Natural History of MADD | NCT07734090

NCT07734090

Overview

NCT ID: NCT07734090
Lead Sponsor: Icahn School of Medicine at Mount SinaiOTHER
Official Title: Natural History of Multiple Acyl-CoA Dehydrogenase Deficiency (MADD)
Brief Summary: 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.
Conditions: Multiple Acyl-CoA Dehydrogenase Deficiency
Interventions: No data available

Ages Eligible for Study: Child (0-17)Adult (18-64)Older Adult (65+)
Sexes Eligible for Study: All
Accepts Healthy Volunteers?: No
Eligibility Criteria: Inclusion Criteria:

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

Exclusion Criteria:

- Presence of a major unrelated condition

Central Contacts: Mary Freeman, MS, CGCContact
mary.freeman@mssm.edu(347) 668-9162