Clinical trial NCT06309173: Development of Digital Biomarkers in Multiple Sclerosis: Validation Study 2
Official title: Development of Digital Biomarkers in Multiple Sclerosis: Validation Study 2
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Multiple Sclerosis
Keywords: Digital Biomarkers
Lead sponsor: University Hospital, Basel, Switzerland
Sponsor class: OTHER
First posted: 2024-03-13
Enrollment: 600
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Persons with MS from the neurological outpatient clinics in the participating centers (European and North American).

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.

Detailed description: Multiple sclerosis (MS) is a chronic inflammatory disease of the central nervous system. Focal demyelination and diffuse neurodegeneration in the gray and white matter of the brain and spinal cord lead to decreased physical and cognitive functioning and disability.

Diagnosis is established by clinical assessment of persons with MS (PwMS), including taking their history and neurologic examination, in combination with imaging and body fluid assessments. Treatment decisions in MS are based on continuous monitoring of MS disease activity and progression through clinical and imaging assessments, while the role of body fluid assessments is not yet universally established. The predictive and prognostic value of these currently used assessments to individualize treatment decisions is still very limited. New and more reliable measures are needed, especially indicators of disease progression, which allow to personalize therapies and care.

With the digitization of healthcare, new opportunities are emerging to provide quasi-continuous and granular health status measurements as digital measures. Such digital measures may allow to monitor MS disease activity and progression more informatively than the only episodical traditional assessments. Digital health technologies allow to remotely capture dynamic fluctuating functions and symptoms in real world settings with minimal disruption of patients' life and usual care.

The investigators developed the dreaMS software program that includes app-based interactions with the patients, patient-reported information via surveys, and continuous monitoring through sensors. In a previous study (NCT04413032), which evaluated the feasibility and acceptance of using our dreaMS software program, we identified digital biomarkers (DB) that are relevant for PwMS. These DB are evaluated and validated in an ongoing study including a larger population of PwMS in Switzerland (NCT05009160).

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.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* NA

Primary outcomes:
measure: Correlation of the digital features with the respective measurements of the clinical reference tests
timeFrame: Baseline, 12 months, 24 months
measure: 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
timeFrame: Baseline and 24 months
Secondary outcomes:
measure: The ability of the digital biomarker to detect worsening in other relevant reference test results creating converging evidence
timeFrame: up to 24 months
measure: The ability of the digital biomarker to detect worsening in standard assessments used for treatment of PwMS (clinical, imaging, body fluids)
timeFrame: up to 24 months
measure: The ability of the digital biomarker to detect change of Patient Reported Outcomes
timeFrame: up to 24 months
measure: The ability of the digital biomarker to detect occurrence of clinical and other meaningful events (relapses, PIRA, serious adverse events, hospitalizations, working capacity)
timeFrame: up to 24 months
measure: The relationship of the digital biomarkers with imaging and body fluid markers
timeFrame: up to 24 months
measure: The relationship of the digital biomarkers with Patient Reported Outcomes
timeFrame: up to 24 months
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: University of California, San Francisco (UCSF) Weill Institute, Department of Neurology
state: California
city: Innsbruck
country: Austria
facility: Innsbruck Medical University, Department of Neurology
state: Tirol
city: Wien
country: Austria
facility: Vienna Medical University, Department of Neurology
Study officials:
affiliation: University Hospital, Basel, Switzerland
name: Ludwig Kappos, Prof.
role: STUDY_DIRECTOR
References: