Multi-Modal Digital Monitoring of Disease Symptoms in Spinocerebellar Ataxia

September 25, 2026 updated by: BioSensics

This is an observational study. Subjects with a documented diagnosis of spinocerebellar ataxia (SCA) will be screened and recruited to participate at two clinical sites: Johns Hopkins University (JHU) and Columbia University (CU).

Researchers at BioSensics, JHU, and CU will use wearable sensors computerized speech, psychomotor, and ocular assessments to evaluate the feasibility of using digital health technologies to monitor symptoms in SCA. Researchers will also measure the Scale for the Assessment and Rating of Ataxia (SARA) and other SCA clinical assessments.

The solution will be deployed for 25 participants with SCA for approximately 12 months with 5 study visits (Screening/Baseline, Day 1, Day 30, Day 90, Day 180, Day 360).

Study Overview

Status

Not yet recruiting

Conditions

Detailed Description

The spinocerebellar ataxias (SCAs) are a clinically and genetically heterogeneous group of rare, autosomal dominant neurodegenerative disorders characterized by progressive degeneration of the cerebellum and its connecting pathways. The four most common subtypes (SCA1, SCA2, SCA3, and SCA6) are caused by expanded polyglutamine-coding CAG repeats, and collectively account for the majority of dominantly inherited ataxias worldwide. The cardinal feature across subtypes is progressive ataxia affecting gait, stance, limb coordination, and speech, frequently accompanied by oculomotor abnormalities and, in some subtypes, extracerebellar signs such as spasticity, peripheral neuropathy, and parkinsonism. Symptoms typically begin in adulthood and progress over many years, leading to severe disability and reduced life expectancy.

The principal means of measuring ataxia severity is the Scale for the Assessment and Rating of Ataxia (SARA), which is the most widely applied clinical outcome assessment (COA) for genetic ataxias and has been used as the primary endpoint in large natural history cohorts and randomized controlled trials. Although the SARA has demonstrated good inter-rater reliability and has been validated across multiple SCA subtypes, it is inherently subjective and semi-quantitative, requires trained personnel to administer, and may not be sufficiently sensitive to detect incremental but clinically meaningful changes over short follow-up intervals.

Movement-associated symptoms are a core feature of SCA and have a substantial negative impact on quality of life. Impairments in gait and balance contribute to falls and loss of independence, while dysarthria and impaired limb coordination affect communication and activities of daily living. Because these assessments are conducted infrequently, typically in a clinic setting, they may not accurately represent an individual's true functional status during everyday life. However, the increasing understanding of disease pathomechanisms and the development of gene-targeting and other novel therapeutic approaches have raised expectations for disease-modifying treatments. The key challenge in developing new therapeutics for SCA and other neurological disorders is that current disease assessment is largely subjective and requires in-person, in-clinic evaluation.

Digital measures, such as those proposed in this study, can provide objective, sensitive, and real-world assessments of how individuals are functioning. A growing body of evidence demonstrates that digital biomarkers derived from body-worn inertial sensors can quantify ataxia-specific gait and balance impairments, and correlate strongly with clinician-rated SARA scores.

Beyond gait and balance, multi-modal digital assessment of speech, hand function, and oculomotor control may capture complementary domains of cerebellar dysfunction that are affected in SCA. Acoustic analysis of speech can quantify dysarthria, finger tapping can characterize upper-limb bradykinesia and dysrhythmia, and oculomotor testing can capture saccadic and antisaccadic abnormalities that are characteristic of cerebellar disease.

Thus, a system that can objectively, sensitively, and frequently monitor multiple clinical domains (motor, speech, and oculomotor function) is necessary to understand the pattern of evolving impairment in individuals with SCA and to support the identification of endpoint measures for therapeutic trials. This study will evaluate the feasibility of a multi-modal digital monitoring platform in a cohort of individuals with SCA and will characterize the relationship between digital measures and established clinical and patient reported outcomes.

Study Type

Observational

Enrollment (Estimated)

25

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Maryland
      • Lutherville, Maryland, United States, 21093
        • Johns Hopkins University School of Medicine
        • Contact:
        • Principal Investigator:
          • Liana Rosenthal, MD PhD
    • New York
      • New York, New York, United States, 10032
        • Columbia University Irving Medical Center/Neurological Institute of New York
        • Principal Investigator:
          • Sheng-Han Kuo, MD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Adults with SCA

Description

Inclusion Criteria:

  • Male or female, aged 18-75 years
  • Fluent in English (able to speak and read)
  • Able to ambulate at least 10 meters independently or with a cane, without other assistive devices, as determined by the investigator
  • Able to independently perform all study activities safely, as determined by the investigator
  • Willing and able to provide informed consent and comply with all study procedures
  • Genetically or clinically confirmed diagnosis of spinocerebellar ataxia (SCA 1, 2, 3, or 6)

Exclusion Criteria:

