Registo de Cancros Raros em Tumores Sólidos Muito Raros para Crianças, Adolescentes e Adultos Jovens, Liderado pela Iniciativa de Dados sobre Cancro Infantil do NCI (CCDI)
Registo de Cancros Raros em Tumores Sólidos Muito Raros em Crianças, Adolescentes e Jovens Adultos, Liderado pela Iniciativa de Dados sobre Cancro Infantil do NCI (CCDI)
Background:
All childhood cancers are rare, but some are called very rare. Very rare cancers are diagnosed in 2 or fewer out of 1 million people each year. Researchers want to gather data so they can learn more about these very rare cancers. They hope to use the data to develop future treatments.
Objective:
To gather data for a registry of very rare cancers found in children, teens, and young adults.
Eligibility:
People aged 1 month to 39 years newly diagnosed (within the past year) with a very rare cancer.
Design:
Participation will be by phone or email. No clinic visits are required.
Researchers will look at the participant s medical records. They will ask for samples of tumor tissue that were already removed. They will use the samples for genetic testing. The results of these tests will be sent to the participant s own doctors.
Some participants will be asked for saliva or cheek swab samples. They will receive a kit in the mail. They will spit into a tube or swab the inside of their cheek. They will mail the sample back to the lab.
Participants will fill out questionnaires once a year for 5 years. They will answer questions about:
Family history, such as other cancers in the family and their income, work, and education.
Demographics, such as their gender, nationality, ethnicity, education, and work history.
Symptoms and treatment for their cancer. This may include level of pain, and emotional and physical well-being.
Participants data will be added to a secure database for other researchers. Their data will be anonymous.
Visão geral do estudo
Status
Status
Condições
Condições
Intervenção / Tratamento
Intervenção / Tratamento
Descrição detalhada
Background:
- Rare cancers are defined by the NCI as fewer than 15 incident cases per 100,000 people per year. Overall, pediatric tumors account for less than 1% of all cancers diagnosed in the United States (US) per year.
- The European Cooperative Study Group for Pediatric Rare Tumors (EXPeRT) group has determined that the definition of very rare pediatric cancers is fewer than 2 cases per million per year and/or those not eligible for clinical trials.
- Frequently, for these very rare solid tumors and hematologic malignancies, little is known about their natural history, including clinical behavior, molecular/genetic characteristics, optimal management, drug response, and evidence-based standard therapy does not exist. Many patients endure the challenges of achieving an accurate and timely diagnosis, along with the difficulty of identifying a center with treatment expertise.
- Although several disease-specific pediatric and adult rare cancer efforts exist, they are frequently siloed without standardization across efforts; and, many rare cancers have not been systematically studied.
Objective:
-To establish a longitudinal observational study and registry for very rare pediatric and Adolescents and Young Adults (AYA) solid tumors
Eligibility:
-Pediatric, adolescent, or young adult participants (<= 39 years old at diagnosis) with very rare solid tumors
Design:
- This will be a longitudinal observational study and registry for children and AYAs with very rare cancers.
- Recruitment will be through 2 mechanisms, the Children s Oncology Group s (COG) Project EveryChild Protocol (APEC14B1, NCT02402244) and physician/self-referral.
- This study will be led by the NCI s CCDI and NCI s POB.
- Enrollment, including consent, distribution of family history/individual medical history/patient report outcome questionnaires, collection of medical records and tissue/saliva/buccal samples, and extraction of data, as well as longitudinal follow-up will be managed centrally.
- Tumor tissue and saliva/buccal samples will be run through the CCDI s Molecular Characterization Initiative (MCI) for comprehensive clinical molecular profiling. The remaining samples will be banked for research analyses. Research analysis will be per the CCDI research characterization pipeline.
- Since long-term follow-up of individuals with very rare cancers is a major feature of the study, we intend to maintain active contact with study participants for up to 5 years.
- Data will be deposited into the CCDI Data Ecosystem.
Tipo de estudo
Tipo de estudo
Inscrição (Estimado)
Inscrição
Contactos e Locais
Contato de estudo
Contato de estudo
- Nome: Mary F Wedekind Malone, D.O.
