Selecting Outcome measures for MITOchondrial disease in children: an explorative study

Selecting Outcome measures for MITOchondrial disease in children: an explorative study - SO-MITO study

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON43301

Enrollment

Registered

2016-11-14

Start date

2017-09-27

Completion date

Unknown

Last updated

2024-04-15

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

Conditions

energy metabolism disorders mitochondriopathy

Interventions

None listed

Eligibility

Age

2 Years to 17 Years

Inclusion criteria

Inclusion criteria: -17 years old at the inclusion date * Mitochondrial myopathy o - Signs of myopathy on examination (muscle weakness, hypotonia) or testing (exercise intolerance). o - No signs or history of encephalopathy (IQ

Exclusion criteria

Exclusion criteria: * Insufficient knowledge of the Dutch language * It is expected that the studies will be too burdensome for the patient or the family * End-of-life expected within 3 months from the initiation of the study * Other disabling disease * Gluten allergy (for TOMASS cracker test only)

Design outcomes

Primary

Measure	Time frame
Feasibility (primary endpoint; % of patients who were able to complete the test), reliability (inter-rater reliability, intra-rater reliability, test-retest reliability), and validity (correlation with predefined anchors and between parameters measuring the same construct) of all instruments tested in this study, per study group.	—

Countries

Netherlands

Outcome results