Biomarker for Sanfilippo Type A-B-C-D Disease (BioSanfilippo) (NCT02298686) | Clinical Trial Compass
WithdrawnNot Applicable
Biomarker for Sanfilippo Type A-B-C-D Disease (BioSanfilippo)
Stopped: Transition to BioMetabol
Egypt, Germany, India0Started 2018-08-20
Plain-language summary
Development of a new MS-based biomarker for the ear-ly and sensitive diagnosis of Sanfilippo Disease Type A-B-C-D from blood (plasma)
Who can participate
Age range2 Months
SexALL
See this in plain English?
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 patient or the parents before any study related procedures.
* Patients of both genders older than 2 month
* The patient has a diagnosis of Sanfilippo Type A-B-C-D disease or a high-grade suspicion for Sanfilippo Type A-B-C-D disease
* High-grade suspicion present, if one or more inclusion criteria are valid:
* Positive family anamnesis for Sanfilippo Type A-B-C-D disease
* Dysostosis multiplex without identifiable cause
* Splenomegaly without identifiable cause
* Hepatomegaly without identifiable cause
* Heparan sulfate excretion in urine
* CNS involvement without identifiable cause
EXCLUSION CRITERIA:
* No Informed consent from the patient or the parents before any study related procedures.
* Patients of both gender younger than 2 month
* No diagnosis of Sanfilippo Type A-B-C-D disease or no valid criteria for profound suspicion of Sanfilippo Type A-B-C-D disease
What they're measuring
1
Development of a new MS-based biomarker for the early and sensitive diagnosis of Sanfilippo Type A-B-C-D disease from plasma