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| Name | Class |
|---|---|
| University Hospital Heidelberg | OTHER |
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This observational study evaluates if drug response testing can be performed within 7 days and analyzes the value of ex-vivo drug screening for hematological malignancies as a biomarker to predict outcome, clinical course and response to treatment.
Targeted treatments have revolutionized care of individual diseases. While a new generation of targeted drugs is emerging in leukemia and lymphoma it remains clinical reality that most genetic information is not used for therapeutic stratification. This is in part based on the shortcomings of traditional biomarker discovery within clinical trials, where throughput is limited in both, drug number and sample size. If it were possible to map the variable pathway dependencies and drug sensitivity patterns in individual patients it is likely to become an asset to identify genotype-phenotype associations, understand the underlying complexities of molecular networks and further precision medicine stratification.
To link clinical outcome and ex-vivo drug response assays, the investigators systematically measure pathway sensitivity and resistance of primary tumor cells ex-vivo using a diverse compound library for individual patients in need of treatment. By systematically analyzing ex-vivo drug response patterns, tumors should be functionally grouped, by response phenotype. While for the purpose of this study selection of a specific treatment will not be based on ex-vivo drug response assays, clinical response- and follow-up data of patients will be prospectively collected in parallel.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ex-vivo drug response assay | Diagnostic Test | drug sensitivity testing of primary patient derived cancer cells |
| Measure | Description | Time Frame |
|---|---|---|
| Rate of completed drug sensitivity testing | Patients' sample (blood, bonemarrow aspirate, tissue of lymphnode) will collected on day 0. Ex-vivo drug sensitivity testing will be performed. | 7 days |
| Measure | Description | Time Frame |
|---|---|---|
| Accuracy of patients' drug response prediction by ex-vivo drug profiling | Ex-vivo drug sensitivity categorizes drugs as sensitive/not sensitive. Results will be compared with clinical outcome of patient (response vs. stable disease as defined in the clinical response definition by protocol | from date of inclusion until date of best treatment response (latest 12 months) |
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Inclusion Criteria:
Exclusion Criteria:
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Patients with diagnosis of a hematological malignany with the need of treatment
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| Name | Affiliation | Role |
|---|---|---|
| Sascha Dietrich, MD | University Hospital Heidelberg and DKFZ | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University Hospital Heidelberg | Heidelberg | Germany |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37783805 | Derived | Liebers N, Bruch PM, Terzer T, Hernandez-Hernandez M, Paramasivam N, Fitzgerald D, Altmann H, Roider T, Kolb C, Knoll M, Lenze A, Platzbecker U, Rollig C, Baldus C, Serve H, Bornhauser M, Hubschmann D, Muller-Tidow C, Stolzel F, Huber W, Benner A, Zenz T, Lu J, Dietrich S. Ex vivo drug response profiling for response and outcome prediction in hematologic malignancies: the prospective non-interventional SMARTrial. Nat Cancer. 2023 Dec;4(12):1648-1659. doi: 10.1038/s43018-023-00645-5. Epub 2023 Oct 2. |
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| ID | Term |
|---|---|
| D006402 | Hematologic Diseases |
| ID | Term |
|---|---|
| D006425 | Hemic and Lymphatic Diseases |
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Blood samples, bonemarrow aspiration, tissue of lymphnodes
| Prediction of time to next treatment | prediction of time to next treatment by a mathematical model based on ex-vivo drug response testing | from date of inclusion until change of treatment (latest 12 months) |