Natural History of MADD
Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorIcahn School of Medicine at Mount Sinai
About this trial
The objective of this study is to conduct a longitudinal, observational investigation to determine the natural history of Multiple Acyl-CoA Dehydrogenase Deficiency (MADD), delineate the spectrum of its clinical features and their progression, identify biomarkers, and develop and validate patient reported outcomes.
Eligibility criteria
Qualifiers
Have a biochemical and/or molecular diagnosis of Multiple Acyl-CoA Dehydrogenase Deficiency (MADD), as confirmed by a study investigator
Provision of signed and dated informed consent form (and assent when applicable) from subject or subject's legal representative
Disqualifiers
None
Trial design
Treatments tested in this trial
- Not listed
Trial groups
50 Participants
are grouped into 1 trial groupSponsors and collaborators
Source ClinicalTrials.gov