AzTrials
AzTrials

Myasthenia GravisNCT04590716

Help Build an A.I. Model to Predict Myasthenia Gravis Symptom Patterns and Flares

An observational study of Myasthenia Gravis, run by doc.ai inc at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

There are limited objective measurements of MG symptoms as well as a dearth of data at a granular level of MG (myasthenia gravis) symptoms and triggers occurring longitudinally. This study is designed to use the strengths of mobile smartphones which enable participant-driven real time capture of data manually and… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myasthenia Gravis
What is being tested?
Data Collection
How many people
113 participants
Where
1 siteUnited States
Study dates
2 Oct 2020to 26 Jul 2021
Phase
Observational
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Study population: Patients with myasthenia gravis (MG) who meet the inclusion criteria will be invited to join this digital health trial. There will be a web pre-screening link where potential participants will self-screen to see if they meet the basic eligibility criteria for this study. The recruitment tool for this trial is developed for diversity, fairness, and inclusion. With the aim to ensure diversity in the demographics of the trial to better understand the health needs of different populations. So, while some interested potential participants do qualify, they may not be invited into the trial due to these diversity requirements.

Who may be able to join

  • Must have a documented diagnosis of Myasthenia Gravis
  • Must have ocular (eye drooping) and/or bulbar (speech) symptoms
  • Must be over the age of 18
  • Must reside in the US for the duration of the study
  • Must be able to read, understand, and write in English
  • Must have a smartphone supported by the doc.ai research app (iOS and Android)

Who cannot join

  • None

Trial locations and contacts

1 site in 1 country.

United States1 site
  • Doc.Ai Mobile Based

    Palo Alto, California, 94301

Other trials in United States

About this study

Official title: A Digital Health Trial That Assesses Participant-driven Data Collection Using Smartphone Modules to Characterize Myasthenia Gravis Symptoms and Develop an A.I. Model to Predict Flares

There are limited objective measurements of MG symptoms as well as a dearth of data at a granular level of MG (myasthenia gravis) symptoms and triggers occurring longitudinally.

This study is designed to use the strengths of mobile smartphones which enable participant-driven real time capture of data manually and through augmented sensors such as video and audio, in order to better characterize MG symptoms and flares.

The study aims to enroll approximately 200 participants for approximately 9 months until analyzable data is available from at least 100 participants. Participants will complete in-app surveys for 3 months with, audiovisual recording of symptoms. This will take approximately 35 minutes per week after the initial survey.

Read the detailed description

Using their smartphones, potential participants will download the doc.ai research mobile app. There will be a web pre-screening link where potential participants will self-screen to see if they meet the basic eligibility criteria for this study. The recruitment tool for this trial is developed for diversity, fairness, and inclusion. With the aim to ensure diversity in the demographics of the trial to better understand the health needs of different populations. So, while some interested potential participants do qualify, they may not be invited into the trial due to these diversity requirements.

Patients with myasthenia gravis (MG) who meet the inclusion criteria will be invited to join this digital health trial. Participants will sign the e-consent and self-enroll into the study. Once their eligibility is confirmed by the study team (to ensure eligibility criteria and validity of participant i.e. eliminate robo sign-ins) they will be asked to take a selfie, provide documented proof of MG diagnosis, respond to a series of survey questions regarding their demographics, current health, medical history, and other MG related information.

Enrolled participants will have a daily brief check-in, 2 weekly check-ins and a weekly check-in which will include an audiovisual check-in, and will maintain an audiovisual diary to keep track of their symptoms, connect data, record their voice (to detect vocal symptoms: weakness, nasality) and take videos of their face (to detect facial symptoms: ocular, mouth droop) on a daily to weekly basis through the various data collecting modules in the doc.ai research app for the duration of their study participation. doc.ai's digital health trial platform will be leveraged to collect this data.

The study aims to enroll approximately 200 participants in approximately 9 months. It is expected that a minimum of 100 participants will be included in the final analysis as at any given time there will be a lag between potential participants expressing interest in the study, their eligibility being assessed by the PI, and participants completing all required study procedures.

At the end of their participation, participants will be asked to complete a questionnaire. After the participant has completed their final survey, they will be able to connect to a link redeemable as an Amazon.com gift card worth $250. All participants will also receive an end-of-trial-summary of the data that they had collected during the study. No medical advice or direction will be given based on this study.

In addition, in the final survey participants will be asked if they would be willing to complete a usability interview after their participation in this trial has ended. This subset of participants invited to be part of a follow-up usability interview will include those who complete all study required procedures and some who may not have completed all study required procedures, in order to assess usability experience of the app for the duration of their participation. Participants will be contacted at their end of their period of participation until a total of 10-15 participants successfully complete the usability interview. Participants who successfully complete the usability interview will receive a link for a $50 in Amazon.com gift card via the app.

For this study the data and, audio and video recordings will be captured directly on the doc.ai research app and securely stored on a HIPAA compliant cloud provider (Google Cloud Platform).

This data will be used to understand the patterns of symptoms and triggers in order to better characterize factors such as the length and timing of flares and any unique symptom patterns in order to create more objective measures of MG symptoms. Ultimately this data would be used to build a machine learning model that could predict MG symptom flares.

Primary Objective:

Use a collection of digital health modules on the smartphone to collect myasthenia gravis (MG) symptoms and triggers to better characterize symptom patterns and flares.

Secondary Objective:

Use the data collected to develop an A.I. model to detect and/or predict symptom flares.

