
Periodo de tiempo: At birth
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Number of infants with congenital anomaly detected at the time of birth will be reported. |
At birth
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Number of Participants by Breastfeeding Status While Receiving Adzynma
Periodo de tiempo: Up to 5.5 years
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Breastfeeding status during Adzynma treatment will be assessed and categorized based on response (Yes, No, or Unknown). |
Up to 5.5 years
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Number of Infant Categorized by Growth and Development Outcomes
Periodo de tiempo: Up to 5.5 years
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Number of infants categorized by growth and development outcomes (normal, abnormal [specify], or unknown) will be reported. |
Up to 5.5 years
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Colaboradores e Investigadores
Patrocinador
Investigadores
- Director de estudio: Study Director, Takeda
Publicaciones y enlaces útiles
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Estimado)
Finalización del estudio (Estimado)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
- Citopenia
- Procesos Patológicos
- Hemorragia
- Manifestaciones de la piel
- Enfermedades hematológicas
- Trastornos de la coagulación de la sangre
- Trastornos de las plaquetas sanguíneas
- Microangiopatías trombóticas
- Púrpura Trombocitopénica
- Púrpura
- Trombocitopenia
- Trombofilia
- Condiciones Patológicas, Signos y Síntomas
- Signos y síntomas
- Enfermedades hemic y linfáticas
- Púrpura Trombótica Trombocitopénica
Otros números de identificación del estudio
- TAK-755-4007
- EUPAS1000000870 (Otro identificador: EU PAS)
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
Descripción del plan IPD
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Criterios de acceso compartido de IPD
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Tipo de información de apoyo para compartir IPD
- PROTOCOLO DE ESTUDIO
- SAVIA
- CIF
- RSC
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
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