Natural History of MADD

ID#: NCT07734090

Age: Birth - 66+

Gender: All

Healthy Subjects: No

Recruitment Status: Recruiting

Start Date: October 01, 2026

End Date: July 01, 2031

Contact Information:
Mary Freeman, MS, CGC
(347) 668-9162
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.
Eligibility:

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