Um Estudo em Crianças e Adultos com Púrpura Trombocitopênica Trombótica Congénita (cTTP) Tratados com Adzynma
Um Estudo de Segurança Pós-Autorização (PASS) para Avaliar Mais a Segurança no Mundo Real em Doentes com Púrpura Trombocitopénica Trombótica Congénita (cTTP) Tratados com 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.
Visão geral do estudo
Status
Intervenção / Tratamento
Tipo de estudo
Inscrição (Estimado)
Contactos e Locais
Contato de estudo
- Nome: Takeda Contact
- Número de telefone: +1-877-825-3327
- E-mail: medinfoUS@takeda.com
Locais de estudo
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Bonn, Alemanha, 53127
- Recrutamento
- Universitatsklinikum Bonn
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Contato:
- Site Contact
- E-mail: heiko.ruehl@ukbonn.de
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Investigador principal:
- Ruehl Heiko
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Cologne, Alemanha, 50937
- Ainda não está recrutando
- Universitaetsklinikum Koeln
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Contato:
- Site Contact
- E-mail: lucas.kuehne@uk-koeln.de
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Investigador principal:
- Kuehne Lucas
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Jena, Alemanha, 7743
- Ainda não está recrutando
- Universitaetsklinikum Jena
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Contato:
- Site Contact
- E-mail: karim.kentouche@med.uni-jena.de
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Investigador principal:
- Kentouche Karim
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Leipzig, Alemanha, 4103
- Recrutamento
- Universitaetsklinikum Leipzig
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Contato:
- Site Contact
- E-mail: maria.weise@medizin.uni-leipzig.de
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Investigador principal:
- Weise Maria
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München, Alemanha
- Ainda não está recrutando
- LMU Klinikum
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Contato:
- Site Contact
- E-mail: ulf.schoenermarck@med.uni-muenchen.de
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Investigador principal:
- Schoenermarck Ulf
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München, Alemanha, 81675
- Ainda não está recrutando
- Klinikum Rechts der Isar der Technischen Universitaet Muenchen
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Investigador principal:
- Braunisch Matthias
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Contato:
- Site Contact
- E-mail: matthiaschristoph.braunisch@mri.tum.de
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Vienna, Áustria, 1090
- Recrutamento
- AKH - Medizinische Universitaet Wien
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Contato:
- Site Contact
- E-mail: johannes.thaler@meduniwien.ac.at
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Investigador principal:
- Johannes Thaler
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Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
- Filho
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Método de amostragem
População do estudo
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.
Descrição
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.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Modelos de observação: COHORT
- Perspectivas de Tempo: RETROSPECTIVE
Coortes e Intervenções
Grupo / Coorte |
Intervenção / Tratamento |
|---|---|
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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. |
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Number of Participants With Hypersensitivity Reactions Following the Index Adzynma Infusions
Prazo: 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
Prazo: 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 resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Number of Participants With Long Term Safety Risk of Hypersensitivity Reactions Following the Index Adzynma Infusion
Prazo: 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
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
|
|
Number of Participants With Induced Abortion
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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)
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
- Diretor de estudo: Study Director, Takeda
Publicações e links úteis
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Real)
Conclusão Primária (Estimado)
Conclusão do estudo (Estimado)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
- Citopenia
- Processos Patológicos
- Hemorragia
- Manifestações de pele
- Doenças Hematológicas
- Distúrbios da Coagulação Sanguínea
- Distúrbios das plaquetas sanguíneas
- Microangiopatias Trombóticas
- Púrpura Trombocitopênica
- Púrpura
- Trombocitopenia
- Trombofilia
- Condições Patológicas, Sinais e Sintomas
- Sinais e sintomas
- Doenças hemic e linfáticas
- Púrpura Trombocitopênica Trombótica
Outros números de identificação do estudo
- TAK-755-4007
- EUPAS1000000870 (Outro identificador: EU PAS)
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
Descrição do plano 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.
Critérios de acesso de compartilhamento 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 informação de suporte de compartilhamento de IPD
- PROTOCOLO DE ESTUDO
- SEIVA
- CIF
- CSR
Informações sobre medicamentos e dispositivos, documentos de estudo
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