Mitochondrial Video Assessment Source Material Study

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

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT06180421

Enrollment

Registered

2023-12-22

Start date

2023-06-16

Completion date

2024-02-02

Last updated

2024-11-14

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

PolG, Primary Mitochondrial Myopathy

Keywords

PoIG, Primary Mitochondrial Myopathy, MELAS, CPEO

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

16 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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.

Countries

United States

Outcome results