Clinical trial · Observational
Extended Analysis for Leukemia/Lymphoma Treatment
A Prospective Investigator-initiated Translational Study Evaluating Individualized Treatment Regiments Based on Respective Biomarker Analyses for Refractory Leukemia and Lymphoma Patients
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Relapsed Hematologic Malignancy | Hematopoietic and Lymphoid Cell Neoplasm | CURATED_BROADER | 0.78 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| ngFDS selected treatment | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- label
- ngFDS selected treatment
- description
- Treatment with commercially available treatments (per package insert instructions) chosen via next-generation functional drug screening (ngFDS).
- interventionNames
- Drug: ngFDS selected treatment
Primary outcomes (1)
- measure
- progression-free survival
- timeFrame
- 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
- description
- 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.
Secondary outcomes (1)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: 1. There are no further standard treatments available for the patient; 2. The patient has undergone at least two lines of previous therapy; 3. Cancer cell-containing biopsies are obtainable; 4. Informed consent from the patient was given; 5. candidate treatments identified by ngFDS are clinically available and considered safe given the patient health state. Exclusion Criteria: 1. 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. 2. No fresh and viable tumor material available. 3. 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. 4. Presence of active gastrointestinal disease or other condition that will interfere significantly with the absorption of drugs. 5. A history of known Human Immunodeficiency Virus (HIV), Hepatitis B Virus (HBV), or Hepatitis C Virus (HCV) infection. 6. A history of known glucose-6-phosphate dehydrogenase (G6PD) deficiency. 7. 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. 8. 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. 9. Pregnant or lactating females. 10. History of alcohol or drug abuse within 6 months prior to screening. 11. No informed consent available. 12. Exclusion criteria effective for respective treatment agents.
References
Publications (2)
- DERIVEDLiebers 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. PMID 37783805
- DERIVEDSnijder B, Vladimer GI, Krall N, Miura K, Schmolke AS, Kornauth C, Lopez de la Fuente O, Choi HS, van der Kouwe E, Gultekin S, Kazianka L, Bigenzahn JW, Hoermann G, Prutsch N, Merkel O, Ringler A, Sabler M, Jeryczynski G, Mayerhoefer ME, Simonitsch-Klupp I, Ocko K, Felberbauer F, Mullauer L, Prager GW, Korkmaz B, Kenner L, Sperr WR, Kralovics R, Gisslinger H, Valent P, Kubicek S, Jager U, Staber PB, Superti-Furga G. Image-based ex-vivo drug screening for patients with aggressive haematological malignancies: interim results from a single-arm, open-label, pilot study. Lancet Haematol. 2017 Dec;4(12):e595-e606. doi: 10.1016/S2352-3026(17)30208-9. Epub 2017 Nov 15. PMID 29153976