Extended Analysis for Leukemia/Lymphoma Treatment (EXALT)
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|ClinicalTrials.gov Identifier: NCT03096821|
Recruitment Status : Completed
First Posted : March 30, 2017
Last Update Posted : February 24, 2021
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Patients with relapsed/ refractory acute leukemia and relapsed/ refractory aggressive lymphoma harboring an activating genetic alteration (gene mutation, gene fusion) or drug-able biomarker / activated signal transduction pathway and resistant to any approved treatment modality will be eligible for this study.
The investigators aim to combine DNA sequencing-based molecular profiling with an ex vivo high-throughput drug screening strategy. For the latter method, viable cells are obtained from the individual patient's lymphoma or leukemia in order to determine i)the expression of relevant therapeutic target molecules and ii)the ex vivo response of the patient's cancer cells to a panel of agents with anticancer activity. In addition, analysis of tumor stroma cells will provide information about the differential target expression and cellular sensitivity aiming at the evaluation of a therapeutic safety window. Thereby, biological material will have to be accessed within 4 weeks before onset of individualized treatment (real-time biopsy). Bioinformatic data-management based on a Bayesian statistical approach will support individualized treatment decisions in this controlled clinical approach.
|Condition or disease||Intervention/treatment|
|Relapsed Hematologic Malignancy||Drug: ngFDS selected treatment|
|Study Type :||Observational|
|Actual Enrollment :||143 participants|
|Official Title:||A Prospective Investigator-initiated Translational Study Evaluating Individualized Treatment Regiments Based on Respective Biomarker Analyses for Refractory Leukemia and Lymphoma Patients|
|Actual Study Start Date :||September 2015|
|Actual Primary Completion Date :||January 30, 2020|
|Actual Study Completion Date :||January 30, 2020|
ngFDS selected treatment
Treatment with commercially available treatments (per package insert instructions) chosen via next-generation functional drug screening (ngFDS).
Drug: ngFDS selected treatment
Treatment based on next-generation functional drug screening (ngFDS). Treatment with commercially available treatments (per package insert instructions) selected by functional drug screening. For drug screening tumor cells from bone marrow aspirates, peripheral blood, pleural effusion, or excised lymph node samples are purified over Ficoll gradient (bone marrow, peripheral blood, pleural effusion) (GE healthcare) or homogenized and filtered (lymph tissue).
Other Name: Treatment based on ngFDS
- progression-free survival [ Time Frame: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 100 months ]To compare progression-free survival using a treatment regimen selected by molecular profiling with progression-free survival for the most recent regimen the patient has progressed on. The treatment concept is deemed for clinical benefit for the individual patient who has a PSF ratio (PSF on molecular profiling-based therapy/PSF on prior therapy) of ≥ 1.3. For this purpose we will reject the null hypothesis, which is defined as follows: ≤ 15% of patients would have a PFS ratio of ≥ 1.3. Thus, the individual patient is his own control. For tumor types with high numbers of patients per cohort, the overall response rate (ORR) will be evaluated.
- overall response rate [ Time Frame: 4 months ]overall response rate (ORR: achieving either CR or PR). For lymphoma patients' responses were classified as complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD) according to the criteria proposed by the International Workshop for malignant lymphoma. For leukemia patients, responses were assessed following the response criteria defined by the recommendations of the European Leukemia net.
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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|
- There are no further standard treatments available for the patient;
- The patient has undergone at least two lines of previous therapy;
- Cancer cell-containing biopsies are obtainable;
- Informed consent from the patient was given;
- candidate treatments identified by ngFDS are clinically available and considered safe given the patient health state.
- Presence of further treatment options, as defined by NCCN guidelines or Austrian guidelines representing a possible further treatment-related response by conventional therapies according to generally accepted medical evidence.
- No fresh and viable tumor material available.
- Unresolved toxicity of National Cancer Institute Common Terminology Criteria for Adverse Events, version 4.0 (NCI CTCAE v4.0) Grade 2 or higher from previous anti-cancer therapy, except alopecia.
- Presence of active gastrointestinal disease or other condition that will interfere significantly with the absorption of drugs.
- A history of known Human Immunodeficiency Virus (HIV), Hepatitis B Virus (HBV), or Hepatitis C Virus (HCV) infection.
- A history of known glucose-6-phosphate dehydrogenase (G6PD) deficiency.
- History of other malignancy. Subjects who have been disease-free for 5 years, or subjects with a history of completely resected non-melanoma skin cancer or successfully treated in situ carcinoma are eligible.
- Uncontrolled medical conditions (i.e, diabetes mellitus, hypertension, etc), psychological, familial, sociological, or geographical conditions that do not permit compliance with the protocol; or unwillingness or inability to follow the procedures required in the protocol.
- Pregnant or lactating females.
- History of alcohol or drug abuse within 6 months prior to screening.
- No informed consent available.
- Exclusion criteria effective for respective treatment agents.
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 ClinicalTrials.gov identifier (NCT number): NCT03096821
|Medical University Vienna|
|Vienna, Austria, 1090|
|Principal Investigator:||Philipp B Staber, MD, PhD||Medical University of Vienna, Department of Medicine I, Division of Hematology and Hemostaseology|
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
|Responsible Party:||Philipp Staber, MD, PhD, Assoc.Prof., Medical University of Vienna|
|Other Study ID Numbers:||
1830/2015 ( Other Identifier: ethic committee of Medical University Vienna )
|First Posted:||March 30, 2017 Key Record Dates|
|Last Update Posted:||February 24, 2021|
|Last Verified:||February 2021|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||Undecided|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
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