  • Individuals who are unable to ambulate at least 10 meters independently or with a cane.
  • Individuals with a neurological, medical, or psychiatric condition (other than SCA) that, in the investigator's judgment, would interfere with safe participation in study activities or confound the assessment of ataxia
  • Pregnant individuals, due to potential changes in gait and physical activity during pregnancy
  • Any condition that, in the investigator's judgment, would prevent the participant from completing the study procedures safely

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: COHORT
  • Time Perspectives: PROSPECTIVE

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Physical Activity Monitoring - Daily Number of Steps (count)
Time Frame: 12 months

Following each clinical visit, participants will be asked to wear a PAMSys pendant sensor, PAMSys ULM wrist sensors, and PAMSys Gait for 7 days at home. The average daily number of steps will be measured using the PAMSys pendant.
12 months
Physical Activity Monitoring - Total number of goal-directed movement (GDM)
Time Frame: 12 months

Following each clinical visit, participants will be asked to wear a PAMSys ULM sensor, a wearable sensor for long-term monitoring of upper limb and activities of daily living (ADL). The sensor will record the daily number GDMs as participants perform ADLs.
12 months
Physical Activity Monitoring - Stride Velocity (m/s)
Time Frame: 12 months

Following each clinical visit, participants will be asked to wear a PAMSys Gait sensor, a wearable sensor for long-term monitoring of gait during activities of daily living (ADL). The sensor will record the average stride velocity (m/s) during ADLs.
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Scale for the Assessment and Rating of Ataxia (SARA)
Time Frame: 12 months

The SARA is an 8-item clinical sale to measure severity and progression of cerebellar ataxia. Total scores range from 0 (no ataxia) to 40 (most severe ataxia).
12 months
Digital Speech Assessment - Intelligibility (%)
Time Frame: 12 months

Using a study tablet, participants will read the Rainbow Passage. Intelligibility is a digital measure of speech clarity that determines how easily a listener can understand spoken words. Intelligibility ranges scores from 0 to 100%
12 months
1 Minute Walk Test - Gait Speed (m/s)
Time Frame: 12 months

Performed at each clinical visit, the participant will complete the 1 Minute Walk Test. Gait speed will be measured using LEGSys.
12 months
Digital Occulomotor Assessments - Median velocity (pixel/s)
Time Frame: 12 months

Using a study tablet, participants will perform a series of oculomotor assessments (saccades). The video will be analyzed to calculate the median velocity of the pupil during saccade movements.
12 months
EuroQoL 5-Dimension 5-Level questionnaire (EQ-5D-5L)
Time Frame: 12 months

The EQ-5D-5L is a standardized 5-item tool used to measure health-related quality of life. EQ-5D-5L scores are interpreted by analyzing three distinct components: a 5-digit health profiles. Each dimension uses five severity levels (1 = no problems, 2 = slight, 3 = moderate, 4 = severe, 5 = extreme/unable to).
12 months
1 Minute Walk Test with Cognitive Load- Gait Speed (m/s)
Time Frame: 12 months

Performed at each clinical visit, the participant will complete the 1 Minute Walk Test with cognitive load (counting backwards). Gait speed will be measured using LEGSys.
12 months
Timed Up and Go (TUG) - Sit to Stand duration (s)
Time Frame: 12 months

Performed at each clinical visit, the participant will complete the TUG Test. Sit to stand duration will be measured using LEGSys.
12 months
30S Chair Sit to Stand - Sit to Stand duration (s)
Time Frame: 12 months

Performed at each clinical visit, the participant will complete the 30S Chair Sit to Stand Test. Sit to stand duration will be measured using LEGSys.
12 months
Digital Occulomotor Assessment - Median duration (s)
Time Frame: 12 months

Using a study tablet, participants will perform a series of oculomotor assessments (saccades). The video will be analyzed to calculate the median duration of the pupil during saccade movements.
12 months
EQ-VAS (Visual Analog Scale)
Time Frame: 12 months

The EQ VAS: The participant self-rates their overall health on a vertical thermometer-like scale from 0 to 100, where 100 is the best health the participant can imagine, and 0 is the worst health the participant can imagine.
12 months

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Digital Fine Motor Control Assessment - Number of taps (count)
Time Frame: 12 months

Using a study tablet, participants will perform a series of tapping tests. The tablet will calculate the count of all recorded taps made by a finger on the screen.
12 months
Digital Fine Motor Control Assessment - Average tap rate (s)
Time Frame: 12 months

Using a study tablet, participants will perform a series of tapping tests. The tablet will calculate the number of taps per second.
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

BioSensics

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

December 15, 2026

Primary Completion (Estimated)

December 31, 2028

Study Completion (Estimated)

January 31, 2029

Study Registration Dates

First Submitted

September 16, 2026

First Submitted That Met QC Criteria

September 25, 2026

First Posted (Actual)

October 1, 2026

Study Record Updates

Last Update Posted (Actual)

October 1, 2026

Last Update Submitted That Met QC Criteria

September 25, 2026

Last Verified

September 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BioDigit SCA-02

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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