Observational Study of Multiple Acyl-CoA Dehydrogenase Deficiency (MADD)
This is an observational study looking at the natural history of Multiple Acyl-CoA Dehydrogenase Deficiency (MADD), a genetic condition. Researchers want to understand how MADD changes over time, what its symptoms are, and to find ways to measure the disease's progression. This study is not testing a new treatment. You can join if you have a confirmed diagnosis of MADD. The main goal is to enroll 50 participants in a patient registry over five years. The current recruitment status is unclear.
- Study design
- This is an observational study, meaning researchers will watch and collect information without giving any specific treatments. It plans to include 50 participants.
- What's involved
- If you have a molecular diagnosis, you will have annual follow-ups (or every 6 months if under 2 years old). This involves standard medical assessments, questionnaires, and sample collection.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants with a molecular diagnosis will have annual follow-ups, or every 6 months for those under 2 years old.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Natural History of MADD
At a glance
Conditions
Where it's being run
2 sites across 2 statesStudy leadership
- Mary Kate LoPiccolo, MD · PRINCIPAL_INVESTIGATOR · Icahn School of Medicine at Mount Sinai
Who to contact
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Do you actually qualify for this trial?
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Inclusion
What this trial measures
- Number of participants enrolled in the patient registry5 years
The number of participants enrolled in the patient registry will be assessed.