Clinical trial NCT03409692: Validation of a Personalised Medicine Tool for Multiple Myeloma That Predicts Treatment Effectiveness in Patients
Official title: Validation of a Personalised Medicine Tool for Multiple Myeloma That Predicts Treatment Effectiveness in Patients
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Multiple Myeloma
Lead sponsor: University of Turin, Italy
Sponsor class: OTHER
First posted: 2018-01-24
Enrollment: 278
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: MM patients enrolled in clinical trials that have been conducted over the past 5 years.

Brief summary: The consortium aims to commercialise the MMpredictor as a personalised medicine tool that predicts the most effective treatment strategy for individual Multiple Myeloma (MM) patients. MM is the second most common form of blood cancer contributing to 15% of all blood cancers and \~1,5% and 2% of all cancer deaths annually in the EU and US, respectively.

Patients show a large variability in treatment response and side effects due to tumour heterogeneity and the patient's intrinsic characteristics. Therefore, not every treatment will be suitable for each patient, and treatment strategies are often based on trial-and-error. The availability of multiple (\>20) treatment options complicates treatment decision-making even more. With the current development of many more promising treatments, there is an urgent unmet clinical need for a diagnostic assay that supports personalised cancer treatment in order to improve patient health outcomes, prevent side effects and reduce healthcare costs.

SkylineDx has previously developed the MMprofiler, a microarray-based diagnostic test that can subtype MM patients and reliably predict MM patient survival (prognosis). In this project, the test's clinical value will be expanded to include the prediction of treatment effectiveness in individual patients based on Gene Expression Profiling. An addendum for new intended use will be filed to the current in vitro diagnostic (IVD) registration, while renaming the test to MMpredictor. The project will also focus on positioning the test as a cost-effective IVD test for personalised medicine, that will increase health outcome and quality of life of patients and reduce healthcare costs.

The consortium consists of a life science SME specialised in molecular diagnostics, clinical centres with world renowned KOLs, a leading health economic institute, and a European MM patient advocacy organisation combining all the required complementary expertise to successfully bring the MMpredictor to market.


Eligibility criteria: Inclusion Criteria:

* The bio-banked MM patient samples and clinical data will be obtained from previous European clinical trials that have been conducted over the past 5 years within the participating clinical centres and other clinical centers. The samples were not collected for the purposes of this project. The subjects from which the data was initially taken cannot be identified from the data/records. However, the patients from the above mentioned trial have explicitly consented for the use of their samples for other (future) clinical research purposes.

Exclusion Criteria:

-

Primary outcomes:
measure: MMpredictor as a personalised medicine tool
timeFrame: 1 year
Secondary outcomes:
measure: - Genetic subtyping with the MMprofiler of 800 bio-banked MM patient samples
timeFrame: 2 years
measure: - Clinical validation of genetic subtypes correlating with specific treatment effect
timeFrame: 2 years
measure: - Establish a treatment decision matrix that will guide physicians in treatment decision-making
timeFrame: 2 years
measure: - Perform a Medical Technology Assessment (MTA) to evaluate health economic benefits
timeFrame: 2 years
measure: - File addendum to current CE-IVD registration, while also renaming the test to "MMpredictor"
timeFrame: 2 years
measure: - Develop and execute commercialisation and marketing plan for the MMpredictor
timeFrame: 2 years
Other outcomes:
Locations:
city: Turin
country: Italy
facility: University of Turin
Study officials:
References: