Validation of a Personalised Medicine Tool for Multiple Myeloma That Predicts Treatment Effectiveness in Patients (MMpredict)

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.

Study Overview

• Status: Completed
• Conditions: Multiple Myeloma
• Intervention / Treatment: Diagnostic test: Gene Expression Profiling

• Study Type: Observational
• Enrollment (Actual): 278

Contacts and Locations

Study Locations

• Italy
  • Turin, Italy, 10125
    • University of Turin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

MM patients enrolled in clinical trials that have been conducted over the past 5 years.

Description

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:

-

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
MMpredictor as a personalised medicine tool	The main objective of the MMpredict project is to commercialise the MMpredictor as a personalised medicine tool that predicts the most effective treatment strategy for individual Multiple Myeloma (MM) patients.	1 year

Secondary Outcome Measures

Outcome Measure	Time Frame
- Genetic subtyping with the MMprofiler of 800 bio-banked MM patient samples	2 years
- Clinical validation of genetic subtypes correlating with specific treatment effect	2 years
- Establish a treatment decision matrix that will guide physicians in treatment decision-making	2 years
- Perform a Medical Technology Assessment (MTA) to evaluate health economic benefits	2 years
- File addendum to current CE-IVD registration, while also renaming the test to "MMpredictor"	2 years
- Develop and execute commercialisation and marketing plan for the MMpredictor	2 years

Collaborators and Investigators

• Sponsor: Mario Boccadoro

Study record dates

Study Major Dates

• Study Start (Actual): June 14, 2017
• Primary Completion (Actual): November 30, 2017
• Study Completion (Actual): July 25, 2022

Study Registration Dates

• First Submitted: January 16, 2018
• First Submitted That Met QC Criteria: January 16, 2018
• First Posted (Actual): January 24, 2018

Study Record Updates

• Last Update Posted (Actual): June 29, 2023
• Last Update Submitted That Met QC Criteria: June 28, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Immunoproliferative Disorders; Hematologic Diseases; Hemorrhagic Disorders; Hemostatic Disorders; Paraproteinemias; Blood Protein Disorders; Multiple Myeloma; Neoplasms, Plasma Cell
• Other Study ID Numbers: Horizon2020 - FTI Pilot Study

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No