Rare Malignant Neoplasm

The goal of this Phase I/II observational and interventional platform study is to evaluate the safety and efficacy of multiple types of innovative anti-tumor drugs and new technologies in patients with rare solid tumors. The study utilizes multi-dimensional precision screening (including WES, RNAseq, mIHC, and quantitative proteomics) to match patients with specific sub-protocols. Key questions it aims to answer: Assess the safety of innovative therapies in rare tumor populations. Evaluate the objective response rate (ORR) and other efficacy metrics. Explore biomarkers related to therapeutic efficacy. Participants: Patients with metastatic or advanced rare solid tumors who have failed standard therapy or have no standard treatment options.

Some rare cancers are hard-to-treat and patients have a poor prognosis. It is known that some of these patients have targetable molecular alterations, and some benefit from targeted drugs. However in many cases these drugs are not approved for the rare cancers. In this study the aim is to do advanced molecular diagnostics to identify possible targets for therapy, and to treat accordingly.

The goal of this observational study is to collect structured data about diagnosis, treatment and follow-up of rare and highly complex neoplasms. The study has a rerospective part that will consist in gathering data on patients accessing IRCCS Azienda Ospedaliero-Universitaria di Bologna Policlinico di S. Orsola from 01/01/2000 to 28/09/2017 and a prospective one ranging from 29/09/2017 to 31/12/2025.

This is a registry study that aims to collect patients' data with advanced-stage rare cancer in Asia-Pacific region. Data includes clinical information, details of treatment, prognosis, pathological diagnosis and genetic biomarkers by next-generation sequencing. The relationship between cancer types and prognosis, the effect of treatments, and the cancer type-specific incidence of genomic alterations will be investigated to discover more specific and effective treatment.