Overview
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This study validates a telephone-based cognitive assessment battery for Progressive Supranuclear Palsy, enabling remote evaluation and expanding access to clinical trials.
Study Details
- NCT Number: NCT06932809
- Status: Recruiting
- Study Type: Observational
- Conditions: PSP, CBD, FTD
Background and Rationale
Cognitive impairment is a core feature of Progressive Supranuclear Palsy that significantly impacts patient quality of life and functional independence. The standard approach to cognitive assessment in clinical trials has traditionally required in-person visits to specialized research centers, creating substantial barriers to patient participation[@telephone].
Challenges with In-Person Assessment
The conventional model of cognitive testing in PSP faces several limitations that this study aims to address:
Geographic Constraints: PSP is a rare disorder with a prevalence of approximately 5-6 per 100,000 population. Patients often need to travel significant distances to reach specialized centers with expertise in atypical parkinsonism. This geographic barrier is particularly pronounced given the motor impairments that characterize PSP, including gait instability and oculomotor dysfunction that make travel difficult[@steele2021].
Disease Progression Impact: As PSP advances, patients experience progressive gait impairment and balance dysfunction that makes office visits increasingly challenging. The very symptoms that define the disease—vertical gaze palsy, postural instability, and akinesia—also create practical obstacles to attending research appointments.
Caregiver Burden: Participation in clinical trials often requires substantial caregiver involvement, including transportation, accompaniment during visits, and time commitment. For progressive neurological conditions like PSP, this burden increases as the disease advances.
Trial Accessibility: Traditional trial designs require repeated in-person visits for cognitive assessment, limiting the ability to conduct frequent monitoring and reducing the pool of eligible participants.
The Need for Remote Assessment
The development of validated telephone-based cognitive assessments could transform PSP clinical trials by enabling virtual participation models. This approach has particular relevance for:
Decentralized Trials: Remote assessment enables decentralized trial designs that reduce site dependence and expand geographic reach.
Longitudinal Monitoring: More frequent cognitive assessments could provide finer granularity for detecting subtle changes over time.
Patient Centricity: Reducing visit burden improves patient experience and may increase retention in long-term studies.
Cost Efficiency: Virtual assessment models can substantially reduce the costs associated with traditional site-based cognitive testing[@bannon2023].
Study Design and Methodology
Study Type and Duration
This is an observational validation study designed to establish the psychometric properties of a telephone-based cognitive assessment battery for PSP. The study enrolls patients from multiple sites and conducts both telephone and in-person assessments within a short interval to establish equivalence.
Assessment Battery Components
The telephone-based cognitive assessment battery includes several validated instruments adapted for remote administration:
Verbal Memory Tests: Paragraph recall and word list learning tests assess episodic memory function, which is prominently affected in PSP. The telephone versions use auditory presentation rather than visual, requiring adaptation of standard protocols["@wylie2022"].
Executive Function Measures: Trail-making tests adapted for verbal administration, phonemic fluency (e.g., generating words beginning with specific letters), and working memory tasks assess prefrontal cortical function. Executive dysfunction is a hallmark of PSP-related cognitive impairment.
Language Assessment: Naming tests, comprehension checks, and semantic fluency (e.g., naming animals in a minute) evaluate language function. While language is relatively preserved in classic PSP compared to some other parkinsonian syndromes, subtle changes can be detected with sensitive measures.
Behavioral Questionnaires: Telephone administration of standardized behavioral assessments evaluates apathy, disinhibition, and other behavioral changes that commonly accompany PSP["@litvan2017"].
Validation Procedures
The study employs a crossover design where participants complete both telephone and in-person cognitive assessments. The order of assessment modality is counterbalanced across participants to control for practice effects.
Test-Retest Reliability: Administration of the same battery twice within one week establishes test-retest reliability coefficients for each measure.
Parallel Forms: Alternate versions of tests are available to reduce practice effects in repeated assessments.
Validity Assessment: Correlation between telephone and in-person scores establishes criterion validity. Sensitivity to disease severity is evaluated by correlating cognitive scores with established clinical measures.
Population and Eligibility
Inclusion Criteria
The study enrolls patients meeting the following criteria:
- Age 40-85 years
- Clinical diagnosis of PSP (Richardson syndrome or variant) per established criteria
- Diagnosis confirmed by movement disorder specialist
- MMSE score ≥ 15 (allow for moderate cognitive impairment)
- Access to telephone
- Willingness to complete both telephone and in-person assessments
- Informed consent from patient and caregiver
Exclusion Criteria
- Severe auditory impairment preventing telephone communication
- Severe language impairment precluding valid assessment
- Active psychiatric disorder affecting cognition
- Unstable medical condition affecting cognitive function
- Participation in interventional trial within 30 days
Target Enrollment
The study aims to enroll approximately 100 participants across multiple sites to provide adequate statistical power for validation analyses. This sample size allows detection of moderate correlation coefficients with 80% power.
Outcome Measures
Primary Outcomes
- Correlation coefficient between telephone and in-person cognitive scores
- Test-retest reliability coefficients for telephone battery
- Sensitivity to disease severity (correlation with PSP Rating Scale)
Secondary Outcomes
- Performance comparison across PSP subtypes
- Relationship with caregiver-reported functional measures
- Comparison with in-person cerebrospinal fluid or imaging biomarkers
Exploratory Outcomes
- Feasibility metrics (completion rates, time burden)
- Subgroup analyses by disease stage
- Development of composite cognitive index
Clinical Significance
Impact on PSP Clinical Trials
Successful validation of telephone cognitive assessment for PSP would have substantial implications for clinical trial design:
Virtual Trial Capabilities: Trial designs incorporating remote cognitive assessment could reduce site visits to only essential motor and imaging evaluations, potentially enabling fully virtual designs for certain endpoints.
Expanded Access: Patients unable to travel to specialized centers could participate in trials, accelerating enrollment and improving generalizability.
More Frequent Monitoring: Serial cognitive assessments could provide detailed trajectories not achievable with quarterly in-person visits.
Cost Reduction: Reduced site visits translate to substantial cost savings in large-scale trials.
Clinical Utility Beyond Trials
Beyond clinical trials, validated telephone cognitive assessment could improve:
Clinical Care: Remote monitoring of cognitive function in routine clinical practice.
Natural History Studies: More frequent cognitive assessments in observational studies.
Telemedicine: Integration with telehealth platforms for remote neurological care.
See Also
- [Alzheimer's Disease](/diseases/alzheimers-disease)
- [Parkinson's Disease](/diseases/parkinsons-disease)
External Links
- [PubMed](https://pubmed.ncbi.nlm.nih.gov/)
- [KEGG Pathways](https://www.genome.jp/kegg/pathway.html)
References
Unknown, Telephone Cognitive Assessment - ClinicalTrials.gov (n.d.)
Steele L, et al, Prevalence and incidence of progressive supranuclear palsy: a systematic review (2021)
Bannon L, et al, Remote cognitive assessment in neurodegenerative diseases: a review of current capabilities and future directions (2023)
Wylie SA, et al, Telephone-based cognitive assessment in atypical parkinsonism: validation study (2022)
Litvan I, et al, Diagnostic criteria for progressive supranuclear palsy (2017)Pathway Diagram
The following diagram shows the key molecular relationships involving Telephone Cognitive Assessment in PSP (NCT06932809) discovered through SciDEX knowledge graph analysis:
Mermaid diagram (expand to render)