SELECTING OUTCOME MEASURES IN PAEDIATRIC MITOCHONDRIAL ENCEPHALOPATHY: A PILOT STUDY

SELECTING OUTCOME MEASURES IN PAEDIATRIC MITOCHONDRIAL ENCEPHALOPATHY: A PILOT STUDY - SO-MITO encephalopathy

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON45750

Enrollment

Registered

2018-06-06

Start date

2019-04-01

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 disturbance

Interventions

None listed

Eligibility

Age

2 Years to 17 Years

Inclusion criteria

Inclusion criteria: - Genetically confirmed mitochondrial disease - Encephalopathy (e.g. psychomotor retardation, epilepsy, abnormalities at neurological examination, etc).

Exclusion criteria

Exclusion criteria: - The treating physician estimates that it is too burdensome for the patient to visit to participate in this study - Vision problems (

Design outcomes

Primary

Measure	Time frame
Functional test (easy to difficult): Accelerometry Spasticity- Tardieu Barry Albright Dystonia Scale Gross Motor Function measure (GMFM) 9 hole peg test 10m walk or run test Scale for the Assessment and Rating of Ataxia (SARA) Gait measurement 6-minute walking test 30 seconds sit to stand test Questionnaires: Pediatric Evaluation of Disabilities Inventory * Computer Adaptive Test (PEDI-CAT) Pediatric Outcomes Data Collection Instrument (PODCI) Caregiver burden scales Newcastle Pediatric Mitochondrial Disease Scale (NPMDS; including short physical examination) International Pediatric Mitochondrial Disease Scale (IPMDS; including short physical examination) Only in young children (	—

Measure

Time frame

Functional test (easy to difficult): Accelerometry Spasticity- Tardieu Barry Albright Dystonia Scale Gross Motor Function measure (GMFM) 9 hole peg test 10m walk or run test Scale for the Assessment and Rating of Ataxia (SARA) Gait measurement 6-minute walking test 30 seconds sit to stand test Questionnaires: Pediatric Evaluation of Disabilities Inventory * Computer Adaptive Test (PEDI-CAT) Pediatric Outcomes Data Collection Instrument (PODCI) Caregiver burden scales Newcastle Pediatric Mitochondrial Disease Scale (NPMDS; including short physical examination) International Pediatric Mitochondrial Disease Scale (IPMDS; including short physical examination) Only in young children (

—

Secondary

Measure	Time frame
Demographic data (age, gender, age at diagnosis, genetic diagnosis, phenotype, height, weight)	—

Countries

Netherlands

Outcome results