Biomarker for Mucolipidosis Disorder Type I, II, III, IV (BioML) (NCT02298673) | Clinical Trial Compass
WithdrawnNot Applicable
Biomarker for Mucolipidosis Disorder Type I, II, III, IV (BioML)
Stopped: Transition into BioMetabol
Egypt, Germany, India0Started 2018-08-20
Plain-language summary
Development of a new MS-based biomarker for the early and sensitive diagnosis of Mucolipidosis Disorder type I,II,III or IV from blood (plasma)
Who can participate
Age range2 Months
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
INCLUSION CRITERIA
* Informed consent will be obtained from the parents before any study related procedures.
* Patients of both genders older than 2 month
* The patient has a diagnosis of Mucolipidosis Disorder type I,II,III or IV or high-grade suspicion for Mucolipidosis Disorder type I,II,III or IV
* High-grade suspicion present, if one or more inclusion criteria are valid:
Positive family anamnesis for Mucolipidosis Disorder type I,II,III or IV
Skeletal abnormalities
Psychomotor retardation
Progressive failure to thrive
Hoarse voice
EXCLUSION CRITERIA
* No Informed consent from the parents before any study related procedures.
* Patients of both genders younger than 2 month
* No diagnosis of Mucolipidosis Disorder type I,II,III or IV or no valid criteria for profound suspicion of Mucolipidosis Disorder type I,II,III or IV
What they're measuring
1
Development of a new MS-based biomarker for the early and sensitive diagnosis of Mucolipidosis disorder type I, II, III or IV from blood (plasma).