Optimization of the International Paediatric Mitochondrial Disease Scale

• Conditions: energy metabolism disturbances; 10027424

• Registration Number: NL-OMON43155
• Lead Sponsor: niversitair Medisch Centrum

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 28 February 2024

Recruitment & Eligibility

• Status: Pending
• Sex: Not specified
• Target Recruitment: 195

Inclusion Criteria

- Study i) Healthy children aged 0-18 years.
- Study ii) Children with a mitochondrial disease admitted for the MItoRoute
- Study iii) Children with a mitochondrial encephalopathy with abnormalities at the neurological examination, admitted for the MItoRoute

Exclusion Criteria

- Study i) Having a known health condition
- Study ii) and iii) The study is estimated to be too burdensome to the patient

Study & Design

• Study Type: Observational non invasive
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>International Paediatric Mitochondrial Disease Score (IPMDS)<br /><br>The newly developed International Paediatric Mitochondrial Disease Score<br /><br>(IPMDS) was designed to follow children with a mitochondrial disease in e.g.<br /><br>clinical trials and detailed natural history studies. It consist of the<br /><br>following subdomains: 1. Complaints and symptoms; 2. Physical examination and<br /><br>3. Functional tests. Its reliability and validity are to be tested in this<br /><br>study.<br /><br>The scale takes about 35 minutes to perform, of which 25 for Domain 1 and 10<br /><br>for Domain 2 and 3 each. The patient should be involved in the assessment of<br /><br>Domain 2 and 3 but not necessarily for Domain 1. </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>na</p><br>