Mitochondrial Video Assessment Source Material Study

Completed

• Conditions: PolG, Primary Mitochondrial Myopathy

• Registration Number: NCT06180421
• Lead Sponsor: The Emmes Company, LLC

Brief Summary

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

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 Timeline

• Study Start: 2023-06-16
• Study First Submitted: 2023-12-12
• Study First Submitted QC: 2023-12-12
• Study First Posted: 2023-12-22
• Primary Completion: 2024-02-02
• Study Completion: 2024-02-02
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-12
• Last Update Posted: 2024-11-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 28

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Source Material Videos	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).

Secondary Outcome Measures

Name	Time	Method
PGI-S	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.

Trial Locations

Locations (1)

Casimir; 🇺🇸 Kingston, Massachusetts, United States