Neoplasms

All Conditions

Rare Diseases

Sarcoma

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Soft Tissue Sarcoma

Bruno Fuchs, Prof.

Larissa Slater

Christian Michelitsch, Dr. med.

Silke Gillessen Sommer, Prof. Dr. med.

Stefan Breitenstein, Prof. Dr. med.

Bruno Fuchs, MD PhD

Gabriela Studer, Prof. Dr. med.

Beata Bode, Prof. Dr. med.

Paul Magnus Schneider, Prof. Dr.

Annelies Schnider, Dr. med.

Markus Weber, Prof. Dr. med. Dr. h.c.

Bruno Fuchs, Prof

The primary significance of this project is to perform a detailed analysis to assess the diagnostics, treatment and follow-up care, to develop a prediction model for future patient selection regarding various diagnostic and treatment procedures.

Within the framework of interdisciplinary cooperation for a sarcoma problem, predictive and prognostic criteria will be developed that improve the treatment and particularly outcome.

The outcome variables include patient characteristics, tumor types and characteristics, histology, management and treatment strategies as well as recurrence rate and overall survival.

Swiss Sarcoma Network: Prediction Model for Patient Selection in Sarcoma Care

Data collected in the registry will be evaluated and analyzed to determine criteria to improve treatment strategies

Swiss Sarcoma Network

Kantonsspital Winterthur KSW

Immune System Diseases

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Diseases and Abnormalities at or Before Birth

Skin and Connective Tissue Diseases

Symptoms and General Pathology

Osteosarcoma

Hypersensitivity

Wilms Tumor

Sarcoma, Ewing

Rhabdomyosarcoma

Rhabdoid Tumor

Neoplasms, Bone Tissue

Neoplasms, Connective Tissue

Myosarcoma

Kidney Neoplasms

Urologic Neoplasms

Urogenital Neoplasms

Syndrome

Neoplastic Syndromes, Hereditary

Urogenital Diseases

Female Urogenital Diseases

Pregnancy Complications

Female Urogenital Diseases and Pregnancy Complications

Kidney Diseases

Urologic Diseases

Male Urogenital Diseases

Genetic Diseases, Inborn

Wilms' Tumor

Ewing Sarcoma

The investigators intend to enroll newly diagnosed or refractory/relapsed pediatric patients with all types of sarcomas where tumor tissue would be available for ex vivo drug screening and genomic profiling. This observational study will assess how ex vivo drug testing and mutation profiling may predict clinical outcomes (response, survival, or relapse). The treating physician will decide which of the standard treatment options is appropriate independent of the DST results. The results of DST will not be available to the treating physician at the time of decision on the treatment regimen. DST will include all drugs from the standard treatment regimens available for all types of sarcomas

Diana Azzam, PhD

Daria Salyakina, PhD

Maggie Fader, MD

This study is a prospective, non-randomized observational study. Freshly isolated tumor cells will be tested for chemosensitivity to the standard of care drugs as single agents and in combinations using state-of-the-art viability assay designed for ex-vivo high-throughput drug sensitivity testing (DST). In addition, the genetic profile of the tumor will be obtained from the medical records and correlated with drug response.

The excised tumors or a biopsy will be interrogated for sensitivity or resistance to FDA-approved and/or available investigational agents. In addition, normal samples (blood or buccal swab) will be collected for genetic analysis of germline mutations and cancer predisposition markers. The timeframe between the sample acquisition and ex vivo DST results return will be approximately 5-10 working days. All drugs tested in the DST assay will be assigned a hybrid score reflecting the tumor's sensitivity and drug toxicity.

This is an observational study and not a treatment protocol. It will assess how ex vivo drug testing and mutation profiling may predict clinical outcomes (response, survival, or relapse). The treating physician will decide which of the standard treatment options is appropriate independent of the DST results. The results of DST will not be available to the treating physician at the time of decision on the treatment regimen. DST will include all drugs from the standard treatment regimens available for all types of sarcomas

Perform molecular and functional drug testing on blood, biopsy, bone marrow, and tumor samples at relapse.

Drug Sensitivity and Mutation Profiling

Drug Sensitivity Testing and Mutation Profiling in Childhood Sarcomas

Response to therapy (RTT): an event of achieving "partial response" or "complete response" during the study period, based on the best response of the corresponding enrolled patient.

Progression-free survival (PFS): a composite end point defined as the censored event time from enrollment to either disease relapse or mortality. This will be evaluated retrospectively at the end of the study. The investigators will use the two-sample long-rank test to assess the hazard ratio of PFS events between the two groups classified as drug sensitive and insensitive by the DST.The study will enroll 15 patients and it is assumed 50% of these subjects will be classified as drug sensitive based on the DST at the threshold value of 10.

Classification accuracy of DST for predicting the RTT

Classification accuracy of DST for predicting the PFS

The DTS test results will be treated as the continuous classifier, and the binary RTT status for each subject along with the binary PFS status by the end of the study will be used as the binary reference status (with and without RTT/PFS endpoint). The area under the curve (AUC) of the resulting ROC curve will be used a metric for the overall classification accuracy

Nicklaus Children's Hospital f/k/a Miami Children's Hospital

Cornelia T. Bailey Foundation

Florida International University

Assistant Professor

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Swiss Sarcoma Network: Prediction Model for Patient Selection in Sarcoma Care	NCT04300257	Sarcoma		-	Kantonsspital Winterthur KSW
Drug Sensitivity and Mutation Profiling	NCT04956198	Ewing Sarcoma Osteosarcoma Rhabdomyosarcom... Wilms Tumor Sarcoma, Spindl... Sarcoma Rhabdoi... Childhood Sarco...		- 21 Years	Florida International University

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