Biomarker for Wolman Disease (BioWolman) (NCT02383641) | Clinical Trial Compass
WithdrawnNot Applicable
Biomarker for Wolman Disease (BioWolman)
Stopped: Transition to BioMetabol
Germany, India, Sri Lanka0Started 2018-08-20
Plain-language summary
Development of a new MS-based biomarker for the early and sensitive diagnosis of Wolman disease 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 gender older than 2 month
* The patient has a diagnosis of Wolman disease or a high-grade suspicion for Wolman disease
High-grade suspicion present, if one or more inclusion criteria are valid:
* Positive family anamnesis for Wolman disease
* Vomiting, diarrhea
* Malnourishment, difficulty growing and gaining weight
* Enlarged liver and spleen (hepatosplenomegaly), which causes a distended abdomen
* Low muscle tone (hypotonia)
* Anemia
* x-ray reveals calcified adrenal glands
Exclusion Criteria:
* No Informed consent from the parents before any study related procedures.
* Patients of both gender younger than 2 month
* No diagnosis of Wolman disease or no valid criteria for profound suspicion of Wolman disease
What they're measuring
1
Development of a new MS-based biomarker for the early and sensitive diagnosis of Wolman disease from blood