Being tested
Data Collection
Medical subject headings
Myasthenia Gravis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Data CollectionOther

    This is a non-interventional study conducted on the participant's smartphones to record MG related symptoms and conditions.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Audiovisual recording of voice exercises to detect patterns and changes in voice and facial symptoms

    participants to complete the audio and visual data modules designed to capture patient MG symptoms (especially ocular and voice).

    e.g

    • Vocal e.g.:
    • Say "papapapa" for 4 seconds
    • Say "tatatatata" for 4 seconds
    • Say "kakakaka" 4 seconds
    • Say "mamamama" 4 seconds
    • Say "papapapa" 4 seconds
    • Say "buttercup, buttercup, buttercup" 4 seconds
    • Say "aaaahhh" and hold it as long as you can
    • Counting e.g.:
    • Look straight at the camera for 4 seconds
    • Count as precisely as possible from 1 to 25 while looking up
    • Look straight at the camera for 4 seconds

    The recordings will be used to detect change from baseline and any patterns that may occur. This will be used to analyze where and if different features are linked to see if a single or combined effect of the features is connected to flare frequency and/or severity.

    Measured: After enrollment, 3 months with in-app twice a week audiovisual recording of symptoms.

Secondary outcomes (1)
  1. Completion of MG-Quality of Life assessment

    Participants complete MG activities of Daily living and MG-Quality of Life assessments weekly. This assessment has been adapted from www.myasthenia.org/healthprofessionals/educationalmaterials.aspx

    Measured: Approximately 10 minutes each week for 3 months.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Oct 2020
  2. Study start2 Oct 2020
  3. Primary completion26 Jul 2021
  4. Study completion26 Jul 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
113 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04590716
Sponsor's protocol ID
DOC-005-2020
Status last verified
Jul 2021
First submitted
2 Oct 2020
Last updated
29 Jul 2021
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Data will be reviewed, and analysis will be done by personnel of doc.ai, and the medical experts. Population-level results of the data analysis in the form of a presentation/report, as well as the resulting proof-of-concept predictive AI model, will be shared with UCB Biopharma (SRL) (who are funding this study). No participant PII or PHI will be shared with UCB Biopharma (SRL) or any other 3rd parties.

Publications

  • Kent RD, Kent JF, Rosenbek JC. Maximum performance tests of speech production. J Speech Hear Disord. 1987 Nov;52(4):367-87. doi: 10.1044/jshd.5204.367.

    PubMed 3312817
  • Konopka BM, Lwow F, Owczarz M, Laczmanski L. Exploratory data analysis of a clinical study group: Development of a procedure for exploring multidimensional data. PLoS One. 2018 Aug 23;13(8):e0201950. doi: 10.1371/journal.pone.0201950. eCollection 2018.

    PubMed 30138442
  • Zhou ZR, Wang WW, Li Y, Jin KR, Wang XY, Wang ZW, Chen YS, Wang SJ, Hu J, Zhang HN, Huang P, Zhao GZ, Chen XX, Li B, Zhang TS. In-depth mining of clinical data: the construction of clinical prediction model with R. Ann Transl Med. 2019 Dec;7(23):796. doi: 10.21037/atm.2019.08.63.

    PubMed 32042812
  • Kang H. The prevention and handling of the missing data. Korean J Anesthesiol. 2013 May;64(5):402-6. doi: 10.4097/kjae.2013.64.5.402. Epub 2013 May 24.

    PubMed 23741561
  • Borza D, Darabant AS, Danescu R. Real-Time Detection and Measurement of Eye Features from Color Images. Sensors (Basel). 2016 Jul 16;16(7):1105. doi: 10.3390/s16071105.

    PubMed 27438838
  • Hegde S, Shetty S, Rai S, Dodderi T. A Survey on Machine Learning Approaches for Automatic Detection of Voice Disorders. J Voice. 2019 Nov;33(6):947.e11-947.e33. doi: 10.1016/j.jvoice.2018.07.014. Epub 2018 Oct 11.

    PubMed 30316551
  • Duffy, JR: Motor Speech Disorders. Substrates, Differential Diagnosis and Management (1st ed). St. Louis, 1995, Mosby.

  • Duffy, JR: Motor Speech Disorders. Substrates, Differential Diagnosis and Management (2nd ed). New York, 2005, Elsevier Health Sciences.

  • T. Baltrusaitis, A. Zadeh, Y. C. Lim and L. Morency,

  • Panayotov V., Chen G., Povey D., Khudanpur S. (2015). Librispeech: an ASR corpus based on public domain audio books, in Proceedings of the ICASSP (South Brisbane, QLD:), 5206-5210

  • Steyaert S, Lootus M, Sarabu C, Framroze Z, Dickinson H, Lewis E, Steels JC, Rinaldo F. A decentralized, prospective, observational study to collect real-world data from patients with myasthenia gravis using smartphones. Front Neurol. 2023 Aug 1;14:1144183. doi: 10.3389/fneur.2023.1144183. eCollection 2023.

    PubMed 37588667

Questions about this trial

What is this clinical trial studying?

An observational study of Myasthenia Gravis, run by doc.ai inc at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 29 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Audiovisual recording of voice exercises to detect patterns and changes in voice and facial symptoms (time frame: After enrollment, 3 months with in-app twice a week audiovisual recording of symptoms.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04590716, which also lists 11 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jul 2021.

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