Natural History of MADD
Part of paid clinical trials in Aurora, Colorado.
- Sponsor
- Icahn School of Medicine at Mount Sinai
- Study ID
- NCT07734090
- Status
- Not Yet Recruiting
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Conditions
- Multiple Acyl-CoA Dehydrogenase Deficiency
Eligibility Criteria
- Sex
- ALL
- Age
- N/A - N/A
- Healthy Volunteers
- Not accepted
Study Details
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.
Key Dates
- First listed
- Jul 29, 2026
- Start date
- Jul 31, 2026
- Status verified
- Jul 2026
- Primary completion
- Jul 31, 2031
- Completion
- Jul 31, 2031
Study Design
- Enrollment
- 50 participants (estimated)
Arms
- Arm: Multiple Acyl-CoA Dehydrogenase Deficiency (MADD)Patients with a molecular and/or biochemical diagnosis of Multiple Acyl-CoA Dehydrogenase Deficiency (MADD).
Primary Outcome Measure
Number of participants enrolled in the patient registry [ Time Frame: 5 years ]
Central Contacts
- Mary Freeman, MS, CGC(347) 668-9162
Locations (2)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| Children's Hospital Colorado Anschutz Medical Campus | Aurora | Colorado | 80045 | Johan Van Hove, MD, PhD (PRINCIPAL_INVESTIGATOR) |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | - |