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Natural History of MADD

RecruitingObservational study

Natural History of Multiple Acyl-CoA Dehydrogenase Deficiency (MADD)

Who can join

All ages · All sexes

Full 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

About the study

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.

Sponsor: Icahn School of Medicine at Mount Sinai · Participants: 50 · Started: 2026-10

Contact the study team

Official record on ClinicalTrials.gov — NCT07734090

Locations in the U.S.

ColoradoChildren's Hospital Colorado Anschutz Medical Campus, Aurora (Not yet recruiting)
New YorkIcahn School of Medicine at Mount Sinai, New York

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.