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Relapsed and Progressive Sonic Hedgehog Medulloblastoma With U1 Mutation Registry Study 300 Inovador

Em recrutamento
Os detalhes do estudo clínico estão disponíveis principalmente em inglês. No entanto, a IA Trial Radar pode ajudar! Basta clicar em 'Explicar o estudo' para visualizar e discutir as informações do estudo no idioma selecionado.
O estudo clínico NCT07242963 é um estudo observacional para Meduloblastoma recorrente, Medulloblastoma, Childhood, Recurrent, Medulloblastoma, SHH-activated and TP53 Mutant, Medulloblastoma, SHH-activated and TP53 Wildtype. Seu status atual é: em recrutamento. O estudo começou em 30 de setembro de 2025 e pretende incluir 300 participantes. Coordenado por Mohammad H. Abu Arja e deve ser concluído em 30 de dezembro de 2035. Essas informações foram atualizadas no ClinicalTrials.gov em 21 de novembro de 2025.
Resumo
The purpose of this study is to create a biobank for patients diagnosed with Sonic Hedgehog Medulloblastoma at Baylor College of Medicine/Texas Children's Cancer Center. A biobank is a facility that stores and manages biological samples (such as blood, tissue, or DNA) from individuals, along with detailed health information, for use in medical research to study diseases and develop new treatments. The investigators a...Mostrar mais
Descrição detalhada
This is a multicenter, retro- and prospective observational cohort registry study. The registry will collect prospective data on children and young adults between ≥3 and ≤50 years of age at diagnosis, who are diagnosed with relapsed, refractory, or progressive SHH medulloblastoma. Patients and their biological parents will be recruited for this study. In addition, retrospective data will be gathered on patients enrol...Mostrar mais
Título oficial

Registry Study of Children and Adults Patients With Relapsed, Refractory, or Progressive Sonic Hedgehog Medulloblastoma Harboring U1 Mutation

Condições médicas
Meduloblastoma recorrenteMedulloblastoma, Childhood, RecurrentMedulloblastoma, SHH-activated and TP53 MutantMedulloblastoma, SHH-activated and TP53 Wildtype
Outros IDs do estudo
  • H-56521
Número NCT
NCT07242963
Data de início (real)
2025-09-30
Última atualização postada
2025-11-21
Data de conclusão (estimada)
2035-12-30
Inscrição (estimada)
300
Tipo de estudo
Observacional
Status
Em recrutamento
Palavras-chave
SHH medulloblastoma
medulloblastoma
medulloblastoma Recurrent
Braços / Intervenções
Grupo de participantes/BraçoIntervenção/Tratamento
Group 1
Patients treated at registry institutions
N/A
Group 2
Patients not treated at registry institutions
N/A
Group 3
Biological parents of a subject participating in Group 1 or 2
N/A
Desfecho primário
Medida de desfechoDescrição da medidaPrazo
To describe the incidence of the U1 mutation in SHH medulloblastoma subtypes and verify the feasibility of U1 testing. The primary outcome will be detecting the U1 mutation by polymerase chain reaction (PCR) testing or RNA sequencing (RNAseq).
U1 mutation status, as determined by PCR, will be summarized in the overall sample and within each SHH medulloblastoma subtype with counts and percentages, along with the corresponding 95% confidence intervals. Feasibility Sensitivity, specificity, PPV, NPV, and accuracy of the new RNASeq diagnostic test will be estimated, utilizing PCR as the reference standard. Only complete cases will be utilized in the following estimations. Sensitivity will be estimated as the proportion of true positives out of all positive PCR tests while specificity will be estimated as the proportion of true negatives. Positive predictive value will be estimated as the proportion of true positives out of all positive RNASeq tests. NPV will be estimated as the proportion of true negatives out of all negative RNASeq tests. Finally, accuracy will be estimated as the proportion of true positives and true negatives out of all tests completed.
Through study completion, an average of 2 years
Desfecho secundário
Medida de desfechoDescrição da medidaPrazo
To collect and compare the outcomes of the different treatment regimens utilized for pediatric and adult patients with recurrent, refractory, or progressive SHH medulloblastoma with and without U1 mutation.
Overall survival (OS) and event-free survival (EFS) will be summarized descriptively by U1 mutation status and SHH MB subtype. Both OS and EFS will utilize the Kaplan-Meier method to estimate counts, time-specific rates, corresponding 95% confidence intervals, and survival curves. Early withdrawals and those remaining alive and event-free at the end of the study will be censored at the time of last know status. No interim analyses are planned, as this is an observational study not designed for hypothesis testing. Given that this is an observational cohort study, patient safety with respect to adverse events, will not be assessed.
Through study completion, an average of 2 years
Assistente de participação
Critérios de elegibilidade

Idades elegíveis
Criança, Adulto
Idade mínima
3 Years
Sexos elegíveis
Todos

For Groups 1 and 2, subjects are eligible to be included in the study only if all of the following criteria are met:

  • Age Patients must be ≥ 3 and ≤ 50 years of age at the time of initial diagnosis.
  • Diagnosis Participants must have a diagnosis of SHH medulloblastoma by histologic or molecular criteria at the time of original diagnosis or relapse.
  • Disease status The disease must be recurrent, refractory, or progressive following therapy, including radiotherapy and chemotherapy.
  • Available tumor tissue sample for U1 testing Participants must have available tumor tissue samples to be tested for the U1 mutation.

For Group 3, biological parent(s) of a subject participating in Group 1 or 2 are eligible.

Subjects not meeting the inclusion criteria will be excluded.
Mohammad H. Abu Arja logoMohammad H. Abu Arja
Responsável pelo estudo
Mohammad H. Abu Arja, Patrocinador-Investigador, Instructor, Baylor College of Medicine
Contato central do estudo
Contato: Mohammad H Abu-Arja, MD, MSc, 832-824-0592, [email protected]
Contato: Ta Tara Rideau, [email protected]
1 Locais do estudo em 1 países

Texas

Baylor College of Medicine, Houston, Texas, 77004, United States
Mohammad H Abu-Arja, MD, MSc, Contato, 832-824-0592, [email protected]
Ta-Tara Rideau, Contato, [email protected]
Em recrutamento