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Systematic and Mechanism-based Approach to Rational Treatment Trials of Blood Cancer (SMART)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03488641
Recruitment Status : Recruiting
First Posted : April 5, 2018
Last Update Posted : April 12, 2019
University Hospital Heidelberg
Information provided by (Responsible Party):
Sascha Dietrich, German Cancer Research Center

Brief Summary:
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.

Condition or disease Intervention/treatment
Hematologic Diseases Treatment Diagnostic Test: ex-vivo drug response assay

Detailed Description:

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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Study Type : Observational
Estimated Enrollment : 80 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Systematic and Mechanism-based Approach to Rational Treatment Trials of Blood Cancer (SMARTrial)
Actual Study Start Date : April 16, 2018
Estimated Primary Completion Date : July 2021
Estimated Study Completion Date : March 2023

Intervention Details:
  • Diagnostic Test: ex-vivo drug response assay
    drug sensitivity testing of primary patient derived cancer cells

Primary Outcome Measures :
  1. Rate of completed drug sensitivity testing [ Time Frame: 7 days ]
    Patients' sample (blood, bonemarrow aspirate, tissue of lymphnode) will collected on day 0. Ex-vivo drug sensitivity testing will be performed.

Secondary Outcome Measures :
  1. Accuracy of patients' drug response prediction by ex-vivo drug profiling [ Time Frame: from date of inclusion until date of best treatment response (latest 12 months) ]
    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

  2. Prediction of time to next treatment [ Time Frame: from date of inclusion until change of treatment (latest 12 months) ]
    prediction of time to next treatment by a mathematical model based on ex-vivo drug response testing

Biospecimen Retention:   Samples With DNA
Blood samples, bonemarrow aspiration, tissue of lymphnodes

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with diagnosis of a hematological malignany with the need of treatment

Inclusion Criteria:

  1. Diagnosis of a hematological malignancy: patients with leukemia, myeloma or lymphoma (e.g. ALL, AML, CLL, T-PLL, MCL, MM) who are in need of treatment and are willing to donate sufficient tumor material for ex-vivo drug sensitivity testing.
  2. The treating physician needs to indicate treatment.
  3. Measurable disease burden according to criteria as mention in section 3.
  4. Treatment must be scheduled and the patient must be eligible for the planed treatment as judged by the treating physician.
  5. Availability of 5x10e7 cells from peripheral blood draws, bone marrow aspirations or lymph node biopsies.
  6. Patient's written informed consent present.
  7. Ability to understand the nature of the trial and the trial related procedures and to comply with them.

Exclusion Criteria:

  1. Any condition, which precludes initiation of treatment (e.g. breast feeding, pregnancy, infections, etc.) as judged by the treating physician.
  2. Any coexisting medical or psychological condition that would preclude participation in the required study procedures, as judged by the treating physician.
  3. No systemic cancer treatment except for cytoreductive pretreatment within 1 week of enrollment.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03488641

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Contact: Sascha Dietrich, MD +496221568030
Contact: Thorsten Zenz, MD +496221568030

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University Hospital Heidelberg Recruiting
Heidelberg, Germany
Contact: Nora Liebers, MD         
Sponsors and Collaborators
German Cancer Research Center
University Hospital Heidelberg
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Principal Investigator: Sascha Dietrich, MD University Hospital Heidelberg and DKFZ

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Responsible Party: Sascha Dietrich, Sascha Dietrich, MD, German Cancer Research Center Identifier: NCT03488641     History of Changes
Other Study ID Numbers: SMART
First Posted: April 5, 2018    Key Record Dates
Last Update Posted: April 12, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Sascha Dietrich, German Cancer Research Center:
Signal Transduction Pathway Deregulation
Drug sensitivity testing
Targeted Therapy
Genomic landscape
Additional relevant MeSH terms:
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Hematologic Diseases