Development of Digital Biomarkers in Multiple Sclerosis: Validation Study 2

NCT06309173 | Withdrawn

• 1 other identifier: 0000-00000; ko23Hemkens
• Study Type: observational
• Target: N/A
• Locations: 2 countries
• Sites: 3

Brief Summary

Multiple sclerosis (MS) is a chronic inflammatory disease of the central nervous system (CNS). Diagnosis is established by clinical assessment of persons with MS (PwMS), in combination with imaging and body fluid assessments. Treatment decisions in MS are mainly based on periodic monitoring of disease activity and progression through clinical and imaging assessments. The predictive and prognostic value of currently used assessments to individualize treatment decisions is still very limited. Emerging digital measures have the potential to provide granular health status measurements that would allow monitoring MS disease activity and progression continuously and remotely, in real-world settings, with minimal disruption of patients' life. Using the investigators' self developed dreaMS software program the investigators previously identified digital biomarkers (DB) that hold promise to provide detailed and accurate assessments of MS-related health status and disease progression to complement traditional clinical, imaging, or body fluid assessments. This international, observational study aims to evaluate and validate the generalizability of these DB across different languages and cultural settings to provide DB that are helpful for patient care, research, and regulatory decisions. Beyond this, the processes and data structures created for this study are intended to establish a collaborative research platform for subsequent studies, including pragmatic trials, promoting new long-term international academic collaborations.

Conditions

Multiple Sclerosis

Keywords

Digital Biomarkers

Outcome Measures

Primary Outcomes (2)

• Correlation of the digital features with the respective measurements of the clinical reference tests

  Spearman correlation coefficients higher than 0.4 (lower bound of 95% confidence interval) are considered relevant. All scheduled pairs of measurements collected during the study will be used. As the yearly observations of a patient are not independent, standard confidence intervals cannot be used. Therefore, a bootstrap approach will be used to determine a 95% confidence interval for the Spearman correlations (where data will be resampled on the patient level).

  Baseline, 12 months, 24 months

• The ability of measurements of the changes in the digital biomarkers over the two-year follow-up to predict worsening in the clinical reference test over the same period expressed as binary variables

  The change of the digital biomarker over two years allows to distinguish patients experiencing a relevant worsening in the corresponding reference test over the same period from those who do not with an area under the receiver operating characteristic curve (AUC) larger than 0.6 (lower bound of 95% confidence interval).

  Baseline and 24 months

Secondary Outcomes (6)

• The ability of the digital biomarker to detect worsening in other relevant reference test results creating converging evidence

  up to 24 months

• The ability of the digital biomarker to detect worsening in standard assessments used for treatment of PwMS (clinical, imaging, body fluids)

  up to 24 months

• The ability of the digital biomarker to detect change of Patient Reported Outcomes

  up to 24 months

• The ability of the digital biomarker to detect occurrence of clinical and other meaningful events (relapses, PIRA, serious adverse events, hospitalizations, working capacity)

  up to 24 months

• The relationship of the digital biomarkers with imaging and body fluid markers

  up to 24 months

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Persons with MS from the neurological outpatient clinics in the participating centers (European and North American).

You may qualify if:

• Age ≥18
• Diagnosed with MS according to the revised McDonald criteria 2017, all clinical forms inclusive (CIS, RRMS, SPMS, PPMS)
• In possession of a Healios+Me app-compatible smartphone (iOS/Android)
• Corrected close visual acuity of ≥0.5
• Hand motor skills sufficient for using a smartphone
• Ability and intention to follow the study procedures
• Sufficient knowledge of the language for the specific country
• Informed Consent as documented by signature

Sponsors & Collaborators

• Research Center for Clinical Neuroimmunology and Neuroscience Basel: lead

Study Sites (3)

University of California, San Francisco (UCSF) Weill Institute, Department of Neurology

San Francisco, California, 94158, United States

Location

Innsbruck Medical University, Department of Neurology

Innsbruck, Tyrol, 6020, Austria

Location

Vienna Medical University, Department of Neurology

Vienna, Vienna, 1090, Austria

Location

MeSH Terms

Conditions

Multiple Sclerosis

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Ludwig Kappos, Prof.

  University Hospital, Basel, Switzerland

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 6, 2024
• First Posted: March 13, 2024
• Study Start: May 1, 2026
• Primary Completion (Estimated): December 1, 2029
• Study Completion (Estimated): December 1, 2029
• Last Updated: April 1, 2026

Record last verified: 2026-03

Locations

US (1); AU (2)