Clinical trial NCT02999750: EXtendedAnalysis for Cancer Treatment
Official title: EXACT: EXtendedAnalysis for Cancer Treatment A Prospective Investigator-initiated Translational Study Evaluating Individualized Treatment Regiments Based on Respective Biomarker Analyses for Refractory Cancer Patients
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Malignant Solid Tumor
Lead sponsor: Medical University of Vienna
Sponsor class: OTHER
First posted: 2016-12-21
Enrollment: 55
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to prospectively validate treatment benefit of an individualized treatment concept based on molecular profiling (MP) from paraffin-embedded tumor tissue sections obtained before the start of treatment (real time biopsy).

Detailed description: The treatment concept will be considered to be of clinical benefit for the individual patient if a progression-free survival (PFS) ratio (PFS on MP-based therapy / best PFS achieved by prior therapy) will be \> 1.0 thus generating a patient cohort with this very property. Thereby, the null hypothesis (that ≤ 40 % of this patient population would have a PFS ratio of \> 1.0) will be evaluated with each patient being his own control. For tumor types with high numbers of patients per cohort, the overall response rate (ORR) will be evaluated.

Eligibility criteria: Inclusion Criteria:

Consenting patients of \>19 years with advanced cancer fulfilling the criteria of having:

* an advanced malignancy with metastatic spread refractory to conventional treatment
* a life expectancy of \>4 months,
* the possibility to access and biopsy tumour material within 4 weeks before onset of individualized treatment,
* a malignancy amenable to further treatment options with either cytotoxic drugs, tyrosine kinase inhibitors, monoclonal antibodies or related molecules with anti-proliferative potential to cancer cells, as assessed by the ex vivo analysis and a likelihood of treatment response according to the mathematical model (all outlined in detail above),
* agreed to participate by their signature on an informed consent form are eligible.

Exclusion Criteria:

* Presence of further treatment options, as defined by NCCN guidelines which are available in Austria representing a possible further treatment-related response by conventional therapies according to generally accepted medical evidence.
* No fresh and viable tumor material available.
* Current use of therapeutic warfarin.
* 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 those 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)
* 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.

Primary outcomes:
measure: Use of real time biopsy to establish an individual molecular profile by using next generation sequencing
timeFrame: 2 years
Secondary outcomes:
Other outcomes:
Locations:
city: Vienna
country: Austria
facility: AKH Vienna
Study officials:
References: