To test the reliability and validity of the IPMDS in children with mitochondrial disorders.
ID
Source
Brief title
Condition
- Metabolic and nutritional disorders congenital
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The main study endpoints will be the (inter- and intrarater) reliability and
(construct and external) validity of the IPMDS.
Secondary outcome
na
Background summary
The currently used Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) is
designed to follow up patients at the outpatient clinic. It is quick to
administer, but thereby lacks detail and is in our opinion therefore not
suitable for clinical trials. We designed a new, more detailed scoring system
(the International Paediatric Mitochondrial Disease Score (IPMDS)) for
follow-up of children with a mitochondrial disorder in clinical studies in
which more detail is required.
Study objective
To test the reliability and validity of the IPMDS in children with
mitochondrial disorders.
Study design
The study will take place at the outpatient clinic, where four independent
physicians involved in the care of patients with mitochondrial disorders will
perform the test (interrater reliability). Two physicians will be videotaped
and will score the video again after four weeks (intrarater reliability). The
construct validity will be calculated by correlating with the previous NPMDS
scale, with the Paediatric Evaluation of Disability Inventory (PEDI) score and
with the mean severity score (Likert scale 0-10, scored by every physician).
External validity and interrater reliability will be tested in several clinics
specialized in mitochondrial disorders around the world. Test-retest
reliability will be assessed for the subjective domain only.
Study burden and risks
Patients are asked to visit the outpatient clinic to participate in this study.
They will see four physicians, asking approximately the same questions and
performing the same physical examination. Since patients with mitochondrial
disorder tire easily, this study will be burdensome to them. We will limit the
tiredness by pausing regularly. The burden for parents will also be quite high,
since they are asked the same questions four to six times that day. We will
explain this in our recruitment letter so patients and their parents can decide
whether they want to participate.
The risks associated with this study are negligible because no invasive
procedures or interventions are involved. Since this study aims to validate a
scoring system specific to this group, the study cannot be performed in adults
or healthy children. The only benefit from this study is a more complete
overview of the patient*s health status than can be obtained at a regular
outpatient clinic visit. Therefore, a summary letter of all findings will be
written to all physicians caring for the patient.
Geert Grooteplein 10 route 804
Nijmegen 6500HB
NL
Geert Grooteplein 10 route 804
Nijmegen 6500HB
NL
Listed location countries
Age
Inclusion criteria
1. 0 to 18 years of age
2. Mitochondrial disorder established by a confirmed mutation in mtDNA or nuclear DNA
Exclusion criteria
- it is estimated that the study will be too burdensome to the patient
- insufficient knowledge of the Dutch language
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL44833.091.13 |