- Número de telefone: (240) 858-3765
- E-mail: maryfrances.wedekindmalone@nih.gov
Locais de estudo
-
-
Maryland
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Bethesda, Maryland, Estados Unidos, 20892
- Recrutamento
- National Institutes of Health Clinical Center
-
Contato:
- National Cancer Institute Referral Office
- Número de telefone: 888-624-1937
- E-mail: ncimo_referrals@mail.nih.gov
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-
Critérios de participação
Critérios de elegibilidade
Critérios de elegibilidade
Idades elegíveis para estudo
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Método de amostragem
População do estudo
This is a longitudinal observational study and registry for children, adolescents and young adults (AYAs) with very rare cancers.
Descrição
- INCLUSION CRITERIA:
- History of newly diagnosed (within 1 year of diagnosis) very rare solid tumor (defined as an estimated 2 incident cases per million per year).
- Age >= 1 month and <= 39 years at the time of diagnosis.
- Participants must have established care with a local treating physician.
- Ability of the participant, parent/guardian, or Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document.
EXCLUSION CRITERIA:
-
Diagnosis of any of the following at any time:
- Ewing Sarcoma
- Osteosarcoma
- Rhabdomyosarcoma
- Diffuse midline glioma (H3K27 altered)
- Atypical teratoid rhabdoid tumor
- Pleuropulmonary blastoma
- Common adult cancers that occur in pediatric/AYA populations (i.e., colorectal cancer, breast cancer)
-
The participant is unlikely to comply with the terms of the protocol.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Modelos de observação: CASE_ONLY
- Perspectivas de Tempo: PROSPECTIVE
Número de grupos/coortes
Coortes e Intervenções
Grupo / CoorteGrupo / Coorte |
Intervenção / TratamentoIntervenção / Tratamento |
|---|---|
|
1/ Cohort 1
Participants with very rare tumors |
We will collect information about the initial presentation and diagnosis of the disease, management, and tumor treatment history. Participants or parent/guardian will be asked to complete questionnaires and patient-reported outcome (PRO) instruments. Pathology materials (e.g., tissue samples, slides, or blocks) and saliva and/or buccal sample will be requested. |
O que o estudo está medindo?
Medidas de resultados primários
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
To establish a longitudinal observational study and registry for very rare pediatric and AYA solid tumors
Prazo: Through 5 years after enrollment
|
Percentage of participants from identified recruitment sources will be tabulated and described. An analysis of the ability to adequately obtain medical records at initial evaluation and follow-up to perform medical data extraction, which is critical to establishing a registry and longitudinal observational study will be assessed. |
Through 5 years after enrollment
|
Medidas de resultados secundários
Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
To evaluate the feasibility of PRO using validated reporting platforms suitable for pediatric and AYA populations
Prazo: At time of enrollment/study entry, and 2 and 5 years after enrollment
|
The percentage of participants who successfully complete longitudinal data collection will be described. |
At time of enrollment/study entry, and 2 and 5 years after enrollment
|
|
To conduct comprehensive clinical molecular characterization, utilizing CCDI MCI
Prazo: At time of enrollment/study entry
|
The percentage of participants who successfully provide required sample(s) and where correlative analyses are able to be performed will be described. |
At time of enrollment/study entry
|
Colaboradores e Investigadores
Patrocinador
Patrocinador
Investigadores
Investigadores
- Investigador principal: Mary F Wedekind Malone, D.O., National Cancer Institute (NCI)
Publicações e links úteis
Links úteis
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Estimado)
Início do estudo
Conclusão Primária (Estimado)
Conclusão Primária
Conclusão do estudo (Estimado)
Conclusão do estudo
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Primeira postagem
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última Atualização Postada
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Última verificação
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Outros números de identificação do estudo
Outros números de identificação do estudo
- 10002496
- 002496-C
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
Descrição do plano IPD
This study will comply with the NIH Data Management and Sharing (DMS) Policy as waived or approved by the Center for Cancer Research, which applies to all new and ongoing NIH-funded research in the IRP, as of January 25, 2023, that is associated with a ZIA, with a clinical protocol that undergoes scientific review and/or will involve genomic data sharing.
Prazo de Compartilhamento de IPD
Data will be made available as soon as possible or at the time of associated publication. Data not published in a manuscript will be shared via public source once the data set completes QC.
Critérios de acesso de compartilhamento IPD
Clinical data will be made available upon request and with the permission of the study PI. Genomic data are made available via dbGAP through requests to the data custodians.
Tipo de informação de suporte de compartilhamento de IPD
- PROTOCOLO DE ESTUDO
- SEIVA
- CIF
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