Mitochondrial Video Assessment Source Material Study

December 12, 2023 updated by: The Emmes Company, LLC

Development of the Mitochondrial Video Assessment (MVA) as an Outcome Measure for the Evaluation of Patients With Mitochondrial Myopathies

Source material collection of videos to develop the Mitochondrial Video Assessment (MVA) for patients with mitochondrial myopathies.

Study Overview

Status

Recruiting

Conditions

Detailed Description

This observational research is being conducted to facilitate the development of 2 related, disease-specific outcome measures assessing ease of movement in PMM and PolG clinical trials. The Mitochondrial Video Assessment (MVA) utilizes remote video capture to assess subtle shifts in compensatory movement patterns and measure change in function over time, and the Current State Questionnaire asks participants about external factors in their daily lives which may influence fluctuations in their symptoms. As part of the questionnaire, participants are asked to rate their own perception of their functional ability using a Patient Global Impression of Severity (PGI-S) scale.

Study Type

Observational

Enrollment (Estimated)

50

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 Contact Backup

Study Locations

  • United States
    • Massachusetts
      • Kingston, Massachusetts, United States, 02360
        • Recruiting
        • Casimir
        • Contact:
          • Lauren Carroll

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

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Persons aged 16 and older living with a confirmed diagnosis of PMM or PoIG.

Description

Inclusion Criteria:

  • 16 years old and above
  • Confirmed diagnosis of PMM or PolG
  • Ability to understand and speak English
  • Ability and willing to consent
  • Able and willing to participate and perform tasks while being recorded
  • Designating a recording partner to assist with video captures is strongly advised, but not required

Exclusion Criteria:

  • N/A

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

Cohorts and Interventions

Group / Cohort
PolG
Genetically confirmed PoLG patients 16 years or older
PMM
Genetically confirmed Primary Mitochondrial Myopathy patients 16 years or older

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Source Material Videos
Time Frame: 2 weeks
Initiate feasibility testing and collect source material videos from patients with Primary Mitochondrial Myopathy (PMM, n=35) or a PolGamma (PolG) mutation (n=15) to facilitate development of the Mitochondrial Video Assessment (MVA).
2 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
PGI-S
Time Frame: 2 weeks
Identify external factors which may influence participant performance on functional assessments and collect Patient Global Impression of Severity (PGI-S) at the time of each capture to facilitate the development of a patient-reported Current State Questionnaire to be deployed in conjunction with the MVA.
2 weeks

Collaborators and Investigators

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

Sponsor

The Emmes Company, LLC

Collaborators

United Mitochondrial Disease Foundation
PolG Foundation Mito Australia

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 (Actual)

June 16, 2023

Primary Completion (Estimated)

January 12, 2024

Study Completion (Estimated)

February 23, 2024

Study Registration Dates

First Submitted

December 12, 2023

First Submitted That Met QC Criteria

December 12, 2023

First Posted (Actual)

December 22, 2023

Study Record Updates

Last Update Posted (Actual)

December 22, 2023

Last Update Submitted That Met QC Criteria

December 12, 2023

Last Verified

December 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CAS-CAS009

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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