Clinical trial NCT05421832: A Novel Measurement Concept to Objectively Quantify Severity of Vocal and Speech Related Symptoms Associated With Parkinson's Disease
Official title: A Novel Measurement Concept to Objectively Quantify Severity of Vocal and Speech Related Symptoms Associated With Parkinson's Disease
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Parkinson Disease
Lead sponsor: Northwestern University
Sponsor class: OTHER
Collaborators: name: Koneksa Health
name: Michael J. Fox Foundation for Parkinson's Research
First posted: 2022-06-16
Enrollment: 120
Sex: ALL
Minimum age (years): 30
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: * PD Cohort I (n=30): PD patients assessed via the Hoehn \& Yahr (H\&Y) Scale to be in Stages 1-2 of PD (inclusive)
* PD Cohort II (n = 30): PD patients assessed via the H\&Y Scale to be in Stages 3-4 of PD (inclusive)
* Prodromal PD Cohort (n = 30): Patients who meet PI-defined criteria for prodromal PD, i.e., the latent phase of disease progression during which clinical PD symptoms have yet to manifest
* Age \& Sex Matched Healthy Control Cohort (n = 30) - age \& sex matched healthy control subjects who have not been diagnosed with PD

Brief summary: The aim of this research program is to develop and validate a smartphone app-based digital measurement concept that:

* Objectively quantifies the severity of Parkinson's Disease (PD) related vocal and speech symptoms;
* Accurately and sensitively identifies vocal and speech abnormalities associated with the prodromal stage of PD.

Detailed description: Although multiple approaches to this problem have been proposed in addition to commercially available speech analytics platforms, there is currently no established measure which incorporates the disparate aspects of affected speech to fully characterize Parkinson's symptom progression, particularly in the prodromal phase.

The measurement concept being evaluated in the present study utilizes a custom smartphone-based speech assessment tool to extract multiple hypothesis-driven acoustic features from patient speech in a real-life environment. The resultant features will be used to train a pair of supervised machine learning models to predict clinical PD symptom severity scores, and to distinguish prodromal PD patients from both healthy matched controls and PD patients in more advanced phases of disease progression.

Eligibility criteria: Inclusion Criteria:

PD:

1. Male or female age 30 years or older at Screening Visit.
2. Diagnosis of PD as defined by MDS PD diagnostic criteria \[1\]
3. PD severity at Screening Visit of either:

   * PD Hoehn and Yahr Stage 1-2, inclusive (PD Cohort I)
   * PD Hoehn and Yahr Stages 3-4, inclusive (PD Cohort II)
4. Able and willing to complete all aspects of the study, including at home smartphone app and Zoom telehealth assessments.
5. Able to provide informed consent.

Prodromal PD:

1. Confirmation that participant is eligible based on clinician determined predictive criteria of known risk of PD including

   1. Rapid eye movement sleep behavior disorder (RBD), possible, probable or definite, OR
   2. Hyposmia defined as less than 10th percentile on University of Pennsylvania Smell Identification Test (UPSIT), age and gender adjusted, OR
   3. Known genetic variants associated with PD risk, AND Confirmed eligible DAT scan.
2. Male or female age 30 or older at Screening Visit.
3. Able and willing to complete all aspects of the study, including at home smartphone app and Zoom telehealth assessments
4. Able to provide informed consent.

Age \& Sex Matched Healthy Control:

1. Male or female age 30 years or older at Screening visit.
2. Able and willing to complete all aspects of the study, including at home smartphone app and Zoom telehealth assessments
3. Able to provide informed consent.

Exclusion Criteria:

PD:

1. Late-stage PD diagnosis (i.e., Hoehn \& Yahr Stage 5) at Screening Visit
2. Symptomatic or atypical PD syndromes due to either drugs (e.g., metoclopramide, flunarizine, neuroleptics) or metabolic disorders (e.g., Wilson's disease), encephalitis, or degenerative diseases (e.g., progressive supranuclear palsy).
3. Current or active clinically significant neurological disorder other than PD (in the opinion of the Investigator).
4. Significant cognitive impairment or clinical dementia at Screening that, in the opinion of the Investigator, would interfere with study evaluation.
5. History of drug and/or alcohol abuse within the past year prior to Screening Visit.
6. Inability or unwillingness to complete all aspects of the study - including use of a provisioned smartphone for study assessments; completion of telehealth assessments.
7. Any other medical or psychiatric condition, which in the opinion of the investigator might preclude participation.

Prodromal PD:

1. Clinical diagnosis of PD, other parkinsonism, or dementia.
2. Any current or active clinically significant neurological disorder (in the opinion of the Investigator).
3. Previously obtained MRI scan with evidence of clinically significant neurological disorder (in the opinion of the Investigator).
4. Significant cognitive impairment or clinical dementia at Screening that, in the opinion of the Investigator, would interfere with study evaluation.
5. History of drug and/or alcohol abuse within the past year prior to Screening Visit.
6. Inability or unwillingness to complete all aspects of the study - including use of a provisioned smartphone for study assessments; completion of telehealth assessments.
7. Any other medical or psychiatric condition, which in the opinion of the investigator might preclude participation.

Age \& Sex Matched Healthy Control:

1. First degree relative with PD (i.e., biologic parent, sibling, child).
2. Any current or active clinically significant neurological disorder (in the opinion of the Investigator).
3. Previously obtained MRI scan with evidence of clinically significant neurological disorder (in the opinion of the Investigator).
4. Significant cognitive impairment or clinical dementia at Screening that, in the opinion of the Investigator, would interfere with study evaluation.
5. History of drug and/or alcohol abuse within the past year prior to Screening Visit.
6. Inability or unwillingness to complete all aspects of the study - including use of a provisioned smartphone for study assessments; completion of telehealth assessments.
7. Any other medical or psychiatric condition, which in the opinion of the investigator might preclude participation

Primary outcomes:
measure: Compliance of digital speech assessment data recorded via smartphone assessments
timeFrame: 8 weeks
measure: Quality of digital speech assessment data recorded via smartphone assessments
timeFrame: 8 weeks
measure: Usability of digital speech assessments
timeFrame: 8 weeks
measure: Content validity of digital speech assessments
timeFrame: 8 weeks
Secondary outcomes:
measure: Characterization and reliability of digital speech assessment features
timeFrame: 8 weeks
measure: Reliability of digital speech assessment features
timeFrame: 8 weeks
measure: Predictive performance of machine learning (ML) regression model
timeFrame: 8 weeks
measure: Predictive performance of ML classification model
timeFrame: 8 weeks
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
Study officials:
References: